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Stereotactic Radiosurgery (SRS) Dose-Escalation Study for Brain Metastasis (SRS)
SRS dose escalation for brain metastases in radiation-naïve patients will establish true tolerable doses, which may exceed the current standard doses. This may lead to an improvement in local control, patient survival, and/or quality-of life.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Robert Timmerman
ALL
18 Years and over
NA
NCT02645487
STU 022015-106
Inclusion Criteria
• Biopsy-proven non-hematopoietic malignancy, except for small cell lung cancer, germ cell cancer, or unknown primary tumor.
• Radiographic evidence by MRI (or by CT scan with CT contrast if ineligible or intolerant of MRI) of brain metastasis. (If patient is unable to tolerate MRI contrast, an MRI without contrast is acceptable if lesions are visible)
• All brain metastases must be outside the brain stem (midbrain, pons and medulla).
• Patient must have 10 or less brain metastases.
• The maximum diameter of any lesion must be less than or equal to 3.0 cm.
• Previous treatment with surgery, radiation, chemotherapy, immunotherapy or any targeted agents are allowed provided that: * Radiation was not to the brain. * Surgery to the brain was \> 7 days prior to SRS and there remains at least one additional brain metastasis that can be targeted with SRS
• Age ≥ 18 years.
• ECOG Performance Score of 2 or better/Karnofsky Performance Status score of 50-60 or better.
• All men, as well as women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. A female of child-bearing potential is any woman (regardless of sexual orientation, marital status, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria: * Has not undergone a hysterectomy or bilateral oophorectomy; or * Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months).
• Ability to understand and the willingness to sign a written informed consent. Exclusion Criteria
• Patients had craniotomy and surgery to the brain within 7 days from the date of SRS.
• Patients with leptomeningeal metastasis. NOTE: For the purposes of exclusion, LMD is a clinical diagnosis, defined as positive CSF cytology and/or equivocal radiologic or clinical evidence of leptomeningeal involvement. Patients with leptomeningeal symptoms in the setting of leptomeningeal enhancement by imaging (MRI) would be considered to have LMD even in the absence of positive CSF cytology, unless a parenchymal lesion can adequately explain the neurologic symptoms and/or signs. In contrast, an asymptomatic or minimally symptomatic patient with mild or nonspecific leptomeningeal enhancement (MRI) would not be considered to have LMD. In that patient, CSF sampling is not required to formally exclude LMD, but can be performed at the investigator's discretion based on level of clinical suspicion.
• Patients with a contraindication to both MRI (with or without contrast) and CT scan (with contrast)
• Patients with life expectancy \< 3 months.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that, in the opinion of the investigator, would limit compliance with study requirements.
• Subjects must not be pregnant or nursing at the time of SRS treatment due to the potential for congenital abnormalities and the potential of this regimen to harm nursing infants.
• Biopsy-proven non-hematopoietic malignancy, except for small cell lung cancer, germ cell cancer, or unknown primary tumor.
• Radiographic evidence by MRI (or by CT scan with CT contrast if ineligible or intolerant of MRI) of brain metastasis. (If patient is unable to tolerate MRI contrast, an MRI without contrast is acceptable if lesions are visible)
• All brain metastases must be outside the brain stem (midbrain, pons and medulla).
• Patient must have 10 or less brain metastases.
• The maximum diameter of any lesion must be less than or equal to 3.0 cm.
• Previous treatment with surgery, radiation, chemotherapy, immunotherapy or any targeted agents are allowed provided that: * Radiation was not to the brain. * Surgery to the brain was \> 7 days prior to SRS and there remains at least one additional brain metastasis that can be targeted with SRS
• Age ≥ 18 years.
• ECOG Performance Score of 2 or better/Karnofsky Performance Status score of 50-60 or better.
• All men, as well as women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. A female of child-bearing potential is any woman (regardless of sexual orientation, marital status, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria: * Has not undergone a hysterectomy or bilateral oophorectomy; or * Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months).
• Ability to understand and the willingness to sign a written informed consent. Exclusion Criteria
• Patients had craniotomy and surgery to the brain within 7 days from the date of SRS.
• Patients with leptomeningeal metastasis. NOTE: For the purposes of exclusion, LMD is a clinical diagnosis, defined as positive CSF cytology and/or equivocal radiologic or clinical evidence of leptomeningeal involvement. Patients with leptomeningeal symptoms in the setting of leptomeningeal enhancement by imaging (MRI) would be considered to have LMD even in the absence of positive CSF cytology, unless a parenchymal lesion can adequately explain the neurologic symptoms and/or signs. In contrast, an asymptomatic or minimally symptomatic patient with mild or nonspecific leptomeningeal enhancement (MRI) would not be considered to have LMD. In that patient, CSF sampling is not required to formally exclude LMD, but can be performed at the investigator's discretion based on level of clinical suspicion.
• Patients with a contraindication to both MRI (with or without contrast) and CT scan (with contrast)
• Patients with life expectancy \< 3 months.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that, in the opinion of the investigator, would limit compliance with study requirements.
• Subjects must not be pregnant or nursing at the time of SRS treatment due to the potential for congenital abnormalities and the potential of this regimen to harm nursing infants.
RADIATION: Stereotactic Radiosurgery
Brain Neoplasms, Adult, Malignant, Brain and Nervous System, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Cardiovascular, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Eye and Orbit, Gall Bladder, Head and Neck, Hodgkins Lymphoma, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Lymphoid Leukemia, Lymphoma, Melanoma, skin, Multiple Myeloma, Nose, Other, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Sarcoma, Small Intestine, Soft Tissue, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva
UT Southwestern; Parkland Health & Hospital System
JoLT-Ca Sublobar Resection (SR) Versus Stereotactic Ablative Radiotherapy (SAbR) for Lung Cancer (STABLE-MATES)
To Determine if SAbR improves survival over SR in High Risk Operable Stage I NSCLC
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Robert Timmerman
ALL
18 Years and over
PHASE3
NCT02468024
STU 022015-069
• 0 Inclusion Criteria
• 1 Age \> 18 years.
• 2 ECOG/Zubrod performance status (PS) 0, 1, or 2 (reference Appendix C).
• 3 Radiographic findings consistent with non-small cell lung cancer, including lesions with ground glass opacities with a solid component of 50% or greater.
• 4 The primary tumor in the lung must be biopsy confirmed non-small cell lung cancer within 180 days prior to randomization.
• 5 Tumor ≤ 4 cm maximum diameter, including clinical stage IA and selected IB by PET or PET integrated with a simultaneous CT scan (PET-CT) of the chest and upper abdomen performed within 180 days prior to randomization (reference Appendix A \& B). Repeat imaging within 90 days prior to randomization is recommended for re-staging but is not required based on institutional norms.
• 6 All clinically suspicious mediastinal N1, N2, or N3 lymph nodes (\> 1 cm short-axis dimension on CT scan and/or positive on PET scan) confirmed negative for involvement with NSCLC by one of the following methods: mediastinoscopy, anterior mediastinotomy, EUS/EBUS guided needle aspiration, CT-guided, video-assisted thoracoscopic or open lymph node biopsy within 180 days of randomization.
• 7 Tumor verified by a thoracic surgeon to be in a location that will permit sublobar resection.
• 8 Tumor located peripherally within the lung. NOTE: Peripheral is defined as not touching any surface within 2 cm of the proximal bronchial tree in all directions. See bronchial tree diagram below. Patients with non-peripheral (central) tumors are NOT eligible.
• 9 No evidence of distant metastases.
• 10 Availability of pulmonary function tests (PFTs - spirometry, DLCO, +/- arterial blood gases) within 180 days prior to registration. Patients with tracheotomy, etc, who are physically unable to perform PFTs (and therefore cannot be tested for the Major criteria in 3.1.11 below) are potentially still eligible if a study credentialed thoracic surgeon documents that the patient's health characteristics would otherwise have been acceptable for eligibility as a high risk but nonetheless operable patient (in particular be eligible for sublobar resection).
• 11 To define eligibility of patients being at high risk for surgery, certain criteria must be met. Any one (1) of the following major criteria will define the high risk status for eligibility: Major Criteria * FEV1 ≤ 50% predicted (pre-bronchodilator value) * DLCO ≤ 50% predicted (pre-bronchodilator value) * Study credentialed thoracic surgeon believes the patient is potentially operable but that a lobectomy or pneumonectomy would be poorly tolerated by the patient for tangible or intangible reasons. The belief must be declared and documented in the medical record prior to randomization. If any of the major criteria are met, the patient is eligible based on high risk for surgery and minor criteria do not need to be considered. However, if no major criteria is met, at least two (2) minor criteria being met will also define eligibility for meeting the high risk status. Any two (2) of the following minor criteria will define the high risk status for eligibility: * Minor Criteria * Age ≥75 * FEV1 51-60% predicted (pre-bronchodilator value) * DLCO 51-60% predicted (pre-bronchodilator value) * Pulmonary hypertension (defined as a pulmonary artery systolic pressure greater than 40mm Hg) as estimated by echocardiography or right heart catheterization * Poor left ventricular function (defined as an ejection fraction of 40% or less) * Resting or Exercise Arterial pO2 ≤ 55 mm Hg or SpO2 ≤ 88% * pCO2 \> 45 mm Hg * Modified Medical Research Council (MMRC) Dyspnea Scale ≥ 3.
• 12 No prior intra-thoracic radiation therapy for previously identified intra-thoracic primary tumor (e.g. previous lung cancer) on the ipsilateral side. NOTE: Previous radiotherapy as part of treatment for head and neck, breast, or other non-thoracic cancer is permitted to the ipsilateral side so long as possible radiation fields would not overlap. NOTE: Radiotherapy to the contralateral lung is allowed so long as it was completed more than 3 years prior to randomization and there is no overlap of radiation fields.
• 13 Previous chemotherapy, radiotherapy, or surgical resection specifically for the lung cancer being treated on this protocol is NOT permitted.
• 14 No prior lung resection on the ipsilateral side.
• 15 Non-pregnant and non-lactating. Women of child-bearing potential must have a negative urine or serum pregnancy test prior to registration. Peri-menopausal women must be amenorrheic \> 12 months prior to registration to be considered not of childbearing potential.
• 16 No prior invasive malignancy, unless disease-free for ≥ 3 years prior to registration (exceptions: non-melanoma skin cancer, in-situ cancers).
• 17 Ability to understand and sign a written informed consent.
• 0 Exclusion Criteria
• 1 Age \<18
• 2 ECOG/Zubrod performance status (PS) greater than 3.
• 3 Radiographic findings with ground glass opacities and less than 50% solid component will be excluded.
• 4 The primary tumor in the lung, biopsy confirmed non-small cell lung cancer greater than 180 days prior to randomization.
• 5 Tumor \> 5 cm maximum diameter, including clinical stage IA and selected IB by PET or PET integrated with a simultaneous CT scan (PET-CT) of the chest and upper abdomen and/or performed greater than 180 days prior to randomization.
• 6 Lymph node biopsy greater than 180 days prior to randomization.
• 7 Thoracic surgeon confirms unable to remove tumor with sublobar resection.
• 8 Tumor located non-peripheral (central) region of lung (see bronchial tree diagram in 3.1.8).
• 9 Evidence of distant metastases.
• 10 Pulmonary function test (PFT - spirometry, DLCO, +/- arterial blood gases) greater than 180 days prior to registration. Patients physically unable to perform PFT's, such as patients with tracheotomy, that do not have written documentation from study credentialed thoracic surgeon stating eligibility.
• 11 Patients that do not meet either Major criteria or Minor criteria.
• 12 Prior intra-thoracic radiation therapy on ipsilateral side. Radiotherapy to the contralateral lung completed less than 3 years prior to randomization, with radiation field overlap.
• 13 Prior chemotherapy, radiotherapy, or surgical resection specifically for the lung cancer being treated on this protocol.
• 14 Prior lung resection on the ipsilateral side.
• 15 Pregnant and lactating women.
• 16 Prior invasive malignancy and less than 3 years disease free prior to registration (unless non-melanoma skin cancer, in-situ cancers).
• 17 Unable to understand and/ or sign a written informed consent.
PROCEDURE: Lung Surgery, RADIATION: Radiation therapy
Non-Small Cell Lung Cancer, Lung/Thoracic
UT Southwestern; Parkland Health & Hospital System
CAR-T Long Term Follow Up (LTFU) Study (PAVO)
Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow up of treated patients is required. The purpose of this study is to monitor all patients exposed to CAR-T therapied for 15 years following their last CAR-T (e.g. CTL019) infusion to assess the risk of delayed adverse events (AEs), monitor for replication competent lentivirus (RCL) and assess long-term efficacy, including vector persistence.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Samuel John
ALL
0 Years to 100 Years old
PHASE3
NCT02445222
STU 032015-068
Inclusion Criteria:
* All patients who have received a CAR-T therapy and completed or discontinued early from a Novartis sponsored treatment protocol that utilized CAR-T cells or from any CAR-T trial sponsored by the University of Pennsylvania with which Novartis has a contractual agreement to co-develop the CAR technology.
* Patients who have provided informed consent for the long term follow up study prior to their study participation .
Exclusion Criteria:
* There are no specific exclusion criteria for this study. GENETIC: Previously treated CAR-T patients
Long Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access Program, Leukemia, Other
Children’s Health
Safety and Durability of Sirolimus for Treatment of LAM (MIDAS)
The MIDAS study aims to follow male and female LAM patients who are currently taking, have previously failed or been intolerant of, or may (at some time in the future) take mTOR inhibitors (sirolimus or everolimus) as part of their clinical care. Adult female TSC patients may also enroll, with or without lung cysts.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Rhoda.AnnohGordon@UTSouthwestern.edu
Carlos Girod
FEMALE
18 Years and over
NCT02432560
STU 022017-055
Inclusion Criteria:
* Female or male, age 18 or over
* Diagnosis of LAM based on ATS/JRS criteria
* Signed and dated informed consent
* On chronic therapy, newly treated or may be considered for therapy with mTOR inhibitors or previously intolerant of or having failed mTOR inhibitor therapy
Exclusion Criteria:
* Inability to attend at least one RLD Clinic visit per year
* Inability to give informed consent
* Inability or unwillingness to perform pulmonary function testing DRUG: Sirolimus, DRUG: Everolimus
Lymphangioleiomyomatosis, Lung/Thoracic
Lymphangioleiomyomatosis, LAM, rare lung, Rare Lung Disease
UT Southwestern
Study to Determine the Pharmacokinetics and Pharmacodynamic Effects of Phenylephrine on BP Via IV
The primary objective of this study is to evaluate the dose effect of Phenylephrine Hydrochloride Injection on the treatment of clinically relevant decreased blood pressure in the pediatric population, ≥12 to 16 year old patients undergoing general and neuraxial anesthesia. The secondary objectives are to describe changes in blood pressure and heart rate, time to onset and to maximal response, and the duration of response; to assess the safety of the product in this population; and to characterize the pharmacokinetics of phenylephrine hydrochloride.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kiley.Poppino@UTSouthwestern.edu
Peter Szmuk
All
12 Years to 16 Years old
Phase 4
NCT02323399
STU 082014-004
Inclusion Criteria:
• Subject's age is between ≥12 and 16 years, inclusive
• Subject is scheduled for a procedure that requires general or neuraxial anesthesia
• Subjects must have normal or clinically acceptable physical exam
• Subjects with controlled diabetes prior to entry must have a mean systolic/diastolic office blood pressure ≤128/78 mmHg (sitting, after 5 minutes of rest)
• Females must have a urine or serum pregnancy test (Human Chorionic Gonadotropin) that is negative at Screening and Day 1
• Subject's parent or legal guardian gives informed consent and subject gives assent.
Exclusion Criteria:
• Subject has a contraindication to vasoconstrictor therapy for control of blood pressure
• Subject has participated in other clinical trials for investigational drugs and/or devices within 30 days prior to enrollment
• Subject has any serious medical condition which, in the opinion of the investigator, is likely to interfere with study procedures
• Subjects who have a history of any clinically significant local or systemic infectious disease within four weeks prior to initial treatment administration
• Subjects who are positive for hepatitis B surface antigen or hepatitis C antibody
• Subjects taking antihypertensive medication
• Subject is moribund (death is likely to occur in less than 48 hours)
• Females who are pregnant, nursing or unwilling to use/practice adequate contraception.
Drug: Phenylephrine
Hypotension
Children’s Health
LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell Histiocytosis
The LCH-IV is an international, multicenter, prospective clinical study for pediatric Langerhans Cell Histiocytosis LCH (age \< 18 years).
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Erin Butler
ALL
up to 18 Years old
PHASE2
NCT02205762
STU-2018-0071
Inclusion Criteria:
* Stratum I
* Patients must be less than 18 years of age at the time of diagnosis.
* Patients must have histological verification of the diagnosis of Langerhans cell histiocytosis according to the criteria described in Section 6.1
* Signed informed consent form
* Stratum II
* Patients of Stratum I who have:
* Progressive disease (AD worse) in non-risk organs after 6 weeks (Initial Course
* AD intermediate or worse in non-risk organs or AD better in risk organs after 12 weeks (Initial Course 2)
* Disease progression (AD worse) in non-risk organs at any time during continuation treatment
* Active disease at the end of Stratum I treatment
* Disease reactivation in non-risk organs at any time after completion of Stratum I treatment
* Stratum III
* Patients from Stratum I who fulfill the following criteria:
* AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD intermediate in risk organs after week 12 (after Initial Course 2).
* Presence of unequivocally severe organ dysfunction at the above mentioned evaluation points (hematological dysfunction, liver dysfunction, or both of them) as
* Hb \<70 g/L (\<7.0 g/dl) and/or transfusion dependency
* PLT \<20 x109/L (20,000/μL) and/or transfusion dependency (both criteria have to be fulfilled) AND/OR
* Liver dysfunction (or digestive involvement with protein loss)
* Total protein \<55 g/L or substitution dependency
* Albumin \<25 g/L or substitution dependency (at least one of the two criteria to be fulfilled)
* Stratum IV
* Patients from Stratum I or Stratum III who fulfill the following criteria:
* AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD intermediate in risk organs after week 12 (after Initial Course 2) of Stratum I OR
* AD worse after the 2nd and 3rd 2-CdA/Ara-C course, and those AD worse or AD intermediate after the 4th 2-CdA/Ara-C course of Stratum III AND
* Presence of unequivocally severe organ dysfunction at the above mentioned evaluation points (hematological dysfunction, liver dysfunction, or both of them) as defined in Table XI (see Section 10.3.1).
* Informed consent: All patients or their legal guardians (if the patient is \<18 years of age) must sign an Ethics or institutional Review Board approved consent form indicating their awareness of the investigational nature and the risks of this study. When appropriate, younger patients will be included in all discussions in order to obtain assent.
* Adequate organ function: Patients should have adequate hepatic, renal, cardiac and pulmonary function to undergo reduced intensity HCT based upon local institutional guidelines, or at a minimum meet requirements noted in eligibility checklist Appendix A-VIII_1. However, significant hepatic and pulmonary dysfunction, if secondary to underlying LCH disease activity, will not exclude patients from protocol enrollment and should be discussed with the National PI Coordinator and the Coordinating Principal Investigator.
* Stratum V
* All patients with verified diagnosis of LCH and MRI findings consistent with ND-CNSLCH irrespective of previous treatments (also those not registered to other Strata ofLCH-IV).
* Patients with isolated tumorous CNS-LCH (including isolated DI with mass lesion in the hypothalamus-pituitary axis). In patients with already established diagnosis of LCH and radiologic finding of CNS lesions compatible with LCH, a biopsy of the lesion is not obligatory. In all other cases a biopsy of the lesion is needed for inclusion into the study
* Stratum VI
-- Patients with newly diagnosed SS-LCH and localization other than "multifocal bone",isolated tumorous CNS lesion, or isolated "CNS-risk" lesion.
* Stratum VII -- All patients registered in LCH IV (regardless of treatment) as long as consent for longterm follow-up has not been withheld.
Exclusion Criteria:
* Stratum I
* Pregnancy (patients of child-bearing age must be appropriately tested before chemotherapy)
* LCH-related permanent consequences (e.g. vertebra plana, sclerosing cholangitis, lung fibrosis, etc.) in the absence of active disease
* Prior systemic therapy
* Stratum II
* Patients with progressive disease in risk organs
* Permanent consequences (e.g. sclerosing cholangitis, lung fibrosis, etc.) without evidence of active LCH in the same organ or in any other locations
* No written consent of the patient or his/her parents or legal guardian
* Stratum III
* The presence of any of the following criteria will exclude the patient from the study:
* Isolated sclerosing cholangitis without evidence of active hepatic LCH as the only evidence of risk organ involvement.
* Inadequate renal function as defined by serum creatinine \> 3x normal for age
* Stratum IV
* Pulmonary failure (requiring mechanical ventilation) not due to active LCH.
* Isolated liver sclerosis or pulmonary fibrosis, without active LCH.
* Uncontrolled active life-threatening infection.
* Decreased renal function with a GFR of less than 50ml/1.73m2/min.
* Pregnancy or active breast feeding
* Failure to provide signed informed consent
* Stratum VI
* Patients with SS-LCH who have an isolated tumorous CNS lesion (they are eligible for Stratum V),
* Patients with isolated "CNS-risk" or multifocal bone lesions (they are eligible for Stratum I, Group 2) DRUG: Prednisone, DRUG: Vinblastine, DRUG: mercaptopurine, DRUG: INDOMETHACIN, DRUG: Methotrexate, DRUG: Cytosine Arabinoside, DRUG: 2-chlorodeoxyadenosine, PROCEDURE: hematopoietic stem cell transplantation (RIC-HSCT), BIOLOGICAL: Intravenous immunoglobulin
Langerhans Cell Histiocytosis, Liver, Bones and Joints, Brain and Nervous System, Lung/Thoracic, Other Hematopoietic
Langerhans cell histiocytosis
Children’s Health
Adjuvant Curcumin to Assess Recurrence Free Survival in Patients Who Have Had a Radical Prostatectomy
This is a prospective study to determine if the adjuvant use of Curcumin improves recurrence-free survival.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Yair Lotan
MALE
30 Years to 80 Years old
PHASE3
NCT02064673
STU 042013-080
Inclusion Criteria:
* Status post radical prostatectomy for histologically confirmed adenocarcinoma of the prostate
* pathologically confirmed T1-T3 disease
* no sign of lymph node or metastatic disease
* pT1-pT3pNxMx patients in whom standard NCCN or AUA guidelines would suggest are at low risk for pelvic lymph node or metastatic disease and who would not require confirmatory imaging for metastatic disease. This includes patients with Gleason 6 or 7(T2 disease) and PSA less than 20.
* Eastern Cooperative Oncology Group(ECOG) status 0-2
* adequate renal and liver function as well as bone marrow reserve (measured serum creatinine \<2mg/dl, bilirubin ≤ 1.5 mg/dl, ANC ≥ 1.5 x 10 (3) uL, platelets ≥ 50 x K/uLL, and hemoglobin ≥ 10 g/dL)
* 30-80 y/o at time of diagnosis with a life expectancy of \>= 3 yrs
* focally positive surgical margins are permitted
* no plan to receive adjuvant hormone or radiation therapy
* PSA at the time of enrollment must be undetectable
* life expectancy of 3 years
Exclusion Criteria:
* must not have exceeded 3 months from time of surgery to enrollment into study
* T3b or T4 or node positive disease
* macroscopic residual disease after surgery
* hormone therapy before surgery
* history of gallbladder problems or gallstones, or biliary obstruction, unless patient had cholecystectomy
* radiation therapy as primary treatment after surgery
* INR value greater than 1.5
* AST/ALT are equal or greater than 2 times the upper limit of normal
* antiplatelet or anticoagulant agents- patients taking 81mg of Aspirin will be allowed with close observation
* history of gastric or duodenal ulcers or untreated hyperacidity syndromes
* patients who are currently taking curcumin and are unwilling to stop or plan to take curcumin during the study DRUG: Curcumin, DRUG: placebo
Prostate Cancer, Prostate
prostate cancer, radical prostatectomy
UT Southwestern; Parkland Health & Hospital System
Metabolic Biomarkers in Thoracic Cancers
The purpose of this research study is to develop a method of using magnetic resonance imaging (MRI) to evaluate lung tumors and other thoracic malignancies. An MRI is a scanning device that uses magnets to make images (pictures) of the body. This study is being done to determine what series of reactions (metabolic pathways) pulmonary nodules use as they burn sugar as fuel for growth. The manner in which the tumor burns (metabolizes) sugar for fuel is being investigated by using a natural, slightly modified, sugar solution (13C-glucose) and studying a small sample of the tumor once it is removed at the time of surgery.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Kemp Kernstine
ALL
18 Years and over
NA
NCT02095808
STU 052012-065
Inclusion Criteria:
• Patients must have known or probable malignant lesions requiring surgical biopsy or excision.
• Subjects of all races and ethnic origins over 18 years of age.
Exclusion Criteria:
• Not a surgical candidate.
• Poorly controlled diabetes.
PROCEDURE: Imaging Biomarkers
Lung Cancer, Lung/Thoracic
UT Southwestern
Testing Docetaxel-Cetuximab or the Addition of an Immunotherapy Drug, Atezolizumab, to the Usual Chemotherapy and Radiation Therapy in High-Risk Head and Neck Cancer
This phase II/III trial studies how well radiation therapy works when given together with cisplatin, docetaxel, cetuximab, and/or atezolizumab after surgery in treating patients with high-risk stage III-IV head and neck cancer the begins in the thin, flat cells (squamous cell). Specialized radiation therapy that delivers a high dose of radiation directly to the tumor may kill more tumor cells and cause less damage to normal tissue. Drugs used in chemotherapy, such as cisplatin and docetaxel, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Cetuximab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. Immunotherapy with monoclonal antibodies, such as atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. The purpose of this study is to compare the usual treatment (radiation therapy with cisplatin chemotherapy) to using radiation therapy with docetaxel and cetuximab chemotherapy, and using the usual treatment plus an immunotherapy drug, atezolizumab.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
David Sher
ALL
18 Years and over
PHASE2
NCT01810913
STU 022013-055
Inclusion Criteria:
* PHASE II INCLUSION CRITERIA (COMPLETE AS OF 20-MAR-2020)
* Pathologically (histologically or cytologically) proven diagnosis of head and neck squamous cell carcinoma (HNSCC) involving the oral cavity (excluding lips), oropharynx (p16 negative), larynx, or hypopharynx
* Patients must have undergone gross total surgical resection of high-risk oral cavity, oropharynx (p16 negative), larynx, or hypopharynx within 63 days prior to registration; Note: patients may have biopsy under general anesthesia in an operating room followed by definitive ablative cancer surgery representing gross total resection; the gross total resection has to be done within 63 days prior to registration; if, however, patients have ablative resection but shortly recur or are determined to have persisting disease requiring re-resection to achieve gross total resection, then the patient is not eligible
* Patients must have at least 1 of the following high-risk pathologic features: extracapsular nodal extension or invasive cancer at the primary tumor resection margin (tumor on ink)
* Pathologic stage III or IV HNSCC, including no distant metastases, based upon the following minimum diagnostic workup:
* General history and physical examination by a radiation oncologist and/or medical oncologist within 84 days prior to registration;
* Examination by an ear nose throat (ENT) or head \& neck surgeon prior to surgery; a laryngopharyngoscopy (mirror and/or fiber optic and/or direct procedure), if appropriate, is recommended but not required; intra-operative examination is acceptable documentation
* Pre-operative (op) Imaging of the head and neck: A neck computed tomography (CT) (with contrast) or CT/positron emission tomography (PET) (with contrast) and/or an magnetic resonance imaging (MRI) of the neck (T1 with gadolinium and T2) within 84 days prior to surgery; Note: this imaging data (diagnostic pre-operative scan showing gross disease) is to be submitted in Digital Imaging and Communications in Medicine (DICOM) format via TRIAD; the report is to be uploaded into Rave
* Chest CT scan (with or without contrast) or CT/PET that includes the chest (with or without contrast) either within 84 days prior to surgery or within 120 days prior to registration; Note: if the CT/PET with or without contrast is done within 84 days prior to surgery, it fulfills the chest imaging requirement
* Zubrod performance status of 0-1 within 14 days prior to registration
* Age \>= 18
* Absolute granulocyte count (AGC) \>= 1,500 cells/mm\^3 (obtained within 14 days prior to registration on study)
* Platelets \>= 100,000 cells/mm\^3 (obtained within 14 days prior to registration on study)
* Hemoglobin \>= 8.0 g/dl (Note: the use of transfusion or other intervention to achieve hemoglobin \[Hgb\] \>= 8.0 g/dl is acceptable)
* Total bilirubin \< 2 x institutional upper limit of normal (ULN) within 14 days prior to registration
* Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \< 3 x institutional ULN within 14 days prior to registration
* Serum creatinine institutional ULN within 14 days prior to registration or; creatinine clearance (CC) \>= 50 ml/min within 14 days prior to registration determined by 24-hour collection or estimated by Cockcroft-Gault formula
* Negative urine or serum pregnancy test within 14 days prior to registration for women of childbearing potential
* The following assessments are required within 14 days prior to registration: sodium (Na), potassium (K), chloride (Cl), glucose, calcium (Ca), magnesium (Mg), and albumin; Note: patients with an initial magnesium \< 0.5 mmol/L (1.2 mg/dl) may receive corrective magnesium supplementation but should continue to receive either prophylactic weekly infusion of magnesium and/or oral magnesium supplementation (e.g., magnesium oxide) at the investigator's discretion
* Patients with feeding tubes are eligible for the study
* Women of childbearing potential and male participants who are sexually active must agree to use a medically effective means of birth control
* Patient must provide study specific informed consent prior to study entry, including consent for mandatory tissue submission for epidermal growth factor receptor (EGFR) analysis and for oropharyngeal cancer patients, human papilloma virus (HPV) analysis
* PHASE III: Pathologically (histologically or cytologically) proven diagnosis of head and neck squamous cell carcinoma (HNSCC) involving the oral cavity (excluding lips), oropharynx (p16 negative), larynx, or hypopharynx
* PHASE III: Patients with oropharyngeal cancer must have p16-negative based on central review prior to Step 2 registration. All patients with oropharyngeal primary must consent for mandatory tissue submission for central p16 confirmation
* PHASE III: Patients must have undergone gross total surgical resection of high-risk oral cavity, oropharynx (p16 negative), larynx, or hypopharynx within 63 days prior to registration
* Note: Patients may have biopsy under general anesthesia in an operating room followed by definitive ablative cancer surgery representing gross total resection. The gross total resection has to be done within 63 days prior to registration. If, however, patients have ablative resection but shortly recur or are determined to have persisting disease requiring re-resection to achieve gross total resection, then the patient is not eligible
* PHASE III: Patients must have at least 1 of the following high-risk pathologic features: extracapsular nodal extension or invasive cancer at the primary tumor resection margin (tumor on ink or tumor in a final separately submitted margin)
* PHASE III: Pathologic stage III or IV HNSCC (American Joint Committee on Cancer \[AJCC\] 7th edition), including no distant metastases, based upon the following minimum diagnostic workup:
* General history and physical examination by a radiation oncologist or medical oncologist within 84 days prior to registration;
* Examination by an ENT or head \& neck surgeon prior to surgery; a laryngopharyngoscopy (mirror or fiberoptic or direct procedure), if appropriate, is recommended but not required. Intra-operative examination is acceptable documentation.
* Pre-op Imaging of the head and neck: A neck CT (with contrast and of diagnostic quality) or PET/CT (with contrast and of diagnostic quality) and/or an MRI of the neck of diagnostic quality (T1 with gadolinium and T2) within 84 days prior to surgery; Note: this imaging data (diagnostic pre-operative scan showing gross disease) is to be submitted in DICOM format via TRIAD. The report is to be uploaded into Rave.
* Chest CT scan (with or without contrast) or PET/CT that includes the chest (with or without contrast) either within 84 days prior to surgery or within 120 days prior to registration; Note: If the PET/CT with or without contrast is done within 84 days prior to surgery, it fulfills the chest imaging requirement
* PHASE III: Zubrod performance status of 0-1 within 14 days prior to registration
* PHASE III: Age \>= 18
* PHASE III: Leukocytes \>= 2,500 cells/mm\^3 (obtained within 14 days prior to registration on study)
* PHASE III: Absolute neutrophil count (ANC) \>= 1,500 cells/mm\^3 (obtained within 14 days prior to registration on study)
* PHASE III: Platelets \>= 100,000 cells/mm\^3 (obtained within 14 days prior to registration on study)
* PHASE III: Hemoglobin \>= 8.0 g/dL (Note: The use of transfusion or other intervention to achieve Hgb \>= 8.0 g/dL is acceptable) (obtained within 14 days prior to registration on study)
* PHASE III: Total bilirubin =\< 1.5 x institutional upper limit of normal (ULN) (however, patients with known Gilbert disease who have serum bilirubin level =\< 3 x institutional ULN may be enrolled) (within 14 days prior to registration)
* PHASE III: AST or ALT =\< 3 x institutional ULN (within 14 days prior to registration)
* PHASE III: Alkaline phosphatase =\< 2.5 x institutional ULN (within 14 days prior to registration)
* PHASE III: Creatinine clearance (CrCl) \>= 50 mL/min within 14 days prior to registration determined by 24-hour collection or estimated by Cockcroft-Gault formula
* PHASE III: Patients with feeding tubes are eligible for the study
* PHASE III: Negative urine or serum pregnancy test within 14 days prior to registration for women of childbearing potential
* PHASE III: All patients must provide study specific informed consent prior to study entry
* PHASE III: Patients positive for human immunodeficiency virus (HIV) are allowed on study, but HIV-positive patients must have:
* A stable regimen of highly active anti-retroviral therapy (HAART);
* No requirement for concurrent antibiotics or antifungal agents for the prevention of opportunistic infections;
* A CD4 count above 250 cells/mcL and an undetectable HIV viral load on standard polymerase chain reaction (PCR)-based tests
Exclusion Criteria:
* PHASE II EXCLUSION CRITERIA (COMPLETE AS OF 20-MAR-2020)
* Prior invasive malignancy (except non-melanomatous skin cancer) unless disease free for a minimum of 1095 days (3 years); noninvasive cancers (for example, carcinoma in situ of the breast, oral cavity, or cervix are all permissible) are permitted even if diagnosed and treated \< 3 years ago
* Patients with simultaneous primaries or bilateral tumors are excluded, with the exception of patients with bilateral tonsil cancers or patients with T1-2, N0, M0 resected differentiated thyroid carcinoma, who are eligible
* Prior systemic chemotherapy or anti-epidermal growth factor (EGF) therapy for the study cancer; note that prior chemotherapy for a different cancer is allowable
* Prior radiotherapy to the region of the study cancer that would result in overlap of radiation therapy fields
* Severe, active co-morbidity, defined as follows:
* Unstable angina and/or congestive heart failure requiring hospitalization within 6 months prior to registration
* Transmural myocardial infarction within 6 months prior to registration
* Acute bacterial or fungal infection requiring intravenous antibiotics at the time of registration
* Chronic obstructive pulmonary disease exacerbation or other respiratory illness requiring hospitalization or precluding study therapy at the time of registration
* Idiopathic pulmonary fibrosis or other severe interstitial lung disease that requires oxygen therapy or is thought to require oxygen therapy within 1 year prior to registration
* Hepatic insufficiency resulting in clinical jaundice and/or coagulation defects; note, however, that laboratory tests for coagulation parameters are not required for entry into this protocol
* Acquired immune deficiency syndrome (AIDS) based upon current Centers for Disease and Control and Prevention (CDC) definition; note: human immunodeficiency virus (HIV) testing is not required for entry into this protocol; the need to exclude patients with AIDS from this protocol is necessary because the treatments involved in this protocol may be significantly immunosuppressive; protocol-specific requirements may also exclude immuno-compromised patients.
* Grade 3-4 electrolyte abnormalities (Common Terminology Criteria for Adverse Events \[CTCAE\], version \[v.\] 4):
* Serum calcium (ionized or adjusted for albumin) \< 7 mg/dl (1.75 mmol/L) or \> 12.5 mg/dl (\> 3.1 mmol/L) despite intervention to normalize levels
* Glucose \< 40 mg/dl (\< 2.2 mmol/L) or \> 250 mg/dl (\> 14 mmol/L)
* Magnesium \< 0.9 mg/dl (\< 0.4 mmol/L) or \> 3 mg/dl (\> 1.23 mmol/L) despite intervention to normalize levels
* Potassium \< 3.0 mmol/L or \> 6 mmol/L despite intervention to normalize levels
* Sodium \< 130 mmol/L or \> 155 mmol/L despite intervention to normalize levels
* Pregnancy or women of childbearing potential and men who are sexually active and not willing/able to use medically acceptable forms of contraception; this exclusion is necessary because the treatment involved in this study may be significantly teratogenic
* Prior allergic reaction to cetuximab
* PHASE III: Prior invasive malignancy (except non-melanomatous skin cancer) unless disease free for a minimum of 1095 days (3 years) with the following exceptions: T1-2, N0, M0 resected differentiated thyroid carcinoma; Note that noninvasive cancers (For example, carcinoma in situ of the breast, oral cavity, or cervix) are permitted even if diagnosed and treated \< 3 years ago
* PHASE III: Patients with simultaneous primaries or bilateral tumors are excluded, with the exception of patients with bilateral tonsil cancers or patients with T1-2, N0, M0 resected differentiated thyroid carcinoma, who are eligible
* PHASE III: Prior systemic therapy, including cytotoxic chemotherapy, biologic/targeted therapy (such as anti-EGF therapy), or immune therapy for the study cancer; note that prior chemotherapy for a different cancer is allowable, however, a prior anti-PD-1, anti-PD-L1, or anti-PD-L2 agent is not permitted
* PHASE III: Prior radiotherapy to the region of the study cancer that would result in overlap of radiation therapy fields
* PHASE III: Severe, active co-morbidity, defined as follows:
* Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification; to be eligible for this trial, patients should be class 2B or better within 6 months prior to registration
* Transmural myocardial infarction within 6 months prior to registration;
* Severe infections within 4 weeks prior to registration including, but not limited to, hospitalization for complications of infection, bacteremia, or severe pneumonia;
* Acute bacterial or fungal infection requiring intravenous antibiotics at the time of registration; Note: Patients receiving prophylactic antibiotics (e.g., for prevention of a urinary tract infection or chronic obstructive pulmonary disease exacerbation) are eligible.
* Chronic obstructive pulmonary disease exacerbation or other respiratory illness requiring hospitalization or precluding study therapy at the time of registration;
* History of idiopathic pulmonary fibrosis, pneumonitis (including drug induced), organizing pneumonia (i.e., bronchiolitis obliterans, cryptogenic organizing pneumonia, etc.), or evidence of active pneumonitis on screening chest computed tomography (CT) scan. History of radiation pneumonitis in a prior radiation field (fibrosis) is permitted, provided that field does not overlap with the planned radiation field for the study cancer;
* Patients with active tuberculosis (TB) are excluded;
* Known clinically significant liver disease, including active viral, alcoholic, or other hepatitis; cirrhosis; fatty liver; and inherited liver disease;
* Patients with past or resolved hepatitis B infection (defined as having a negative hepatitis B surface antigen \[HBsAg\] test and a positive anti-HBc \[antibody to hepatitis B core antigen\] antibody test) are eligible.
* Patients positive for hepatitis C virus (HCV) antibody are eligible only if polymerase chain reaction (PCR) is negative for HCV RNA.
* History of allogeneic bone marrow transplantation or solid organ transplantation.
* A diagnosis of immunodeficiency:
* Acquired immune deficiency syndrome (AIDS) based upon current CDC definition; note: HIV testing is not required for entry into this protocol; the need to exclude patients with AIDS from this protocol is necessary because the treatments involved in this protocol may be significantly immunosuppressive.
* Is receiving treatment with systemic immunosuppressive medications (including, but not limited to, prednisone, cyclophosphamide, azathioprine, methotrexate, thalidomide, and anti-tumor necrosis factor \[anti-TNF\] agents) within 2 weeks prior to registration.
* Note: Patients who have received acute, low dose, systemic immunosuppressant medications (e.g., a one-time dose of dexamethasone for nausea) may be enrolled.
* Note: The use of inhaled corticosteroids and mineralocorticoids (e.g., fludrocortisone) for patients with orthostatic hypotension or adrenocortical insufficiency is allowed.
* History or risk of autoimmune disease, including, but not limited to, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, vascular thrombosis associated with antiphospholipid syndrome, Wegener's granulomatosis, Sjogren's syndrome, Guillain-Barré syndrome, multiple sclerosis, vasculitis, or glomerulonephritis.
* Patients with a history of autoimmune hypothyroidism who are asymptomatic and/or are on a stable dose of thyroid replacement hormone are eligible.
* Patients with controlled Type 1 diabetes mellitus on a stable insulin regimen are eligible.
* Patients with eczema, psoriasis, lichen simplex chronicus, or vitiligo with dermatologic manifestations only (e.g., patients with psoriatic arthritis would be excluded) are permitted provided that they meet the following conditions:
* Patients with psoriasis must have a baseline ophthalmologic exam to rule out ocular manifestations
* Rash must cover less than 10% of body surface area (BSA)
* Disease is well controlled at baseline and only requiring low potency topical steroids (e.g., hydrocortisone 2.5%, hydrocortisone butyrate 0.1%, flucinolone 0.01%, desonide 0.05%, aclometasone dipropionate 0.05%)
* No acute exacerbations of underlying condition within the last 12 months (not requiring psoralen plus ultraviolet A radiation \[PUVA\], methotrexate, retinoids, biologic agents, oral calcineurin inhibitors; high potency or oral steroids)
* PHASE III: Grade 3-4 electrolyte abnormalities (CTCAE, v. 4) within 14 days prior to registration:
* Serum calcium (ionized or adjusted for albumin) \< 7 mg/dL (1.75 mmol/L) or \> 12.5 mg/dL (\> 3.1 mmol/L) despite intervention to normalize levels;
* Glucose \< 40 mg/dL (\< 2.2 mmol/L) or \> 250 mg/dL (\> 14 mmol/L);
* Magnesium \< 0.9 mg/dL (\< 0.4 mmol/L) or \> 3 mg/dL (\> 1.23 mmol/L) despite intervention to normalize levels;
* Potassium \< 3.0 mmol/L or \> 6 mmol/L despite intervention to normalize levels;
* Sodium \< 130 mmol/L or \> 155 mmol/L despite intervention to normalize levels.
* PHASE III: Pregnancy or women of childbearing potential and men who are sexually active and not willing/able to use medically acceptable forms of contraception for up to 5 months from last study treatment; this exclusion is necessary because the treatment involved in this study may be significantly teratogenic. Women who are breastfeeding and unwilling to discontinue are also excluded
* PHASE III: History of severe allergic, anaphylactic, or other hypersensitivity reactions to chimeric or humanized antibodies or fusion proteins
* PHASE III: Patients taking bisphosphonate therapy for symptomatic hypercalcemia. Use of bisphosphonate therapy for other non-oncologic reasons (e.g., osteoporosis) is allowed
* PHASE III: Patients requiring treatment with a RANKL inhibitor (e.g. denosumab) for non-oncologic reasons who cannot discontinue it before registration
* PHASE III: Patients with known distant metastatic disease are excluded
* PHASE III: Known hypersensitivity to Chinese hamster ovary cell products or other recombinant human antibodies
* PHASE III: Major surgical procedure within 28 days prior to registration or anticipation of need for a major surgical procedure during the course of the study
* PHASE III: Administration of a live, attenuated vaccine within 4 weeks prior to registration or anticipation that such a live, attenuated vaccine will be required during the study and for patients receiving atezolizumab, up to 5 months after the last dose of atezolizumab.
* Influenza vaccination should be given during influenza season only (approximately October to DRUG: Atezolizumab, PROCEDURE: Biopsy Procedure, PROCEDURE: Biospecimen Collection, BIOLOGICAL: Cetuximab, DRUG: Cisplatin, PROCEDURE: Computed Tomography, DRUG: Docetaxel, RADIATION: Intensity-Modulated Radiation Therapy, PROCEDURE: Magnetic Resonance Imaging, OTHER: Survey Administration
Oropharyngeal p16INK4a-Negative Squamous Cell Carcinoma, Stage III Hypopharyngeal Squamous Cell Carcinoma AJCC v7, Stage III Laryngeal Squamous Cell Carcinoma AJCC v6 and v7, Stage III Oral Cavity Squamous Cell Carcinoma AJCC v6 and v7, Stage III Oropharyngeal Squamous Cell Carcinoma AJCC v7, Stage IV Hypopharyngeal Squamous Cell Carcinoma AJCC v7, Stage IV Laryngeal Squamous Cell Carcinoma AJCC v7, Stage IV Oral Cavity Squamous Cell Carcinoma AJCC v6 and v7, Stage IV Oropharyngeal Squamous Cell Carcinoma AJCC v7
UT Southwestern; Parkland Health & Hospital System
Morphea in Adults and Children (MAC) Cohort Study: A Morphea Registry and DNA Repository (MAC)
The Morphea in Adults and Children (MAC) cohort is the first registry for both children and adults with morphea (also known as localized scleroderma) in the country. The purpose of the registry is to learn more about morphea, specifically: * How morphea behaves over time * How frequently specific problems occur along with morphea (for example, arthritis) * Whether morphea has an autoimmune background
Call 214-648-5005
studyfinder@utsouthwestern.edu, Aleuna.Lee@UTSouthwestern.edu
Heidi Jacobe
ALL
up to 90 Years old
N/A
NCT01808937
STU 112010-028
Inclusion Criteria:
• Patient must have a clinical diagnosis of morphea confirmed by the primary investigator and by histopathological examination.
• Ages 0-90 years old
• Children must weigh more than 20 lbs. in order to satisfy Children's Medical Center policy for the maximum amount of blood drawn in a 24 hour period.
• Patient or legal guardian must be able to speak and read at a 6th grade reading level.
• Both male and female patients will be eligible
• All races and ethnic backgrounds will be included
• Relationships to proband: All patients with morphea will be included. A patient's family history will be reviewed and if there is a family history of morphea or systemic sclerosis then we will give the study patient the investigator's contact information and ask the family member to call the study team to answer any questions and enroll them in the study if they choose to do so.
• Ability to give informed consent: Patients must be able to give informed consent or they will give assent with parent or guardian consent as a minor to be a part of the morphea registry.
Exclusion Criteria:
• Patients who have been coded as morphea (701.0), but do not have morphea/localized scleroderma (examples: steroid atrophy, acquired keratoderma, keloids, nephrogenic fibrosing dermopathy, systemic sclerosis, lichen sclerosis)
OTHER: Morphea
Scleroderma, Localized, Morphea, Frontal Linear Scleroderma en Coup de Sabre, Scleroderma, Circumscribed, Scleroderma, Linear, Other Skin
Children’s Health
Maximum Tolerated Dose, Safety, and Efficacy of Rhenium Nanoliposomes in Recurrent Glioma (ReSPECT)
This is a multi-center, sequential cohort, open-label, volume and dose escalation study of the safety, tolerability, and distribution of 186RNL given by convection enhanced delivery to patients with recurrent or progressive malignant glioma after standard surgical, radiation, and/or chemotherapy treatment. The study uses a modified Fibonacci dose escalation, followed by an expansion at the maximum tolerated dose (MTD) to determine efficacy. The starting absorbed dose is 1mCi in a volume of 0.660mL.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Toral Patel
ALL
18 Years and over
PHASE1
NCT01906385
STU-2020-0096
Inclusion Criteria:
• At least 18 years of age.
• Ability to understand the purposes and risks of the study and has signed a written informed consent form approved by the investigator's IRB/Ethics Committee.
• Histologically confirmed Grade III/IV recurrent Glioma (following 2021 WHO CNS5 glioma nomenclature, e.g., Astrocytoma, IDH-mutant grade 3 or 4; Glioblastoma, IDH-wildtype grade 4).
• Progression by RANO criteria or other clinically accepted neurooncology evaluation, following standard treatment options with known survival benefit for any recurrence (e.g., surgery, temozolomide, radiation, and tumor treating fields). Patient may be included in study if medically unable or unwilling to follow standard treatment options for any recurrence.
• Patients who receive treatment with antiepileptic medications must have a two-week history of stable dose of antiepileptic without seizures prior to study start (dosing).
• Patients with corticosteroid requirements to control cerebral edema must be maintained at a stable or decreasing dose for a minimum of two weeks without progression of clinical symptoms prior to study start (dosing).
• Patients with Grade III/IV Glioma (following 2021 WHO CNS5 glioma nomenclature, e.g., Astrocytoma, IDH-mutant grade 3 or 4; Glioblastoma, IDH-wildtype grade 4) which falls within the treatment field volume.
• ECOG performance status of 0 to 2; Karnofsky Performance Status ≥ 60.
• Life expectancy of at least 2 months.
• Acceptable liver function:
• Bilirubin ≤ 1.5 times upper limit of normal
• AST (SGOT) and ALT (SGPT) ≤ 3.0 times upper limit of normal (ULN)
• Acceptable renal function: a. Serum creatinine ≤1.5xULN
• Acceptable hematologic status (without hematologic support):
• ANC ≥1000 cells/uL
• Platelet count ≥100,000/uL
• Hemoglobin ≥9.0 g/dL
• All women of childbearing potential must have a negative serum pregnancy test and male and female subjects must agree to use effective means of contraception (for example, surgical sterilization or the use of barrier contraception with either a condom or diaphragm in conjunction with spermicidal gel or an IUD) with their partner from entry into the study through 6 months after the last dose.
Exclusion Criteria:
• The subject has evidence of acute intracranial or intratumoral hemorrhage either by magnetic resonance imaging (MRI) or computerized tomography (CT) scan. Subjects with resolving hemorrhage changes, punctate hemorrhage, or hemosiderin are eligible.
• The subject is unable or contraindicated to undergo MRI scan (e.g., has pacemaker or medically unstable).
• The subject has not recovered to CTCAE v4.0 Grade ≤1 from AEs (except alopecia, anemia, and lymphopenia) due to antineoplastic agents, investigational drugs, or other medications that were administered prior to study.
• The subject is pregnant or breast-feeding.
• The subject has serious intercurrent illness, as determined by the treating physician, which would compromise either patient safety or study outcomes such as: * hypertension (two or more blood pressure readings performed at screening of \>150 mmHg systolic or \>100 mmHg diastolic) despite optimal treatment * active medically significant infection unresponsive to antibiotics (e.g., non- healing wound, ulcer), uncontrolled systemic infection, or bone fracture * clinically significant cardiac arrhythmias not controlled by appropriate medications * untreated hypothyroidism * symptomatic congestive heart failure or unstable angina pectoris within 3 months prior to study drug * myocardial infarction, stroke, or transient ischemic attack within 6 months prior to study drug * known active malignancy (other than glioma) except non-melanoma skin cancer or carcinoma in-situ in the cervix unless PI determines it would not impact patient safety or efficacy determinations
• The subject has inherited bleeding diathesis or coagulopathy with the risk of bleeding.
• The subject has received any of the following prior anticancer therapy: * Prior treatment with Bevacizumab * Non-standard radiation therapy such as brachytherapy, systemic radioisotope therapy, or intra-operative radiotherapy (IORT) to the target site * Radiation therapy within 12 weeks of screening * Systemic therapy (including investigational agents and small-molecule kinase inhibitors) or non-cytotoxic hormonal therapy (e.g., tamoxifen) within 14 days or 5 half-lives, whichever is shorter, prior to study start (dosing) * Biologic agents (antibodies, immune modulators, vaccines, cytokines) within 21 days prior to study start (dosing) * Nitrosoureas or mitomycin C within 42 days, or metronomic/protracted low- dose chemotherapy within 14 days, or other cytotoxic chemotherapy within 28 days, prior to study start (dosing) * Prior treatment with carmustine wafers * Patients who are currently receiving any other investigational agents and/or who have received an investigational agent in 28 days prior to study start (dosing)
• Multifocal progression or involvement of the leptomeninges.
• Psychiatric illness/social situations that would limit compliance with the study requirements
• Infratentorial disease
• The subject has a tumor located within 1-2 cm of a ventricle AND it is determined by the surgeon, PI, and sponsor to be a risk for drug extravasation to the subarachnoid space if given catheter placement and drug administration.
• Phase 2 only: The subject should have a tumor volume of ≤20 cm3 to be included in the Phase 2 portion of the study. Subjects with tumor volumes of greater than 20 cm3 are excluded from the Phase 2 portion of the study.
DRUG: Rhenium Liposome Treatment
Glioma, Brain and Nervous System
Glioma, Brain Tumor, Radiotherapy, Glioblastoma, Recurrent Glioblastoma, Rhenium, Rhenium Nanoliposome, Brain Cancer, GBM, High Grade Glioma, Glioblastoma Multiform, Grade IV Astrocytoma
UT Southwestern
Diaphragmatic Hernia Research & Exploration, Advancing Molecular Science (DHREAMS)
The goal of this study is to identify genes that convey susceptibility to congenital diaphragmatic hernia in humans. The identification of such genes, and examination of their structure and function, will enable a delineation of molecular pathogenesis and, ultimately, prevention or treatment of congenital diaphragmatic hernia. There are many different possible modes of inheritance for congenital anomalies, including autosomal dominant, autosomal recessive, and multifactorial. Multi-factorial inheritance is responsible for many common medical disorders, including hypertension, myocardial infarction, diabetes and cancer. This type of inheritance pattern appears to involve environmental factors as well as a combination of genetic variations that together can predispose to or produce congenital anomalies, such as congenital diaphragmatic hernia. Our study is designed to establish a small, well-defined genetic resource consisting of 1) Nuclear families suitable for linkage analysis by parametric,non-parametric (e.g. sib pairs, TDT) and association techniques, 2) Individuals with congenital diaphragmatic hernia who can be directly screened for allelic variation in candidate genes, and 3) Individuals who can serve as controls (are unaffected by congenital diaphragmatic hernia). Neonates and their families will be collected from homogenous and heterogeneous populations. By characterizing diverse populations, it should be possible to increase the likelihood of demonstration of genetic variation in selected candidate genes that can then be used in association and linkage studies in individual subjects with congenital diaphragmatic hernia.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Simi.Pottoore@Childrens.com
Lauren Gillory
ALL
Not specified
N/A
NCT00950118
STU-2021-1094
Inclusion Criteria:
* All individuals affected with a congenital diaphragmatic hernia (CDH), or with a family history of a CDH
Exclusion Criteria:
* Individuals with no personal history of a CDH or family history of a family member affected with congenital diaphragmatic hernia Congenital Diaphragmatic Hernia
Congenital Diaphragmatic Hernia (CDH), Genes, Genetic, Genetic testing, exome sequencing, genome sequencing, RNAseq
Children’s Health
Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).
The Increlex® Global Registry is a descriptive, multicenter, observational, prospective, open-ended, non interventional, post-authorisation surveillance registry. The main purpose of this global registry is to collect, analyse and report safety data during and up to at least 5 years after the end of treatment in children and adolescents receiving Increlex® therapy for SPIGFD according to the locally approved product information.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Michelle.Murphy@UTSouthwestern.edu
Melissa Ham
ALL
2 Years to 18 Years old
NCT00903110
STU-2021-1124
Inclusion Criteria:
* For US : patients starting or planning to start or currently receiving treatment with Increlex® therapy for severe primary IGF-1 deficiency as defined by the US Increlex® prescribing information or for growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH.For EU : patients starting or planning to start or currently receiving treatment with Increlex® therapy according to the locally approved product information.
* Parents or legally authorized representatives if applicable must give signed informed consent before any registry-related activities are conducted. Assent from the subject should also be obtained as appropriate
Exclusion Criteria:
* Subject currently participating in an Increlex® clinical trial
* Subject currently participating in any clinical trial for growth retardation
* Patient with any contraindication to Increlex® or any condition subject to special warning as per the locally approved label
* For US patients, these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia and patients with closed epiphyses.
* For EU patients: these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia or any condition or medical history which increases the risk of benign or malignant neoplasia and patients with closed epiphyses DRUG: Increlex®
IGF1 Deficiency
Children’s Health
Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea
This study is a prospective, single-arm study conducted under a common implant and follow-up protocol. The objective will be to follow fifty-seven (57) adolescents and young adults (10-21 years of age), with Down syndrome, moderate to severe sleep apnea, and post-adenotonsillectomy, for 12 months after undergoing implant of the Inspire Upper Airway Stimulation (UAS) System. The study is being conducted in order to evaluate objective change in cognition and expressive language after implant and therapy with the Inspire UAS System.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Francesca.Chambers@UTSouthwestern.edu
Ron Mitchell
All
10 Years to 21 Years old
Phase 2
NCT04801771
STU-2021-0286
Inclusion Criteria:
• Diagnosis of Down syndrome
• Age 10-21 years
• Prior adenotonsillectomy
• Severe OSA (AHI > 10, AHI < 50, no more than 25% AHI attributable to central events) based on prior in-lab PSG performed after adenotonsillectomy and within 18 months of enrollment
• Approval from at least two of the three physician reviewers based upon the results of a routine drug-induced sleep endoscopy (DISE) having occurred within 12 months of enrollment
• Subjects must have either tracheotomy or be ineffectively treated with CPAP due to non-compliance, discomfort, un-desirable side effects, persistent symptoms despite compliance use, or refusal to use the device
• Children and their parents/guardians must be willing to have stimulation hardware permanently implanted, and be willing to participate in follow-up visits, postoperative PSG, and questionnaire completion
• Children's parents/guardians must complete a questionnaire confirming that their child is capable of communicating feelings of pain or discomfort. They must also confirm they are able to assess their child for adverse effects related to device implantation
• Children and their parents/guardians must be proficient in English
Exclusion Criteria:
• Body mass index (BMI) above the 95th percentile for subject's age
• Circumferential airway collapse at the level of the velopharynx observed during DISE
• Other medical conditions resulting in medical instability (eg. congestive heart failure, recent open heart surgery, immunosuppression, or chronic lung disease or aspiration)
• Presence of another medical condition requiring future magnetic resonance imaging (MRI) of the chest
• Patients with another implantable device which could interact unintentionally with the Inspire system
• Any contraindication for general anesthesia
• History of bleeding or clotting disorders and those on blood thinning or NSAID medications for the week prior to implantation surgery. Subjects will be asked to refrain from the use of NSAIDS for two weeks after implantation or any revision surgeries
• Subject is currently taking muscle relaxant medication
• Life expectancy less than 12 months
• Subject's inability to communicate pain or discomfort to their caretaker/parent, based on parental or investigator assessment
• Nonverbal candidates will be excluded due to an inability to complete testing procedures including expressive language sampling
• Subjects with a co-occurring diagnosis of autism spectrum disorder
• Subjects that have a positive β-HCG
• Subjects deemed unfit for participation by the investigator for any other reason
Device: Inspire Upper Airway Stimulation (UAS) System
Down Syndrome, Obstructive Sleep Apnea, Ear, Nose, Throat
Hypoglossal nerve stimulation
Children’s Health
COMPASSION S3 Post-Approval Study
This study will monitor device performance and outcomes of the SAPIEN 3 Transcatheter Heart Valve (THV) System in subjects with a dysfunctional right ventricular outflow tract (RVOT) conduit or previously implanted surgical valve in the pulmonic position with a clinical indication for intervention.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kirstie.LeDoux@UTSouthwestern.edu
Thomas Zellers
All
Not specified
NCT04860765
STU-2021-0535
Inclusion Criteria:
• Dysfunctional RVOT conduit or previously implanted surgical valve
• RVOT/PV with ≥ moderate regurgitation and/or a mean RVOT/PV gradient of ≥ 35 mmHg
Exclusion Criteria:
• Inability to tolerate an anticoagulation/antiplatelet regimen
• Active bacterial endocarditis or other active infections
Device: SAPIEN 3 THV
Complex Congenital Heart Defect, Dysfunctional RVOT Conduit, Pulmonary Valve Insufficiency, Pulmonary Valve Degeneration, Pulmonary Valve, Obstruction, Cardiovascular
Transcatheter pulmonary valve replacement, Transcatheter pulmonary valve implantation, SAPIEN 3
Children’s Health
A Study of ISIS 678354 Administered to Participants With Severe Hypertriglyceridemia
The purpose of the study is to evaluate the efficacy of ISIS 678354 as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Lakeisha.Cade@UTSouthwestern.edu
Zahid Ahmad
All
18 Years and over
Phase 3
NCT05079919
STU-2021-0926
Key
• Fasting TG ≥ 500 mg/dL (5.65 mmol/L) at Screening and Qualification
• Patients should be on standard of care lipid-lowering medications per local guidelines unless intolerant. Lipid-lowering medications should be optimized and stabilized for at least 4 weeks prior to Screening to minimize changes in these medications during the study. Key
• Hemoglobin A1c (HbA1c) ≥ 9.5% at Screening
• Platelet count < 100K/cubic millimeters at Screening or Qualification
• Alanine aminotransferase or aspartate aminotransferase > 3.0 × upper limit of normal
• Total bilirubin > upper limit of normal unless due to Gilbert's syndrome
• Estimated GFR < 40 mL/min/1.73 m^2
Inclusion Criteria:
• Fasting TG ≥ 500 mg/dL (5.65 mmol/L) at Screening and Qualification
• Patients should be on standard of care lipid-lowering medications per local guidelines unless intolerant. Lipid-lowering medications should be optimized and stabilized for at least 4 weeks prior to Screening to minimize changes in these medications during the study. Key
Exclusion Criteria:
• Hemoglobin A1c (HbA1c) ≥ 9.5% at Screening
• Platelet count < 100K/cubic millimeters at Screening or Qualification
• Alanine aminotransferase or aspartate aminotransferase > 3.0 × upper limit of normal
• Total bilirubin > upper limit of normal unless due to Gilbert's syndrome
• Estimated GFR < 40 mL/min/1.73 m^2
Drug: ISIS 678354, Drug: Placebo
Severe Hypertriglyceridemia
ISIS 678354, Fasting Triglycerides, Apolipoprotein C-III, Very Low-Density Lipoprotein Cholesterol, High-Density Lipoprotein-Cholesterol, Non-High-Density Lipoprotein-Cholesterol
UT Southwestern
A Study to Test if Fremanezumab is Effective in Preventing Migraine in Children and Adolescents
The primary objective of the study is to evaluate the long-term safety and tolerability of subcutaneous fremanezumab in the preventive treatment of migraine in pediatric participants 6 to 17 years of age (inclusive at enrollment in the pivotal study). Secondary objectives are to evaluate the efficacy of subcutaneous fremanezumab in pediatric participants with migraine and to evaluate the immunogenicity of fremanezumab and the impact of ADAs on clinical outcomes in pediatric participants exposed to fremanezumab. The total duration of the study is planned to be up to 60 months.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kiley.Poppino@UTSouthwestern.edu
Deryk Walsh
All
6 Years to 17 Years old
Phase 3
NCT04530110
STU-2020-1130
Inclusion Criteria:
Inclusion Criteria for Participants Rolling Over from the Pivotal Efficacy Studies
(TV48125-CNS-30082 or TV48125-CNS-30083):
• Participants have completed the pivotal efficacy study and, in the opinion of the Investigator or the Sponsor, are able to complete the study in a safe and compliant way.
• Participants may continue with a stable dose/regimen of the preventive medication they were taking during the pivotal efficacy studies.
• The participant continues to meet appropriate criteria carried forward from the pivotal efficacy study/
• The participant has received all recommended age-appropriate vaccines according to local standard of care and schedule.
• The participant weighs at least 17.0 kg on the day of study enrollment. NOTE: Additional criteria apply; please contact the investigator for more information. Inclusion Criteria for Participants Rolling Over from the Phase 1 Pediatric Pharmacokinetic Study (Study TV48125-CNS-10141):
• The participant/caregiver has demonstrated compliance with the electronic headache diary during the 28-day baseline period by entry of headache data on a minimum of 21 out of 28 days (approximately 75% diary compliance).
• The participant has received all recommended age-appropriate vaccines according to local standard of care and schedule.
• The participant weighs at least 17.0 kg on the day of study enrollment.
• The participant has a body mass index ranging from the 5th to 120% of the 95th percentile, inclusive, on the day of study enrollment.
• Not using preventive medications or using no more than 2 preventive medications for migraine or other medical condition, as long as the dose and regimen have been stable for at least 2 months prior to screening (visit 1). NOTE: Additional criteria apply; please contact the investigator for more information. Inclusion Criteria for Participants Rolling Over from the Pivotal Efficacy Studies (TV48125-CNS-30082 and TV48125-CNS-30083) for Safety and antidrug antibody (ADA) Assessment Only: • Participants may be included in this study if they sign and date the informed consent document or upon consent of a parent or guardian, if the participant is younger than the age of consent, accompanied by assent of the participant.
Exclusion Criteria:
Exclusion Criteria for Participants Rolling Over from the Pivotal Efficacy Studies
(TV48125-CNS-30082 or TV48125-CNS-30083):
• In the judgment of the investigator, the participant has a clinically significant abnormal finding on study entry, including hematology, blood chemistry, coagulation tests, or urinalysis values/findings (abnormal tests may be repeated for confirmation).
• The participant has a current history of a clinically significant psychiatric condition, any prior history of a suicide attempt, or a history of suicidal ideation with a specific plan within the past 2 years, at the discretion of the investigator.
• The participant has an ongoing infection or a known history of human immunodeficiency virus infection, tuberculosis, Lyme disease, or chronic hepatitis B or C, or a known active infection of coronavirus disease 2019 (COVID-19).
• The participant has a history of hypersensitivity reactions to injected proteins, including mAbs, or a history of Stevens-Johnson Syndrome or toxic epidermal necrolysis syndrome, or the participant is concomitantly using lamotrigine.
• The participant received a live attenuated vaccine (eg, intranasal flu vaccine, and measles, mumps, and rubella vaccine) within the 12-week period prior to screening. Note: If a medical need arises during the study, the participant may receive a live attenuated vaccine.
• The participant is pregnant or nursing.
• In the judgment of the investigator, the participant has an abnormal finding on the baseline 12-lead ECG considered clinically significant.
• The patient has a current or past medical history of hemiplegic migraine. NOTE: Additional criteria apply; please contact the investigator for more information. Exclusion Criteria for Participants Rolling Over from the Phase 1 Pharmacokinetic Study (TV48125-CNS-10141):
• The participant has any clinically significant cardiovascular (including congenital cardiac anomalies or thromboembolic events), endocrine, gastrointestinal, genitourinary, hematologic, hepatic, immunologic, neurologic, ophthalmic, pulmonary, renal disease, or complications of an infection, at the discretion of the investigator.
• The participant has a current history of a clinically significant psychiatric condition, any prior history of a suicide attempt, or a history of suicidal ideation with a specific plan within the past 2 years, at the discretion of the investigator.
• The participant has an ongoing infection or a known history of human immunodeficiency virus infection, tuberculosis, Lyme disease, or chronic hepatitis B or C, or a known active infection of coronavirus disease 2019 (COVID-19).
• The participant has a history of hypersensitivity reactions to injected proteins, including mAbs, or a history of Stevens-Johnson Syndrome or toxic epidermal necrolysis syndrome, or the participant is concomitantly using lamotrigine.
• The participant received a live attenuated vaccine (eg, intranasal flu vaccine, and measles, mumps, and rubella vaccine) within the 12-week period prior to screening. Note: If a medical need arises during the study, the participant may receive a live attenuated vaccine.
• The participant is pregnant or nursing.
• In the judgment of the investigator, the participant has an abnormal finding on the baseline 12-lead ECG considered clinically significant.
• The patient has a current or past medical history of hemiplegic migraine. NOTE: Additional criteria apply; please contact the investigator for more information. Exclusion Criteria for Participants Rolling Over from the Pivotal Efficacy Studies (TV48125-CNS-30082 and TV48125-CNS-30083) for Safety and antidrug antibody (ADA) Assessment Only: Not Applicable
Drug: Fremanezumab
Migraine
episodic migraine, chronic migraine
Children’s Health
A Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency
This is a phase 3 open-label multicenter extension study designed to evaluate the long-term safety and efficacy of Lonapegsomatropin administered once-weekly. The study participants are adults (males and females) with confirmed growth hormone deficiency (GHD) having completed the treatment period in study TCH-306 (foresiGHt).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Oksana.Hamidi@UTSouthwestern.edu
Oksana Hamidi
All
23 Years to 81 Years old
Phase 3
NCT05171855
STU-2022-0087
Inclusion Criteria:
• Signing of the trial specific informed consent
• Completion of the treatment period and Visit 7 assessments of trial TCH-306, including collection and upload of Visit 7 DXA scan
• Fundoscopy at Visit 7 in trial TCH-306 without signs/symptoms of intracranial hypertension or diabetic retinopathy stage 2 / moderate or above
Exclusion Criteria:
• Diabetes mellitus if any of the following are met:
• Poorly controlled diabetes, defined as HbA1C higher than 7.5% according to central laboratory at Visit 6 in trial TCH-306
• Use of diabetes mellitus drugs other than metformin and/or dipeptidyl peptidase-4 (DPP-4) inhibitors
• Active malignant disease or history of malignancy. Exceptions are:
• Resection of in situ carcinoma of the cervix uteri
• Complete eradication of squamous cell or basal cell carcinoma of the skin
• Known history of hypersensitivity and/or idiosyncrasy to the investigational product (somatropin or excipients)
• Female who is pregnant, plans to become pregnant, or is breastfeeding
• Female participant of childbearing potential (i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile) not willing throughout the trial to use contraceptives as required by local law or practice. Details included in Appendix 4/section 10.4 of the protocol
• Male participant not willing throughout the trial to use contraceptives as required by local law or practice. Details included in Appendix 4/ section 10.4 of the protocol
• Any disease or condition that, in the judgement of the investigator, may make the participant unlikely to comply with the requirements of the protocol or any condition that presents undue risk from the investigational product or trial procedures
Drug: Lonapegsomatropin
Adult Growth Hormone Deficiency, Endocrine System Diseases, Hormone Deficiency, Other Endocrine System
Human Growth Hormone, hGH, rhGH, GHD, Adult Growth Hormone Deficiency, Long Acting Growth Hormone, Lonapegsomatropin, Prodrug, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon hGH, Skytrofa
UT Southwestern
Study to Assess the Effect of Ofatumumab in Treatment Naïve, Very Early RRMS Patients Benchmarked Against Healthy Controls. (AGNOS)
This study will evaluate the impact of ofatumumab in Relapsing Remitting Multiple Sclerosis (RRMS) participants that are very early in the course of their disease using clinical and magnetic resonance imaging (MRI) outcomes. The study will also assess changes in disease using monitoring techniques including digital biometric device use, biomarker analysis and non-conventional MRI. Select outcomes in the ofatumumab treated group will be compared to a group of Healthy participants to determine if there are similarities between the groups after the patients with MS undergo treatment with ofatumumab.
Call 214-648-5005
studyfinder@utsouthwestern.edu, mahi.patel@utsouthwestern.edu
Darin Okuda
All
18 Years to 35 Years old
Phase 4
NCT05084638
STU-2021-1189
Key
• Signed informed consent must be obtained prior to participation in the study
• Age 18-35 years Patients in the healthy control arm eligible for inclusion must fulfill the following criteria:
• Able to obtain MRI (HC with abnormal MRI at Screening will be excluded) and use wearable device
• Able to provide blood sample (no CSF will be collected in HC) Patients in the ofatumumab-treated arm eligible for inclusion must fulfill the following criteria:
• Diagnosis of RRMS per McDonald Criteria (2010/2017)
• Within 6 months of diagnosis of clinically definite MS (CDMS)
• EDSS 0-3.0 (Inclusive)
• Treatment-naïve to MS DMT
• Able to obtain MRI and attend study visits at sites
• Able to use wearable device
• Able to provide blood sample (and CSF for sub-group n=15) Key
• Confounding medical condition as determined by the investigator RRMS patients fulfilling any of the following exclusion criteria are not eligible for inclusion in this study:
• Diseases other than multiple sclerosis responsible for the clinical or MRI presentation
• Patients with neuromyelitis optica, Radiologic/ Clinically Isolated Syndrome, Secondary Progressive or Primary Progressive MS diagnosis
• Use of experimental or investigational drugs for MS
• Previous use of Disease Modifying Therapy (DMT) or chemotherapeutic medications for MS
• Relapse between screening and Baseline visits
• Known sensitivity to gadolinium; patients with chronic, severe kidney disease
• Known history of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes
• CNS anomalies that are better accounted for by another disease process or MRI anomalies causing clinically apparent impairments
• Known active malignancies
• Pregnant or nursing (lactating) women
• Females of childbearing potential (all women physiologically capable of becoming pregnant) should use effective contraception while receiving ofatumumab and for 6 months after the last treatment of ofatumumab
• Patients with an active chronic disease (or stable but treated with immune therapy) of the immune system other than MS or with immunodeficiency syndrome
• Patients with active infections including systemic bacterial, viral (including SARS-CoV-2/COVID-19) or fungal infections, or known to have AIDS or to test positive for HIV antibody at Screening
• Patients with neurological findings consistent with Progressive Multifocal Leukoencephalopathy (PML), or confirmed PML
• Patients with IgG or IgM levels below LLN at Screening
• Patients that have received any live or live-attenuated vaccines within 4 weeks prior to first dose of study drug administration
• Patients at risk of developing or having reactivation of hepatitis
Inclusion Criteria:
Participants eligible for inclusion in this study must meet all of the following criteria:
• Signed informed consent must be obtained prior to participation in the study
• Age 18-35 years Patients in the healthy control arm eligible for inclusion must fulfill the following criteria:
• Able to obtain MRI (HC with abnormal MRI at Screening will be excluded) and use wearable device
• Able to provide blood sample (no CSF will be collected in HC) Patients in the ofatumumab-treated arm eligible for inclusion must fulfill the following criteria:
• Diagnosis of RRMS per McDonald Criteria (2010/2017)
• Within 6 months of diagnosis of clinically definite MS (CDMS)
• EDSS 0-3.0 (Inclusive)
• Treatment-naïve to MS DMT
• Able to obtain MRI and attend study visits at sites
• Able to use wearable device
• Able to provide blood sample (and CSF for sub-group n=15) Key
Exclusion Criteria:
Participants in the healthy control arm meeting any of the following criteria are not
eligible for inclusion in this study:
• Confounding medical condition as determined by the investigator RRMS patients fulfilling any of the following exclusion criteria are not eligible for inclusion in this study:
• Diseases other than multiple sclerosis responsible for the clinical or MRI presentation
• Patients with neuromyelitis optica, Radiologic/ Clinically Isolated Syndrome, Secondary Progressive or Primary Progressive MS diagnosis
• Use of experimental or investigational drugs for MS
• Previous use of Disease Modifying Therapy (DMT) or chemotherapeutic medications for MS
• Relapse between screening and Baseline visits
• Known sensitivity to gadolinium; patients with chronic, severe kidney disease
• Known history of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes
• CNS anomalies that are better accounted for by another disease process or MRI anomalies causing clinically apparent impairments
• Known active malignancies
• Pregnant or nursing (lactating) women
• Females of childbearing potential (all women physiologically capable of becoming pregnant) should use effective contraception while receiving ofatumumab and for 6 months after the last treatment of ofatumumab
• Patients with an active chronic disease (or stable but treated with immune therapy) of the immune system other than MS or with immunodeficiency syndrome
• Patients with active infections including systemic bacterial, viral (including SARS-CoV-2/COVID-19) or fungal infections, or known to have AIDS or to test positive for HIV antibody at Screening
• Patients with neurological findings consistent with Progressive Multifocal Leukoencephalopathy (PML), or confirmed PML
• Patients with IgG or IgM levels below LLN at Screening
• Patients that have received any live or live-attenuated vaccines within 4 weeks prior to first dose of study drug administration
• Patients at risk of developing or having reactivation of hepatitis
Drug: Ofatumumab
Relapse Remitting Multiple Sclerosis, Brain and Nervous System
early relapsing multiple sclerosis, ofatumumab, healthy control, treatment naïve, young adult population, MS-related disability, biomarker, MRI
UT Southwestern
Prospective Treatment Efficacy in IPF Using Genotype for Nac Selection (PRECISIONS) Trial (PRECISIONS)
The purpose of this study is to compare the effect of n-acetylcysteine (NAC) plus standard care with matched placebo plus standard of care in patients diagnosed with idiopathic pulmonary fibrosis (IPF) who have the TOLLIP rs3750920 TT genotype. The study will compare the time to a composite endpoint of relative decline in lung function [10% relative decline in forced vital capacity (FVC), first respiratory hospitalization, lung transplantation, or all-cause mortality] The secondary objectives will be to examine the effect of NAC on the components of the primary composite endpoint, the rates of clinical events, change in physiology, change in health status, and change in respiratory symptoms.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Uma.Gude@UTSouthwestern.edu
Chad Newton
All
40 Years and over
Phase 3
NCT04300920
STU-2021-0623
Inclusion Criteria:
• ≥ 40 years of age
• Diagnosed with IPF according to 2018 ATS/ERS/JRS/ALAT, confirmed by enrolling investigator
• Signed informed consent
• If taking pirfenidone or nintedanib, must be on stable dose for at least 6 weeks prior to enrollment visit
• Confirmed rs3570920 TT TOLLIP genotype
Exclusion Criteria:
• Pregnancy or planning to become pregnant
• Women of childbearing potential not willing to remain abstinent (refrain from heterosexual intercourse) or use two adequate methods of contraception, including at least one method with a failure rate of <1% per year during study participation
• Significant medical, surgical or psychiatric illness that in the opinion of the investigator would affect subject safety, including liver and renal failure
• Receipt of an investigational drug or biological agent within the previous 4 weeks of the screening visit or 5 times the half-life, if longer
• Supplemental or prescribed NAC therapy within 60 days of enrollment
• Listed for lung transplantation at the time of screening
• History of lung cancer
• Inability to perform spirometry
• Forced vital capacity (FVC) less than 45% predicted, using the global lung function index (GLI) equation at Visit 1
• Active respiratory infection requiring treatment with antibiotics within 4 weeks of Visit 1
Drug: N-acetyl cysteine, Drug: Placebo
Idiopathic Pulmonary Fibrosis
IPF, Pulmonary Fibrosis, n-acetylcysteine, NAC
UT Southwestern
A Study to Compare Teclistamab in Combination With Daratumumab and Lenalidomide (Tec-DR) in Participants With Newly Diagnosed Multiple Myeloma (MajesTEC-7)
The purpose of this study is to compare the efficacy of teclistamab in combination with daratumumab and lenalidomide (Tec-DR) versus daratumumab, lenalidomide, dexamethasone (DRd).
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Aimaz Afrough
All
18 Years and over
Phase 3
NCT05552222
STU20250678
Inclusion Criteria:
• Have a diagnosis of multiple myeloma according to the International Myeloma Working Group (IMWG) diagnostic criteria
• Be newly diagnosed and not considered a candidate for high-dose chemotherapy with autologous stem cell transplant (ASCT) due to: ineligible due to advanced age OR; ineligible due to the presence of comorbid condition(s) likely to have a negative impact on tolerability of high-dose chemotherapy with ASCT OR; deferral of high-dose chemotherapy with ASCT as initial treatment
• Have an Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2
• A female participant must agree not to be pregnant, breastfeeding, or planning to become pregnant while enrolled in this study or within 3 months after the last dose of study treatment
• A male participant must agree not to plan to father a child while enrolled in this study or within 3 months after the last dose of study treatment
Exclusion Criteria:
• Received a cumulative dose of systemic corticosteroids equivalent to greater than or equal to (>=) 20 milligrams (mg) of dexamethasone within 14 days before randomization
• Had plasmapheresis within 28 days of randomization
• Had a stroke, transient ischemic attack, or seizure within 6 months prior to randomization
• Known allergies, hypersensitivity, or intolerance to teclistamab excipients
• Known contraindications to the use of daratumumab or lenalidomide per local prescribing information
Drug: Teclistamab, Drug: Daratumumab, Drug: Lenalidomide, Drug: Dexamethasone
Multiple Myeloma
UT Southwestern
A Prospective, Multi-center, Randomized Controlled Blinded Trial Demonstrating the Safety and Effectiveness of VNS Therapy® System as Adjunctive Therapy Versus a No Stimulation Control in Subjects With Treatment-Resistant Depression (RECOVER)
Objectives of this study are to determine whether active VNS Therapy treatment is superior to a no stimulation control in producing a reduction in baseline depressive symptom severity, based on multiple depression scale assessment tools at 12 months from randomization.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Hila.AbushSegev@UTSouthwestern.edu
Kala Bailey
All
18 Years and over
N/A
NCT03887715
STU-2022-0515
Inclusion Criteria:
The patient must be in a major depressive disorder (MDD) episode for ≥ two years or have
had at least four episodes of MDD, including the current episode.
The patient's depressive illness meets a minimum criterion of four prior failed treatments
of adequate dose and duration as measured by a tool designed for this purpose.
The patient is experiencing a major depressive episode (MDE) as measured by a guideline
recommended depression scale assessment tool on two visits, within a 45-day span prior to
implantation of the VNS device.
Patients must maintain a stable medication regimen for at least four weeks before device
implantation.
Exclusion Criteria:
Current or lifetime history of psychotic features in any MDE;
Current or lifetime history of schizophrenia or schizoaffective disorder;
Current or lifetime history of any other psychotic disorder;
Current or lifetime history of rapid cycling bipolar disorder;
Current secondary diagnosis of delirium, dementia, amnesia, or other cognitive disorder;
Current suicidal intent; or
Treatment with another investigational device or investigational drugs.
Device: Vagus Nerve Stimulation (VNS)
Treatment Resistant Depression, Brain and Nervous System
VNS, Depression, TRD
UT Southwestern
Abatacept in Immune Checkpoint Inhibitor Myocarditis (ATRIUM)
The primary aim is to test whether abatacept, as compared to placebo, is associated with a reduction in major adverse cardiac events (MACE) among participants hospitalized with myocarditis secondary to an immune checkpoint inhibitor (ICI). The primary outcome, MACE, is a composite of first occurrence of cardiovascular death, non-fatal sudden cardiac arrest, cardiogenic shock, significant ventricular arrythmias, significant bradyarrythmias, or incident heart failure.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Vlad Zaha
All
18 Years and over
Phase 3
NCT05335928
STU-2022-0624
Inclusion Criteria:
• Must have provided informed consent in a manner approved by the Investigator's Institutional Review Board (IRB) prior to any study-related procedure being performed. If a participant is unable to provide informed consent due to his/her medical condition, the participant's legally authorized representative may consent on behalf of the study participant, as permitted by local law and institutional Standard Operating Procedures;
• Aged greater than or equal to 18 years at the time of informed consent;
• Recent use of an FDA-approved immune checkpoint inhibitor (ICI, defined as administered an immune checkpoint inhibitor ≤ 6 months of myocarditis diagnosis), alone or in combination with other cancer therapies (i.e. chemotherapy, radiation therapy or targeted therapy). The FDA-approved ICI could be given as part of a clinical trial but not in combination with a new investigational agent which may cause myocarditis;
• A diagnosis of myocarditis.
• Hospitalized at the time of randomization;
• On 1000 mg of solumedrol per day for myocarditis or with an intent to initiate 1000 mg of solumedrol per day for myocarditis within 24 hours of first administration of study drug;
• Serum evidence of ongoing myocardial injury: Serum evidence of ongoing myocardial injury will be defined as an institutional troponin (either conventional or high-sensitivity troponin I or T, using the standard institutional assay) with a value that is ≥5 times the upper limit of the reference standard normal for that institution. The troponin assay may be adjusted based on sex depending on institutional standards. This value of troponin of ≥5 times above the institutional upper limits of normal value must be noted within 10 days prior to potential randomization. The 10-day period can be in the outpatient or inpatient setting. For example, a participant with a troponin value that on one occasion was ≥5 times the upper limits of institutional normal in the 10-day window prior to potential randomization (whether in the inpatient or outpatient setting), but later decreases below that threshold, typically due to starting corticosteroids, would still be considered eligible;
• The following laboratory parameters, not older than 48 hours at the time of randomization, and measured as part of usual care:
• Total white blood cell (WBC) count >2,500/μl
• Absolute neutrophil count (ANC) >1,500/μL
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) <20 times the upper limit of the institutional normal ranges;
• Women of childbearing potential (i.e., not postmenopausal, or surgically sterilized) must have a negative highly sensitive urine or serum pregnancy test prior to randomization. Participating women of childbearing potential must be willing to consistently use effective methods of contraception from screening until at least 90 days after administration of the last dose of study drug. Participating men must also be willing to consistently use effective methods of contraception from screening until at least 90 days after administration of the last dose of study drug; and
• Must be willing and able to abide by all study requirements and restrictions.
Exclusion Criteria:
• Must not have experienced any of the following (as defined in the section on the primary endpoint) in the 30-day period prior to randomization:
• A sudden cardiac arrest
• Cardiogenic shock as defined. A significant bradyarrhythmia (Mobitz type II second degree atrioventricular block or third degree (complete) atrio-ventricular (AV) block, for which an intervention with a temporary or permanent pacemaker is completed or recommended).
• A significant tachyarrhythmia (ventricular fibrillation of any duration or sustained ventricular tachycardia (>30 seconds, >120 beats per minute); or a ventricular tachyarrhythmia requiring intervention.
• Recent (≤2 month) exposure to abatacept or belatacept.
• Concurrent or recent (≤2 month) use of the following non-corticosteroid immunosuppressive therapies prior to randomization: mycophenolate, JAK STAT inhibitors (including but not limited to upadacitinib, tofacitinib, baricitinib, and filgotinib), tacrolimus, anti-thymocyte globulin, alemtuzumab, infliximab, and plasma exchange. The use of intravenous immunoglobulin is permitted prior to randomization and during study treatment.
• Currently enrolled in another interventional study utilizing systemic agents for the management of ICI-related toxicities.
• Female who is pregnant, breastfeeding, or is considering becoming pregnant during the study or for approximately 90 days after the last dose of study drug.
• Male who is considering fathering a child or donating sperm during the study or for approximately 30 days after the last dose of study drug.
• Any active, chronic, or recurrent viral infection that, based on the investigator's clinical assessment, makes the participant an unsuitable candidate for the study. These may include hepatitis B virus (HBV) or hepatitis C virus (HCV), recurrent or disseminated (even a single episode) herpes zoster, and disseminated (even a single episode) herpes simplex. Active HBV and HCV are defined as: HBV: hepatitis B surface antigen (HBs Ag) positive (+) or detected sensitivity on the HBV deoxyribonucleic acid (DNA) polymerase chain reaction (PCR) qualitative test for Hepatitis B core antibody (HBc Ab) positive (+) participants; HCV: HCV ribonucleic acid (RNA) detectable in any participant with anti-HCV antibody (HCV Ab). Patients with active Covid-19 infection will be excluded. This is defined as the period of ongoing symptoms in the setting of a positive Covid-19 test, or until 10 days after symptom onset and after resolution of fever for at least 24 hours, without the use of fever-reducing medications.
• Known active tuberculosis (TB), history of incompletely treated TB, suspected or known extrapulmonary TB, suspected or known systemic bacterial or fungal infections;
• Receipt of any live vaccine within four weeks prior to the first dose of study drug, or expected need of live vaccination during study participation including at least 90 days after the last dose of IV study drug.
• Any medical condition that could interfere with, or for which the treatment might interfere with, the conduct of the study or interpretation of the study results, or that would, in the opinion of the Investigator, increase the risk of the participant by participating in the study.
• Any factors that, in the Investigator's opinion, are likely to interfere with study procedures, such as history of noncompliance with scheduled appointments.
Drug: Abatacept plus, Drug: Placebo
Myocarditis Acute, Cancer, Anus, Bones and Joints, Brain and Nervous System, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Esophagus, Eye and Orbit, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Small Intestine, Soft Tissue, Stomach, Thyroid, Unknown Sites, Urinary Bladder
Immune checkpoint Inhibitor, Myocarditis, Abatacept, Immune therapy, Immune related adverse events
UT Southwestern
Study of R289 in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS)
The study will be an open-label, Phase 1b study of R289 to determine tolerability and preliminary efficacy in patients with LR MDS who are relapsed, refractory/resistant, intolerant, or have inadequate response to prior therapies such as erythropoietin (EPO), thrombopoietin (TPO), luspatercept, or hypomethylating agents (HMAs) for MDS.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Yazan Madanat
All
18 Years and over
Phase 1/Phase 2
NCT05308264
STU-2022-0561
Inclusion Criteria:
• Patient must be ≥ 18 years of age at the time of signing the informed consent.
• Must have definitive diagnosis of MDS with very low, low, or intermediate-1 risk (International Prognostic Scoring System (IPSS)-R ≤ 3.5) and ≤5% bone marrow myeloblasts.
• Must be relapsed, refractory/resistant, intolerant, or have inadequate response to therapies with known clinical benefits for MDS, such as TPOs, EPOs, luspatercept, and HMAs(i.e., azacytidine or decitabine). Patients with del (5q) must have failed prior lenalidomide therapy.
• Must meet at least one of the disease-related criteria for RBC transfusion, or platelet count within 8 weeks prior to initial administration of study treatment:
• Symptomatic anemia untransfused with hemoglobin < 9.0 g/dL within 8 weeks of registration or red blood cell (RBC) transfusion dependent defined as receiving ≥ 2 units of packed red blood cells (PRBCs) within 8 weeks in the preceding 16 weeks for a hemoglobin <9.0 g/dL.
• Clinically relevant thrombocytopenia (platelet counts of <100 × 109/L in at least 2 blood counts prior to study treatment and transfusion dependence). All subjects must have documented marrow iron stain. If marrow iron stain is not available, the transferrin saturation must be >20% or a serum ferritin > 100ng/100mL
• Must have Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2 at screening.
• Must have adequate organ function, defined as:
• Hepatic function:
• aspartate amino transferase (AST) or alanine aminotransferase (ALT) ≤ 1.5 × upper limit of normal (ULN)
• total bilirubin ≤ 1.5 × ULN
• Renal function defined as creatinine clearance > 60 mL/min (using Cockcroft-Gault), or blood creatine < 1.5 mg/dL
Exclusion Criteria:
• Prior treatment for MDS (i.e., TPOs, EPOs, HMAs) concluded < 2 weeks, luspatercept < 3 weeks, prior to study treatment
• Clinically significant anemia resulting from iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis, or GI bleeding.
• MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases.
• Diagnosis of chronic myelomonocytic leukemia.
• History of uncontrolled seizures.
• Uncontrolled bacterial or viral infection (i.e., documented HIV, hepatitis B or hepatitis C).
• History of an active malignancy within the past 2 years prior to study entry, with the exception of:
• Adequately treated in situ carcinoma of the cervix uteri
• Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin, or
• Any other malignancy with a life expectancy of more than 2 years
• History of or active, clinically significant, cardiovascular, respiratory, GI, renal, hepatic, neurological, psychiatric, musculoskeletal, genitourinary, dermatological, or other disorder that, in the Investigator's opinion, could affect the conduct of the study or the absorption, metabolism or excretion of the study treatment.
• Prior history of bone marrow transplantation.
• Marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval > 480 milliseconds [msec]) (Common Terminology Criteria for Adverse Events [CTCAE] Grade 1) using Fridericia's QT correction formula.
• History of additional risk factors for TdP (e.g., heart failure, hypokalemia, family history of Long QT Syndrome).
• Receiving any other concurrent chemotherapy, radiotherapy, or immunotherapy (within 2 weeks of initiating study treatment), or the toxicity of the relevant prior treatment has not been resolved yet.
• Use of concomitant medications that prolong the QT/QTc interval during study treatment
• Use of concomitant medications that are strong CYP3A or CYP2B6 inhibitors or inducers during study treatment
Drug: R906289 Monosodium (R289 Na)
Low Risk Myelodysplastic Syndromes, Myeloid and Monocytic Leukemia
MDS, LR MDS, Myelodysplastic Syndromes, Hematology Oncology, Hem/ Onc
UT Southwestern
Tagraxofusp in Pediatric Patients With Relapsed or Refractory CD123 Expressing Hematologic Malignancies
Tagraxofusp is a protein-drug conjugate consisting of a diphtheria toxin redirected to target CD123 has been approved for treatment in pediatric and adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). This trial aims to examine the safety of this novel agent in pediatric patients with relapsed/refractory hematologic malignancies. The mechanism by which tagraxofusp kills cells is distinct from that of conventional chemotherapy. Tagraxofusp directly targets CD123 that is present on tumor cells, but is expressed at lower or levels or absent on normal hematopoietic stem cells. Tagraxofusp also utilizes a payload that is not cell cycle dependent, making it effective against both highly proliferative tumor cells and also quiescent tumor cells. The rationale for clinical development of tagraxofusp for pediatric patients with hematologic malignancies is based on the ubiquitous and high expression of CD123 on many of these diseases, as well as the highly potent preclinical activity and robust clinical responsiveness in adults observed to date. This trial includes two parts: a monotherapy phase and a combination chemotherapy phase. This design will provide further monotherapy safety data and confirm the FDA approved pediatric dose, as well as provide safety data when combined with chemotherapy. The goal of this study is to improve survival rates in children and young adults with relapsed hematological malignancies, determine the recommended phase 2 dose (RP2D) of tagraxofusp given alone and in combination with chemotherapy, as well as to describe the toxicities, pharmacokinetics, and pharmacodynamic properties of tagraxofusp in pediatric patients. About 54 children and young adults will participate in this study. Patients with Down syndrome will be included in part 1 of the study.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Tamra Slone
All
1 Year to 21 Years old
Phase 1
NCT05476770
STU-2022-1157
Inclusion Criteria:
Age
• Patients must be ≥ 1 and ≤21 years of age at the time of study enrollment. Diagnosis
• Relapsed and/or refractory hematologic malignancy (including, but not limited to, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, mixed phenotype acute leukemia, acute undifferentiated leukemia, blastic plasmacytoid dendritic cell neoplasm, Hodgkin lymphoma, and non-Hodgkin lymphoma).
• Tumor cells must demonstrate surface expression of CD123 at the time of enrollment by flow cytometry or immunohistochemistry, as defined by the local institution. Disease Status: Monotherapy, Part 1
• Second or greater relapse; or
• Refractory after 2 or more chemotherapy cycles; or
• First relapse after primary chemotherapy-refractory disease; or
• BPDCN in first relapse or refractory after 1 or more chemotherapy cycles Combination therapy, Part 2
• First or greater relapse; or
• Refractory after 2 or more chemotherapy cycles; or
• BPDCN in first relapse or refractory after 1 or more chemotherapy cycles For relapsed/refractory leukemia, patients must have:
• >5% blasts in the bone marrow aspirate by morphology or flow cytometry
• Patients with 1% - 5% blasts are eligible for Part 2, Cohort C (only), if A single bone marrow sample with flow cytometry and at least one other test (e.g. karyotype, FISH, PCR, or NGS) shows ≥ 1% leukemic blasts and/or flow cytometry demonstrates a stable or rising level of disease on two serial bone marrows. For relapsed/refractory non-Hodgkin or Hodgkin lymphoma, patients must have:
• Histologic verification of relapse
• Measurable disease documented by radiographic criteria or bone marrow
• Patients in Part 1 may have sites of non-CNS extramedullary disease, but no CNS disease. Patients in Part 2 may have CNS disease and/or other non-CNS extramedullary disease. No cranial irradiation is allowed during the protocol therapy.
• Patients with Down syndrome are eligible. Performance Level
• Karnofsky > 50% for patients > 16 years of age and Lansky > 50% for patients ≤ 16 years of age (See Appendix I for Performance Scales). Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score. Prior Therapy
• Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy, defined as resolution of all such toxicities to ≤ Grade 2 or lower per the inclusion/exclusion criteria. Myelosuppressive chemotherapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study. At least 14 day must have elapsed since the completion of myelosuppressive therapy. However, individuals may receive any of the following medications within 14 days without a "wash-out period":
• Hydroxyurea: Hydroxyurea can be initiated and/or continued for up to 24 hours prior to the start of protocol therapy.
• "Maintenance-style" therapy: therapy including vincristine (dosed a maximum of one-time weekly), oral 6-mercaptopurine, oral methotrexate (dosed a maximum of one-time weekly), intrathecal therapy (dosed a maximum of one-time weekly) and/or dexamethasone (dosed at ≤3 mg/m2/dose twice daily) or prednisone (dosed at ≤20 mg/m2/dose twice daily) can be continued for up to 24 hours prior to entering the study.
• Hematopoietic stem cell transplant: Patients who have experienced their relapse after a HSCT are eligible, provided they have no evidence of acute or chronic Graft-versus-Host Disease (GVHD) and are at least 100 days post-transplant at the time of enrollment.
• Hematopoietic growth factors: It must have been at least 7 days since the completion of therapy with granulocyte colony stimulating factor (GCSF) or other growth factors at the time of enrollment. It must have been at least 14 days since the completion of therapy with pegfilgrastim (Neulasta®).
• Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.
• Monoclonal antibodies: Maximum of 3 half-lives of the antibody or 21 days (whichever is shorter) must have elapsed after the last dose of monoclonal antibody.
• Immunotherapy: At least 30 days from last infusion of chimeric antigen receptor T cell (CART) therapy or tumor vaccine.
• XRT: Craniospinal XRT is prohibited during protocol therapy. No washout period is necessary for radiation given to any extramedullary site other than CNS chloromas; ≥ 90 days must have elapsed if prior TBI or craniospinal XRT.
• Patients that have received other non-tagraxofusp CD123 targeting agents are eligible. Patients that have previously received tagraxofusp are not eligible. Organ Function Requirements Adequate Bone Marrow Function Defined as:
• Patients should not be known to be refractory to red blood cell or platelet transfusions.
• Blood counts are not required to be normal prior to enrollment on trial. However, platelet count must be ≥20,000/mm3 to initiate therapy (may receive platelet transfusions). Adequate Renal Function Defined as:
• Patient must have a calculated creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73m2 OR a normal serum creatinine based on age/gender in the chart below: Maximum Serum Creatinine (mg/dL):
• 1 to < 2 years old - Male: 0.6, Female: 0.6
• 2 to < 6 years old - Male:0.8, Female: 0.8
• 6 to < 10 years old - Male: 1, Female: 1
• 10 to < 13 years old - Male: 1.2, Female: 1.2
• 13 to < 16 years old - Male: 1.5, Female: 1.4
• ≥ 16 years old - Male: 1.7, Female: 1.4 The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC. Adequate Liver Function Defined as:
• Total bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x institutional upper limit of normal for age
• SGPT (ALT) and SGOT (AST) must be less than 3x institutional upper limit of normal.
• Serum albumin ≥3.2 g/dL (albumin infusion independent). Adequate Cardiac Function Defined as:
• Shortening fraction of ≥27% by echocardiogram, or
• Ejection fraction of ≥ 50% by gated radionuclide study/echocardiogram. Adequate Pulmonary Function Defined as:
• Pulse oximetry > 94% on room air (> 90% if at high altitude)
• No evidence of dyspnea at rest and no exercise intolerance. Reproductive Function
• Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed within 2 weeks prior to enrollment.
• Female patients with infants must agree not to breastfeed their infants while on this study.
• Male and female patients of child-bearing potential must agree to use an effective method of contraception approved by the investigator during the study and for 12 weeks after the last dose of tagraxofusp. Exclusion Criteria Disease Status:
• Patients with CNS disease are not eligible for Part 1.
• Patients with isolated CNS disease are not eligible for Part 1 or Part 2.
• Patients with isolated non-CNS disease are eligible for Part 1 and Part 2. Concomitant Medications
• Corticosteroids - Patients receiving corticosteroids for disease control who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible.
• Investigational Drugs - Patients who are currently receiving another investigational drug are not eligible. The definition of "investigational" for use in this protocol means any drug that is not licensed by the FDA, Health Canada or the Therapeutic Goods Administration to be sold in the countries they govern. (United States, Canada and Australia)
• Anti-cancer Agents - Patients who are currently receiving or may receive while on therapy, other anti-cancer agents, radiation therapy or immunotherapy are not eligible [except hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy]. Intrathecal chemotherapy (at the discretion of the primary oncologist) may be given up to one week prior to the initiation of study treatment (day 1 therapy).
• Anti-GVHD or agents to prevent organ rejection post-transplant - Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial. At least 4 weeks must have elapsed after the last dose of GVHD meds. Infection Criteria - Patients are excluded if they have:
• Positive blood culture within 48 hours of study enrollment;
• Fever above 38.2 within 48 hours of study enrollment with clinical signs of infection. Fever that is determined to be due to tumor burden is allowed if patients have documented negative blood cultures for at least 48 hours prior to enrollment and no concurrent signs or symptoms of active infection or hemodynamic instability.
• A positive fungal culture within 30 days of study enrollment.
• Active fungal, viral, bacterial, or protozoal infection requiring IV treatment. Chronic prophylaxis therapy to prevent infections is allowed.
• Patients will be excluded if they have a known allergy to any of the drugs used in the study.
• Patients will be excluded if they have significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance with the protocol treatment or procedures, interfere with consent, study participation, follow up, or interpretation of study results.
• Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded.
Drug: Tagraxofusp, Drug: Fludarabine, Drug: Cytarabine, Drug: Dexamethasone, Drug: Vincristine, Drug: Azacitidine, Drug: Methotrexate, Drug: Cytarabine IT, Drug: Hydrocortisone
Hematologic Malignancy, AML, ALL, BPDCN, MDS, Lymphoblastic Lymphoma, Lymphoma, B-cell, Lymphoma, T-Cell, Hodgkin Lymphoma, Mixed Phenotype Acute Leukemia, Acute Undifferentiated Leukemia, Hodgkins Lymphoma, Leukemia, Not Otherwise Specified, Leukemia, Other, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma
Children’s Health
Physical Rehabilitation for Older Patients With Acute Heart Failure With Preserved Ejection Fraction (REHAB-HFpEF)
The REHAB-HFpEF trial will determine whether a novel physical rehabilitation intervention will improve the primary outcome of combined all-cause rehospitalizations and mortality and the secondary outcome of major mobility disability during 6-month follow-up in patients hospitalized for heart failure and preserved ejection fraction (HFpEF), which is nearly unique to older persons, and for which there are few treatment options.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Pedro.Rosario-Favela@UTSouthwestern.edu
Ambarish Pandey
All
60 Years and over
N/A
NCT05525663
STU-2022-0992
Inclusion Criteria:
• Age >=60 years old
• Ejection Fraction >=45%
• In the hospital setting >24 hours for the management of acute decompensated heart failure (ADHF), or diagnosed with ADHF after being hospitalized for another reason. ADHF will be confirmed by the site physician, and will be defined according to the Food and Drug Administration (FDA) definition of hospitalized heart failure as a combination of symptoms, signs, and HF-specific medical treatments, and requires that all 4 of the following are met:
• At least 1 symptom of HF which has worsened from baseline: a. dyspnea at rest or with exertion; b. exertional fatigue; c. orthopnea; d. paroxysmal nocturnal dyspnea (PND)
• At least 2 of the following signs of HF: a. Pulmonary congestion or edema on physical exam (rales or crackles) or by chest X-ray; b. Elevated jugular venous pressure or central venous pressure >=10 mm Hg; c. peripheral edema; d. wedge or left ventricular end diastolic pressure >=15 mmHg; e. rapid weight gain (>=5 lbs.); f. Increased b-type natriuretic peptide (BNP) (>=100 pg/ml) or N-terminal prohormone BNP (>=220pg/ml)
• Change in medical treatment specifically targeting HF, defined as change in dose or initiation of or augmentation of at least 1 of the following therapies: a. diuretics; b. vasodilators; c. other neurohormonal modulating agents, including angiotensinconverting enzyme inhibitors, angiotensin II receptor blockers (with or without neprilysin inhibitor), beta-blockers, aldosterone inhibitors, direct renin inhibitors, or sodium-glucose co-transporter-2 inhibitors
• The primary cause of symptoms and signs is judged by the investigator to be due to HF
• Adequate clinical stability to allow participation in study assessments and the intervention Independent with basic activities of daily living, including the ability to ambulate independently (with or without the use of an assistive device) prior to admission
• Able to walk 4 meters (with or without the use of an assistive device) at the time of enrollment
Exclusion Criteria:
• Acute myocardial infarction within the past 3 months, or planned coronary artery intervention (percutaneous or surgical) within the next 6 months (Note: given that cardiac biomarkers such as troponin are frequently elevated in HF patients, the diagnosis of acute myocardial infarction should be based on clinical diagnosis, not biomarkers alone)
• Severe aortic or mitral valve stenosis
• Severe valvular heart disease with planned intervention within next 6 months
• Known pericardial constriction, genetic hypertrophic cardiomyopathy, or infiltrative cardiomyopathy including amyloid heart disease (amyloidosis)
• Planned discharge other than to home or a facility where the participant will live independently
• Terminal illness other than HF with life expectancy <1 year
• Impairment from stroke or other medical disorders that preclude participation in the intervention
• Known dementia by medical record documentation, OR patients with Montreal Cognitive Assessment (MoCA) <=18 AND without social support, OR MoCA <10 regardless of social support
• Advanced chronic kidney disease defined as estimated glomerular filtration rate <20 mL/min/1.73 m2 or on chronic or intermittent dialysis or dialysis anticipated within the next 6 months
• Already engaging in regular moderate to vigorous exercise conditioning defined as >30 minutes per day, >= twice per week consistently during the previous 6 weeks
• Enrollment in a clinical trial not approved for co-enrollment
• High risk for non-adherence as determined by screening evaluation
• Inability or unwillingness to comply with the study requirements or give consent
Behavioral: Rehabilitation Intervention
Heart Failure With Preserved Ejection Fraction, Heart
UT Southwestern
Accelerated v's Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumours (P3BEP)
The purpose of this study is to determine whether accelerated BEP chemotherapy is more effective than standard BEP chemotherapy in males with intermediate and poor-risk metastatic germ cell tumours.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Jonathan Wickiser
All
11 Years to 45 Years old
Phase 3
NCT02582697
STU-2018-0042
Inclusion Criteria:
• Age ≥ 11 years and ≤ 45 years on the date of randomisation
• Histologically or cytologically confirmed germ cell tumour (non-seminoma or seminoma); or Exceptionally raised tumour markers (AFP ≥ 1000ng/mL and/or HCG ≥ 5000 IU/L) without histologic or cytologic confirmation in the rare case where pattern of metastases consistent with GCT, high tumour burden, and a need to start therapy urgently
• Primary arising in testis, ovary, retro-peritoneum, or mediastinum
• Metastatic disease or non-testicular primary
• Intermediate or poor prognosis as defined by IGCCC classification3 (modified with different LDH criteria for intermediate risk non-seminoma, and inclusion of ovarian primaries). (See protocol for more information).
• Adequate bone marrow function with ANC ≥1.0 x 10^9/L, Platelet count ≥100 x 10^9/L
• Adequate liver function where bilirubin must be ≤1.5 x ULN, except participants with Gilbert's Syndrome where bilirubin must be ≤2.0 x ULN; ALT and AST must be ≤2.5 x ULN, except if the elevations are due to hepatic metastases, in which case ALT and AST must be ≤ 5 x ULN
• Adequate renal function with estimated creatinine clearance of ≥60 ml/min according to the Cockcroft-Gault formula, unless calculated to be < 60 ml/min or borderline in which case GFR should be formally measured, eg. with EDTA scan
• ECOG Performance Status of 0, 1, 2, or 3
• Study treatment both planned and able to start within 14 days of randomisation.
• Willing and able to comply with all study requirements, including treatment, timing and nature of required assessments
• Able to provide signed, written informed consent
Exclusion Criteria:
• Other primary malignancy (EXCEPT adequately treated non-melanomatous carcinoma of the skin, germ cell tumour, or other malignancy treated at least 5 years previously with no evidence of recurrence)
• Previous chemotherapy or radiotherapy, except if patient has pure seminoma relapsing after adjuvant radiotherapy or adjuvant chemotherapy with 1-2 doses of single agent carboplatin or if patient has non-seminoma and poor prognosis by IGCCC criteria in the rare case where low-dose induction chemotherapy is given prior to registration because patient is not fit enough to receive protocol chemotherapy (eg. organ failure, vena cava obstruction, overwhelming burden of disease). In these instances acceptable regimens include cisplatin 20 mg/m^2 days 1-2 and etoposide 100 mg/m^2 days 1-2; carboplatin AUC 3 days 1-2 and etoposide 100 mg/m^2 days 1-2; or baby-BOP. Patients must meet all other inclusion and exclusion criteria at the time of registration. Additionally participants who need to start therapy urgently prior to completing study-specific baseline investigations may commence study chemotherapy prior to registration and randomisation. Such patients must be discussed with the coordinating centre prior to registration, and must be registered within 10 days of commencing study chemotherapy.
• Significant cardiac disease resulting in inability to tolerate IV fluid hydration for cisplatin
• Significant co-morbid respiratory disease that contraindicates the use of bleomycin
• Peripheral neuropathy ≥ grade 2 or clinically significant sensorineural hearing loss or tinnitus
• Concurrent illness, including severe infection that may jeopardize the ability of the participant to undergo the procedures outlined in this protocol with reasonable safety
• Inadequate contraception. Men must use 2 effective methods of contraception, including use of a condom, during chemotherapy and for a year after completing chemotherapy.
• Known allergy or hypersensitivity to any of the study drugs
• Presence of any psychological, familial, sociological or geographical condition that in the opinion of the investigator would hamper compliance with the study protocol and follow-up schedule, including alcohol dependence or drug abuse The above inclusion and exclusion criteria will apply to stage 1 (n=150) and stage 2 (n=500 including stage 1) of the study. All sites will participate in both stages of the study with the exception of the Children's Oncology Group who will be participate in stage 1 only.
Drug: Bleomycin (active name: Bleomycin Sulfate), Drug: Etoposide, Drug: Cisplatin, Drug: Pegylated G-CSF (Pegfilgrastim), Drug: Filgrastim
Germ Cell Tumor, Other Female Genital, Other Male Genital, Ovary
Germ Cell, Intermediate and poor-risk metastatic germ cell tumours
Children’s Health
GammaPod Registry and Quality of Life Nomogram (GCC 1876)
This study is a prospective, single arm study (registry) summarizing patient-level adverse-event and tumor outcomes as well as a number of feasibility and dosimetric characteristics of delivering a single-fraction boost with the GammaPod.
Call 833-722-6237
canceranswerline@utsouthwestern.edu
Assal Rahimi
Female
18 Years and over
N/A
NCT03562273
STU 052018-052
Inclusion Criteria:
• The patient must sign consent for study participation.
• The patient must be female and have a diagnosis of an invasive or non-invasive breast cancer that was treated surgically by a partial mastectomy.
• The patient must be deemed an appropriate candidate for breast conserving therapy (i.e. not pregnant, never had radiation to the treated breast, breast size would allow adequate cosmesis after volume loss from partial mastectomy).
• Patients with involved lymph nodes are candidates for the study.
• Surgical margins are negative for invasive (no tumor on ink) or non-invasive breast cancer (2 mm negative margin).
• The greatest dimension of the tumor is less than 4cm before surgery.
• Multifocal disease is allowed if it was removed by a single lumpectomy resection and the patient remained a candidate for breast conservation.
• Age 18 years and older.
• Women of childbearing potential (pre-menopausal defined as having a menstrual period within the past 1 year) must have a negative serum pregnancy test or complete a pregnancy waiver form per institutional policy.
• The surgical cavity is clearly visible on CT images. Of note, clips are not required but recommended.
• The patient must weigh less than 150Kg (330lb), which is the limit of the imaging couch.
• The patient must be less than 6'6" in height.
• The patient must feel comfortable in the prone position.
• Diagnosis of prior contralateral breast cancer is allowed.
• Diagnosis of synchronous bilateral cancers is allowed. In this case if bilateral boosts are required, a patient would not have both treatments on the same day.
• Oncoplastic reduction surgery is allowed if the lumpectomy cavity can be clearly visualized.
Exclusion Criteria:
• Patients with proven multi-centric carcinoma (tumors in different quadrants of the breast or tumor separated by at least 4 cm).
• Prior radiation therapy to that breast or that hemi thorax.
• Unable to fit into the immobilization breast cup with an adequate seal.
• Male gender.
• Patient cannot comfortably be set up in the prone position (i.e. physical disability)
• Unable to fit into the breast immobilization device due to breast size or other anatomical reason.
• Mastectomy is the surgery performed.
• Patient has received prior radiotherapy to the involved breast.
• Tumor bed is less than 3 mm from the skin surface.
• Greater than 50% of the target volume is above the upper border of the table.
• Patients with skin involvement, regardless of tumor size.
• Patients with connective tissue disorders specifically systemic lupus erythematosis, scleroderma, or dermatomyositis.
• Patients with psychiatric or addictive disorders that would preclude obtaining informed consent.
• Patients who are pregnant or lactating due to potential exposure of the fetus to RT and unknown effects of RT to lactating females.
• Patients with breast implants/tissue expanders or flap reconstruction.
Radiation: Quality Of Life Sizing Nomogram
Breast Cancer Female, Breast - Female
Breast Nomogram, Breast Cancer Quality of Life, GammaPod Registry
UT Southwestern; Parkland Health & Hospital System
Mechanisms of Exercise Intolerance in Heart Failure With Preserved Ejection Fraction
The global objective of this study is to determine the mechanisms of exercise intolerance and dyspnea on exertion (DOE) in patients with HFpEF and based on this pathophysiology, test whether specific exercise training programs (whole body vs single leg) will result in improved exercise tolerance.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Mary.Childers@UTSouthwestern.edu
Benjamin Levine
All
60 Years to 90 Years old
N/A
NCT04068844
STU-2019-0617
Inclusion Criteria:
• signs and symptoms of heart failure
• an ejection fraction > 0.50
• objective evidence of diastolic dysfunction
Exclusion Criteria:
• age < 60 years
• BMI > 50 kg/m2
• PDE5 inhibitor use
• Severe valvular disease
• Severe COPD
• CKD 4 or higher
• Contra-indication to MRI.
Behavioral: Exercise training
Heart Failure, Diastolic
Exercise, Hemodynamics,, Heart failure
UT Southwestern
PROSpect: Prone and Oscillation Pediatric Clinical Trial
Severe pediatric acute respiratory distress syndrome (PARDS) is a life-threatening and frequent problem experienced by thousands of children each year. Little evidence supports current supportive practices during their critical illness. The overall objective of this study is to identify the best positional and/or ventilation practice that leads to improved patient outcomes in these critically ill children. We hypothesize that children with severe PARDS treated with either prone positioning or high-frequency oscillatory ventilation (HFOV) will demonstrate more days off the ventilator when compared to children treated with supine positioning or conventional mechanical ventilation (CMV).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Eduardo.Rodriguez2@childrens.com
Peter Luckett
All
up to 18 Years old
N/A
NCT03896763
STU-2019-0488
Inclusion criteria:
Intubated and mechanically ventilated with moderate-severe PARDS for <48 hours per PALICC
guidelines (chest imaging consistent with acute pulmonary parenchymal disease and OI ≥12 or
OSI ≥10). We require two blood gases meeting moderate-severe PARDS criteria (separated by
at least 4 ± 2 hours during which time the clinical team is actively working to recruit
lung volume and optimize the patient's hemodynamic status per PALICC guidelines;
specifically, incremental and decremental PEEP changes to optimize lung volume). A second
blood gas is not required for OI ≥16.
Exclusion criteria:
• Perinatal related lung disease
• Congenital diaphragmatic hernia or congenital/acquired diaphragm paralysis
• Respiratory failure explained by cardiac failure or fluid overload
• Cyanotic heart disease
• Cardiomyopathy
• Unilateral lung disease
• Primary pulmonary hypertension
• Intubated for status asthmaticus
• Obstructive airway disease (e.g., Severe airways disease without parenchymal involvement or disease characterized by hypercapnia with FiO2 <0.30 and/or evidence of increased resistance visible on the flow - time scalar and/or presence of intrinsic PEEP)
• Active air leak
• Bronchiolitis obliterans
• Post hematopoietic stem cell transplant; specifically, patients receiving continuous supplemental oxygen for three or more days prior to intubation; receiving noninvasive ventilation for more than 24 hours prior to intubation; receiving more than one vasoactive medication at time of meeting inclusion criteria; spending more than four days in the PICU prior to intubation; supported on or with immediate plans for renal replacement therapies; with two or more allogeneic transplants; who relapsed after the transplant; or with diffuse alveolar hemorrhage
• Post lung transplant
• Home ventilator (including noninvasive) or home oxygen dependent (exception: night-time noninvasive ventilation (CPAP/BiPAP) or oxygen for obstructive sleep apnea is permitted)
• Neuromuscular respiratory failure
• Critical airway (e.g., post laryngotracheal surgery or new tracheostomy) or anatomical obstruction of the lower airway (e.g., mediastinal mass)
• Facial surgery or trauma in previous 2 weeks
• Head trauma (managed with hyperventilation)
• Intracranial bleeding
• Unstable spine, femur or pelvic fractures
• Acute abdominal process/open abdomen
• Morbid obesity (2w-24 months: WHO weight-for-length/height z-score ≥+3; ≥2 years: WHO body mass index (BMI)-for-age z-score ≥+3)
• Currently receiving either prone positioning or any high-frequency mode of MV with current illness (Up to 4 hours of prone positioning and/or any mode of high-frequency mode of MV is allowed as long as the therapies are off for least 4 hours prior to the subject meeting oxygenation criteria.)
• Supported on ECMO during the current admission
• Family/medical team not providing full support (patient treatment considered futile)
• Previously enrolled in current study
• Enrolled in any other interventional clinical trial not approved for co-enrollment
• Known pregnancy
• Perinatal related lung disease
• Congenital diaphragmatic hernia or congenital/acquired diaphragm paralysis
• Respiratory failure explained by cardiac failure or fluid overload
• Cyanotic heart disease
• Cardiomyopathy
• Unilateral lung disease
• Primary pulmonary hypertension
• Intubated for status asthmaticus
• Obstructive airway disease (e.g., Severe airways disease without parenchymal involvement or disease characterized by hypercapnia with FiO2 <0.30 and/or evidence of increased resistance visible on the flow - time scalar and/or presence of intrinsic PEEP)
• Active air leak
• Bronchiolitis obliterans
• Post hematopoietic stem cell transplant; specifically, patients receiving continuous supplemental oxygen for three or more days prior to intubation; receiving noninvasive ventilation for more than 24 hours prior to intubation; receiving more than one vasoactive medication at time of meeting inclusion criteria; spending more than four days in the PICU prior to intubation; supported on or with immediate plans for renal replacement therapies; with two or more allogeneic transplants; who relapsed after the transplant; or with diffuse alveolar hemorrhage
• Post lung transplant
• Home ventilator (including noninvasive) or home oxygen dependent (exception: night-time noninvasive ventilation (CPAP/BiPAP) or oxygen for obstructive sleep apnea is permitted)
• Neuromuscular respiratory failure
• Critical airway (e.g., post laryngotracheal surgery or new tracheostomy) or anatomical obstruction of the lower airway (e.g., mediastinal mass)
• Facial surgery or trauma in previous 2 weeks
• Head trauma (managed with hyperventilation)
• Intracranial bleeding
• Unstable spine, femur or pelvic fractures
• Acute abdominal process/open abdomen
• Morbid obesity (2w-24 months: WHO weight-for-length/height z-score ≥+3; ≥2 years: WHO body mass index (BMI)-for-age z-score ≥+3)
• Currently receiving either prone positioning or any high-frequency mode of MV with current illness (Up to 4 hours of prone positioning and/or any mode of high-frequency mode of MV is allowed as long as the therapies are off for least 4 hours prior to the subject meeting oxygenation criteria.)
• Supported on ECMO during the current admission
• Family/medical team not providing full support (patient treatment considered futile)
• Previously enrolled in current study
• Enrolled in any other interventional clinical trial not approved for co-enrollment
• Known pregnancy
Other: Either supine or prone positioning and either CMV or HFOV
Acute Respiratory Distress Syndrome in Children, Lung/Thoracic
Pediatric Acute Respiratory Distress Syndrome (PARDS), Acute Respiratory Distress Syndrome (ARDS), acute respiratory failure, child, pediatric intensive care unit
Children’s Health