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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 Study to Test if Fremanezumab is Effective in Preventing Chronic Migraine in Patients 6 to 17 Years of Age
The primary objective of the study is to evaluate the effectiveness of fremanezumab as compared to placebo for the preventive treatment of chronic migraine (CM). Secondary objectives are to further demonstrate the efficacy of Fremanezumab as compared to placebo for the preventive treatment of CM, to evaluate the safety and tolerability of Fremanezumab in the preventive treatment of CM and to evaluate the immunogenicity of Fremanezumab and the impact of antidrug antibodies (ADAs) on clinical outcomes in participants exposed to Fremanezumab The total duration of the study is planned to be 48 months.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kiley.Poppino@UTSouthwestern.edu
Deryk Walsh
All
6 Years to 17 Years old
Phase 3
NCT04464707
STU-2020-0683
Inclusion Criteria:
• The participant has a clinical history of recurrent headache consistent with the diagnosis of migraine for at least 6 months before screening, consistent with ICHD-3 criteria (Headache Classification Committee of the IHS 2013), and a history of ≥15 headache days per month, of which ≥8 headache days were assessed as migraine days per month in each of the 3 months prior to screening (visit 1).
• The participant or parent/caregiver maintain a prospectively collected headache diary
• The participant does not have chronic daily headache. For the purposes of this study, chronic daily headache is operationally defined as <4 headache-free days during the 28-day baseline period. NOTE: Additional criteria apply; please contact the investigator for more information.
Exclusion Criteria:
• The participant is using medications containing opioids (including codeine) or barbiturates (including Fiorinal®, Fioricet®, or any other combination containing butalbital) for the treatment of migraine during the 3 months prior to the day of the screening visit.
• The participant has used an intervention/device (eg, scheduled nerve block or transcranial magnetic stimulation) for the treatment of migraine or in the head or neck area for any condition during the 2 months prior to the day of the screening visit.
• 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 past or current history of cancer.
• The participant is pregnant, nursing.
• 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 patient has a current or past medical history of hemiplegic migraine. NOTE: Additional criteria apply; please contact the investigator for more information.
Drug: Fremanezumab, Drug: Placebo
Migraine
Children’s Health
A Study to Compare the Safety and Efficacy of Dysport® and Botox® in Adults With Upper Limb Spasticity. (DIRECTION)
This study is aiming to demonstrate the non-inferiority of AbobotulinumtoxinA (aboBoNT-A) versus OnabotulinumtoxinA (onaBoNT-A) as the primary safety endpoint, and the superiority of aboBoNT-A over onaBoNT-A with respect to duration of response as the key secondary efficacy endpoint when used at optimal doses according to approved prescribing information of each product.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Victoria.Castillo@UTSouthwestern.edu
Fatma Gul
All
18 Years to 75 Years old
Phase 4
NCT04936542
STU-2021-0659
Inclusion Criteria:
• Participant must be 18 to 75 years of age inclusive, at the time of signing the informed consent
• 2a. [US/France] Participants with stable Upper Limb Spasticity (ULS) for at least 3 months, in whom treatment of only one upper limb is necessary for the duration of the study;
• 2b. [Canada] Participants with stable post-stroke ULS for at least 3 months, in whom treatment of only one upper limb is necessary for the duration of the study
• Participants who are either naïve to Botulinum toxin type A (BoNT-A) for ULS or who have been previously treated with BoNT-A for ULS;
• Participants with MAS score of at least 2 at elbow, wrist and finger flexors;
• Participants with DAS score of at least 2 on the Principal Target of Treatment (PTT) (one of four functional domains: dressing, hygiene, limb position and pain);
• Participants who require BoNT-A injection in all of the following muscles: flexor carpi radialis, flexor carpi ulnaris, flexor digitorum profundus, flexor digitorum superficialis and biceps brachii;
• Participants for whom injection of a total dose of 900 Units aboBoNT-A or 360 Units onaBoNT-A is considered by the investigator to be clinically appropriate;
• Participants who have been stable for at least 3 months prior to study entry in terms of oral antispasticity, anticoagulant and/or anticholinergic medication if treated, and for at least 1 month prior to study entry in terms of occupational and/or physiotherapy treatment, if treated, and are considered by the investigator likely to remain stable for the duration of the study;
Exclusion Criteria:
• Major limitations in the passive range of motion in the paretic upper limb;
• Major neurological impairment (other than limb paresis) that could negatively affect functional performance;
• Participants clinically requiring injection into any upper limb muscles other than the five muscles of one arm listed in Section 5.1, or requiring injection into both arms or any lower limb within the timeframe of the study;
• Hypersensitivity to any BoNT product or excipients;
• Hypersensitivity to cow's milk protein (casein);
• Infection at the proposed injection site(s);
• Known peripheral motor neuropathic diseases, amyotrophic lateral sclerosis or neuromuscular junction disorders (e.g. myasthenia gravis or Lambert-Eaton syndrome);
• Any medical condition (including dysphagia or breathing difficulties/compromised respiratory function) that in the opinion of the investigator, might jeopardize the participant's safety;
• Women who are pregnant or lactating
• Participants treated with BoNT of any type for any indication (e.g. bladder injection, headache or cosmetic) within the previous 12 weeks or planned/likely to be treated during the course of the study;
• Prior history of non-responsiveness to BoNT treatment;
• Previous surgery, or administration of alcohol or phenol in the study limb 6 months or earlier from study enrolment or planned/likely to be treated during the course of the study;
• Participants treated with intrathecal baclofen, aminoglycosides or other agents interfering with neuromuscular transmission (e.g. curare-like agents), within the previous 4 weeks or planned/likely to be treated during the course of the study;
• Participants who received a COVID-19 vaccine injection within 7 days before the first planned study intervention injection, or planned/likely to be injected within 7 days after the first planned study intervention injection
• BoNT naïve participants with a history of facial neurogenic disorder (facial paralysis, polyradiculoneuropathy) (only for France).
Biological: AboBoNT-A, Biological: OnaBoNT-A
Upper Limb Spasticity
UT Southwestern; Parkland Health & Hospital System
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, Natalie.Booker@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 Evaluate the Efficacy & Safety of the INTERCEPT Blood System for RBCs in Complex Cardiac Surgery Patients (ReCePI)
The objective of this study is to evaluate the efficacy and safety of RBC transfusion for support of acute anemia in cardiovascular surgery patients based on the clinical outcome of renal impairment following transfusion of red blood cells (RBCs) treated with the INTERCEPT Blood System (IBS) for Red Blood Cells compared to patients transfused with conventional RBCs.
Call 214-648-5005
studyfinder@utsouthwestern.edu, salina.shrestha@utsouthwestern.edu
Ravindra Sarode
All
11 Years and over
Phase 3
NCT03459287
STU-2021-0761
Inclusion Criteria:
• Age ≥ 11 years of age
• Weight ≥ 40 kg
• Scheduled complex cardiac surgery or thoracic aorta surgery. The procedure may be performed either on or off cardiopulmonary bypass machine (CBP or "pump"). For the purposes of this protocol "Repeat procedure" means that the subject had a previous cardiac surgery. Procedures that qualify as complex cardiac surgery include but are not limited to, the following:
• Single Vessel Coronary Artery Bypass Graft, first or repeat procedure
• Multiple Coronary Artery Bypass Grafts, first or repeat procedure
• Single Valve Repair or Replacement, first or repeat procedure
• Multiple Valve Repair or Replacement, first or repeat procedure
• Surgery involving both Coronary Artery Bypass Graft(s) and Valve Repair(s), first or repeat procedure
• One or more of the following procedures, with or without Coronary Bypass Graft(s):
• left ventricular aneurysm repair
• ventricular and/or atrial septal defect repairs
• Batista procedure (surgical ventricular remodeling)
• surgical ventricular restoration
• congenital cardiac defect repair
• aortic procedures
• other cardiac surgery or thoracic aorta surgery types with a high probability of bleeding
• TRUST probability score (Alghamdi, Davis et al. 2006) ≥ 3, or currently on a regimen of aspirin (any dose), clopidogrel (or analogs) and/or GPIIb/IIIa inhibitors or at a high probability for need of a transfusion during or after surgery at the discretion of the Investigator
• Female subjects of child-bearing potential must meet the 2 criteria below at screening:
• Negative serum or urine pregnancy test
• Use at least one method of birth control that results in a low failure rate (i.e., less than 1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptives, some intrauterine devices (IUDs), sexual abstinence or vasectomized partner
• Signed and dated informed consent/assent form
Exclusion Criteria:
• Confirmed positive baseline serum/plasma antibody specific to INTERCEPT RBCs (S-303 specific antibody) screening panel prior to randomization.
• Pregnant or breast feeding
• Refusal of blood products or other inability to comply with the protocol in the opinion of the Investigator or the treating physician
• Treatment with any medication that is known to adversely affect RBC viability, such as, but not limited to dapsone, levodopa, methyldopa, nitrofurantoin, and its derivatives, phenazopyridine and quinidine.
• Planned cardiac transplantation
• Active autoimmune hemolytic anemia
• Left ventricular assist device (LVAD) or extracorporeal membrane oxygenation (ECMO) support pre operatively or planned need post-operatively
• Cardiogenic shock requiring pre-operative placement of an intra-aortic balloon pump (IABP) (NOTE: IABP done for unstable angina or prophylactically for low ejection fraction is not excluded).
• Planned use of autologous or directed donations.
• RBC transfusion during current hospitalization prior to enrollment and randomization (within 7 days).
• Participation in an interventional clinical study concurrently or within the previous 28 days. This includes investigational blood products, pharmacologic agents, imaging materials (including dyes), surgical techniques, or devices. Observational studies of FDA cleared or approved products or nutrition, psychology, or socioeconomic issues are not grounds for exclusion
• Patients with a current diagnosis of either chronic kidney disease or acute kidney injury and with sCr ≥1.8 mg/dL at screening and patients requiring RRT. (NOTE: If sCr at screening is <1.8 mg/dL, a patient with a diagnosis of chronic or acute kidney injury alone is not excluded).
• Patients with a current diagnosis of either chronic or acute hepatic insufficiency and with a total serum bilirubin ≥ 2.0 mg/dL (≥34.2 µmol/L). (NOTE: If total serum bilirubin at screening is <2.0 mg/dL, a patient with a diagnosis of chronic or acute hepatic failure alone is not excluded).
• Pre-existing antibody(ies) to RBC antigens that may make the provision of compatible study RBC components difficult.
• History of TRs requiring washed RBCs, volume reduced RBC, or RBCs with additive solution removed.
• Patients with documented IgA deficiency or a history of severe allergic reactions to blood products.
• Patients who require gamma-irradiated RBC blood components.
• Positive DAT as defined below: A polyspecific DAT reaction strength > 2+, or A polyspecific DAT (any strength) in conjunction with pan-reactivity with a commercial IAT antibody screening panel that precludes the identification of underlying alloantibodies or indicates the presence of autoantibody
Device: INTERCEPT, Device: Control
Anemia
INTERCEPT, Red Blood Cells, RBC, Pathogen Inactivation, Cerus, Pathogen Reduction, Cardiovascular surgery
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, Brian.Morfin@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 Assess the Efficacy, Safety, and Tolerability of Oral LPCN 1148 in Male Subjects With Cirrhosis of the Liver and Sarcopenia
This is a randomized, double-blind, placebo-controlled study to assess the efficacy, safety, and tolerability of LPCN 1148 in men with cirrhosis of the liver and sarcopenia.
Call 214-648-5005
studyfinder@utsouthwestern.edu, NAHID.ATTAR@UTSouthwestern.edu
William Lee
Male
18 Years and over
Phase 2
NCT04874350
STU-2021-1027
Inclusion Criteria:
• Male ≥ 18 years old
• Currently listed, on the liver transplant waitlist for cirrhosis secondary to Hepatitis B or C infection, Alcoholic Liver Disease (ALD), or Non-Alcoholic Steatohepatitis (NASH)
• Evidence of sarcopenia with appropriate cutoff recommended by clinical guidance
Exclusion Criteria:
• Suspected or proven hepatocellular carcinoma (HCC)
• History of current or suspected prostate or breast cancer
• History of malignancies other than prostate, breast, or HCC, unless successfully treated with curative intent and believed to be cured (defined as complete remission lasting at least 5 years)
• History of uncontrolled or recurrent portal hypertensive bleeding, including uncontrolled or recurrent bleeding from varices, gastropathy, colopathy, or hemorrhoidal bleeding.
• History or current thrombosis (including portal vein thrombosis), thromboembolism, or treatment for portal vein thrombosis
• History of hemochromatosis
• History of hypercoagulable state (e.g. Factor V Leiden deficiency, protein C deficiency, protein S deficiency, anti-thrombin III deficiency, or the presence of lupus anticoagulant)
• Prior history of complications of ascites including:
• Spontaneous bacterial peritonitis
• Hepatic hydrothorax
• MELD score > 25
• Abnormal lab value in serum chemistry, hematology, or urinalysis that the PI considers clinically significant, including but not limited to:
• PSA > 4 ng/mL
• Polycythemia (Hematocrit >54%) or history of polycythemia
• ALT or AST > 5x ULN
• ALP > 1.5x ULN
• Platelet count < 30,000/mL
• EGFR < 60 mL/min/1.73 m2
• Total bilirubin > 3.0 mg/dL
• Serum albumin < 2.8 g/dL
• INR > 2.3 or PT prolongation > 6.0 seconds
• Subjects with PSA between 2.5 ng/mL and 4 ng/mL are excluded only if any of the below criteria are met at baseline:
• Hematocrit > 48%
• I-PSS > 19
• Any irregularity found on digital rectal examination of the prostate
• Subjects with PSA > 3 ng/mL are excluded only if any of the below criteria are met at baseline:
• Subject is African American
• Subject has a first-degree relative who has a history of prostate cancer
• Hematocrit > 48%
• I-PSS > 19
• Any irregularity found on digital rectal examination of the prostate
• Clinically significant abnormal prostate digital rectal examination (DRE) in the opinion of the PI, with DRE screening initiated at International Prostate Symptom Score (I-PSS) > 19
• History of bariatric surgery
• History of stroke or myocardial infarction within the past 5 years
• Known positivity for Human Immunodeficiency Virus (HIV) infection
• Acute liver failure as the indication for addition to the liver transplant waitlist
• Estimated life expectancy less than 3 months or expected to undergo liver transplant within 3 months
• Known heart failure of New York Heart Association class III or IV
• Evidence of severe encephalopathy or encephalopathy that is not controlled despite adequate medical therapy
• History of prior organ transplant
• History of Fontan physiology
• History of pulmonary embolus
• Porto-pulmonary hypertension
• Hepatopulmonary syndrome
• Uncontrolled epilepsy or migraine
• Active substance abuse or dependency extending to within the previous 6 months
• History of significant sensitivity or allergy to testosterone, or product excipients.
• Use of known strong inhibitors (e.g., ketoconazole) or inducers (e.g., dexamethasone, phenytoin, rifampin, carbamazepine) of cytochrome P450 3A (CYP3A) within 30 days prior to study drug administration and through the end of the study
• Subjects who are currently receiving any androgens (testosterone or other androgens or androgen-containing supplements) and are unwilling to washout prior to screening a. Washout: 12 weeks following long-acting intramuscular androgen injections; 4 weeks following topical or buccal androgens; 3 weeks following oral androgens
• Uncontrolled hypertension (>160/90 mmHg despite treatment)
• Uncontrolled obstructive sleep apnea
• Use of any investigational drug within 5 half-lives of the last dose or in the past 6 months prior to Study Day -2 without medical monitor and/or Sponsor approval
• Subject who is not willing to use adequate contraception for the duration of the study
• Any other condition, which in the opinion of the investigator would impede compliance to the study protocol (including diet, exercise, and alcohol abstinence) or hinder completion of the study
• Failure to give informed consent
Drug: LPCN 1148, Drug: Placebo
Liver Cirrhosis, Sarcopenia, Liver
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
Late Onset Alzheimer's Disease (LOAD)
The goal of this study is to is to focus on the genetic influences on Alzheimer's Disease (AD) risk. The investigators are looking for families and/or individuals (affected or unaffected) of any ethic background (African American, Caucasian, and Hispanics) with a family history of AD and willing to participate.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kimberly.Martinez@UTSouthwestern.edu
Ihab Hajjar
All
55 Years and over
NCT05010603
STU 082012-046
Inclusion Criteria:
• Established diagnosis of definite or probable AD or have a diagnosis of a related neurodegenerative disorder such as Frontotemporal Dementia (FTD) or Lewy Body Dementia (LBD) (will also recruit sporadic FTD and LBD) cases.
• a living sibling with probable or possible AD;
• a third living relative affected with AD (onset age 50 or older) or unaffected (60 or older);
• participants in the proband's generation with an identified companion serving as an informant;
• participants who have capacity to consent or participants lacking capacity to consent with a surrogate/proxy in place to provide consent.
Exclusion Criteria:
• failure to identify an appropriate informant;
• uncertainty of the clinical diagnosis of Alzheimer's disease or other related disorder;
• discovery of additional diagnosis that could account for the clinical manifestations;
• unwillingness to participate;
• failure to identify a living sibling with AD or other related disorder (except in the cases of sporadic FTD and sporadic LBD);
• participants lacking the capacity to consent who do not have a surrogate or proxy or next of kin to provide consent.
Genetic: Blood Draw, Other: Late Onset Alzheimer's Disease (LOAD) Neuropsychological Battery Test
Alzheimer Disease
Genetic testing, Genetic influence
Two-Part Study for Dose Determination of SRP-5051 (Vesleteplirsen) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)
This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of SRP-5051 (vesleteplirsen) at MAD levels to determine doses to be administered in Part B, and 2) Part B will be conducted to further evaluate the SRP-5051 doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Kristy.Riddle@UTSouthwestern.edu
Susan Iannaccone
Male
7 Years to 21 Years old
Phase 2
NCT04004065
STU-2021-1143
Inclusion Criteria for participants previously treated with SRP-5051:
• Has received prior SRP-5051 treatment in Part A of this study or in Study 5051-102 Exclusion Criteria for participants previously treated with SRP-5051 and new participants enrolling into Part B:
• Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or any other condition that, in the Investigator's opinion, could interfere with participation in the trial. Inclusion Criteria for treatment-naïve participants enrolling into Part B:
• Has a genetic diagnosis of Duchenne muscular dystrophy (DMD) and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
• Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight), or has not received corticosteroids for at least 12 weeks prior to study drug administration.
• Has stable pulmonary function (forced vital capacity [FVC] ≥40% of predicted and no requirement for nocturnal ventilation). Exclusion Criteria for treatment-naive participants enrolling into Part B:
• History of hypomagnesemia within 12 weeks prior to Screening.
• Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.
• Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
• Has a left ventricular ejection fraction (LVEF) <40.0% based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening Visit.
• Treatment with any exon 51-skipping therapy within 4 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time. Other inclusion/exclusion criteria apply.
• Has received prior SRP-5051 treatment in Part A of this study or in Study 5051-102 Exclusion Criteria for participants previously treated with SRP-5051 and new participants enrolling into Part B:
• Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or any other condition that, in the Investigator's opinion, could interfere with participation in the trial. Inclusion Criteria for treatment-naïve participants enrolling into Part B:
• Has a genetic diagnosis of Duchenne muscular dystrophy (DMD) and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
• Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight), or has not received corticosteroids for at least 12 weeks prior to study drug administration.
• Has stable pulmonary function (forced vital capacity [FVC] ≥40% of predicted and no requirement for nocturnal ventilation). Exclusion Criteria for treatment-naive participants enrolling into Part B:
• History of hypomagnesemia within 12 weeks prior to Screening.
• Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.
• Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
• Has a left ventricular ejection fraction (LVEF) <40.0% based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening Visit.
• Treatment with any exon 51-skipping therapy within 4 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time. Other inclusion/exclusion criteria apply.
Drug: SRP-5051
Duchenne Muscular Dystrophy
DMD, Duchenne, Dystrophy, Dystrophin, Exon Skipping, Ambulatory, Duchenne Muscular Dystrophy, Exon 51, Nonambulatory, Pediatric, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)
Children’s Health
A Study to Assess the Efficacy and Safety of Namilumab in Participants With Chronic Pulmonary Sarcoidosis (RESOLVE-Lung)
This is a randomized, double-blind, placebo-controlled study with an open-label extension (OLE).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Fabiola.Gianella@UTSouthwestern.edu
Connie Hsia
All
18 Years and over
Phase 2
NCT05314517
STU-2022-0414
Inclusion Criteria :
• Male or female age ≥18 years
• Able and willing to provide written informed consent, which includes compliance with study requirements and restrictions listed in the consent form
• Greater than or equal to 6-month history of documented sarcoidosis including histological confirmation in the subject's medical records
• Symptomatic as indicated by Medical Research Council Dyspnea scale >1 (i.e., Grade 2 or more) in the prior 6 months
• Body Mass Index (BMI) <40 kg/m2 at Screening
• Vaccinations for COVID-19 with completion of the primary series at least 2 weeks prior to randomization Exclusion Criteria
• Hospitalized for any respiratory illness <30 days prior to Screening
• Greater than or equal to 20% fibrosis as indicated on HRCT-scan assessed by central read prior to randomization
• Hemoglobin <9.5 g/dL
• Participation in another interventional clinical trial (IP/Device) within 6 months prior to Screening
• ECG abnormalities that warrant further clinical investigation or management at Screening
• Systolic blood pressure (SBP) <90 or >180mm Hg; Diastolic blood pressure (DBP) <60
• Has documented laboratory-confirmed SARS-CoV-2 infection as determined by polymerase chain reaction (PCR) or other approved clinical testing <3 months prior to randomization
• Other significant pulmonary disease likely to interfere with the primary endpoint
• Females who are pregnant or breastfeeding or intend to be during the course of the study
• Any other acute or chronic medical condition, psychiatric condition, or laboratory abnormality, that in the judgment of the Investigator or Sponsor, may increase the risk associated with study participation or investigational product administration, or may interfere with the interpretation of study results, and would make the participant inappropriate for entry into this study Other protocol-defined inclusion/exclusion criteria may apply.
• Male or female age ≥18 years
• Able and willing to provide written informed consent, which includes compliance with study requirements and restrictions listed in the consent form
• Greater than or equal to 6-month history of documented sarcoidosis including histological confirmation in the subject's medical records
• Symptomatic as indicated by Medical Research Council Dyspnea scale >1 (i.e., Grade 2 or more) in the prior 6 months
• Body Mass Index (BMI) <40 kg/m2 at Screening
• Vaccinations for COVID-19 with completion of the primary series at least 2 weeks prior to randomization Exclusion Criteria
• Hospitalized for any respiratory illness <30 days prior to Screening
• Greater than or equal to 20% fibrosis as indicated on HRCT-scan assessed by central read prior to randomization
• Hemoglobin <9.5 g/dL
• Participation in another interventional clinical trial (IP/Device) within 6 months prior to Screening
• ECG abnormalities that warrant further clinical investigation or management at Screening
• Systolic blood pressure (SBP) <90 or >180mm Hg; Diastolic blood pressure (DBP) <60
• Has documented laboratory-confirmed SARS-CoV-2 infection as determined by polymerase chain reaction (PCR) or other approved clinical testing <3 months prior to randomization
• Other significant pulmonary disease likely to interfere with the primary endpoint
• Females who are pregnant or breastfeeding or intend to be during the course of the study
• Any other acute or chronic medical condition, psychiatric condition, or laboratory abnormality, that in the judgment of the Investigator or Sponsor, may increase the risk associated with study participation or investigational product administration, or may interfere with the interpretation of study results, and would make the participant inappropriate for entry into this study Other protocol-defined inclusion/exclusion criteria may apply.
Drug: Namilumab, Drug: Placebo
Sarcoidosis, Pulmonary, Lung/Thoracic
UT Southwestern
Developing an Intervention to Promote Lethal Means Safety in Suicidal Adolescents
Study Objective: to develop a phone-based intervention to aid parents of suicidal adolescents to adhere to lethal means safety. Lethal means counseling is the practice of educating patients and their families about limiting access to items that can be used to attempt suicide. Though lethal means counseling is standard practice in treating suicidal patients, there is little experimental literature related to its utilization across different providers and its efficacy. Further, there is a significant gap in the literature on lethal means counseling as it relates to adolescents. The proposed project will investigate a novel text message-based intervention aimed to promote lethal means safety.
Call 214-648-5005
studyfinder@utsouthwestern.edu, Rebecca.Wildman@UTSouthwestern.edu
Beth Kennard
All
12 Years to 100 Years old
Phase 2
NCT05423483
STU-2022-0396
Inclusion Criteria:
• Proficiency in spoken and written English
• Own cell phone that has internet capabilities
• Adolescent participants are patient's at Children's Health SPARC IOP (ages 12-17)
• Adult participants are parents and/or legal guardians of adolescent participants (ages 18+)
Exclusion Criteria:
• Non-English speaking
Behavioral: Safe Home Text Message Reminders
Suicide
Children’s Health
Open-label Extension Study of GB002 in Adult Subjects With Pulmonary Arterial Hypertension (PAH)
This open-label extension study will evaluate the long-term effects of GB002 (seralutinib) in subjects who previously participated in a GB002 PAH study.
Call 214-648-5005
studyfinder@utsouthwestern.edu, tatyana.ganz@utsouthwestern.edu
Kelly Chin
All
18 Years to 80 Years old
Phase 2
NCT04816604
STU-2022-0775
Inclusion Criteria:
Type of Subject and Disease Characteristics
• Subjects must have completed a prior GB002 PAH study and, in the opinion of the Investigator and Sponsor, have been compliant with study procedures and have completed treatment with IP through parent study end-of-treatment (EOT) visit.
• Treatment with standard of care PAH disease-specific background therapies (stable dose). Informed Consent
• Review and signature of an IRB-approved informed consent form.
Exclusion Criteria:
Medical Conditions
• Persistent and clinically significant systemic hypertension or hypotension.
• Interval history of newly developed left-sided heart disease.
• Potentially life-threatening cardiac arrhythmia with an ongoing risk.
• Uncontrolled bacterial, viral, or fungal infections which require systemic therapy.
• Other severe acute or chronic medical or laboratory abnormality that may increase the risk associated with study participation or GB002 administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the subject inappropriate for entry into this study.
• History of portopulmonary hypertension or portal hypertension due to cirrhosis classified as Child-Pugh Class A or higher.
• Subjects with a history of severe milk protein allergy. In addition, subjects with known intolerance or hypersensitivity to lactose who, in the opinion of the investigator, may experience severe symptoms following the ingestion of lactose.
• Current use of inhaled tobacco and/or inhaled marijuana. Ingestible or topical marijuana is allowed, per local restrictions and regulations.
• Current alcohol use disorder as defined by DSM-5, and/or history of current utilization of drugs of abuse (amphetamines, methamphetamines, cocaine, phencyclidine [PCP]).
• Have any other condition or reason that, in the opinion of the Investigator and/or the Sponsor's Medical Monitor (or designee), would prohibit the subject from participating in the study. Diagnostic Assessments
• Chronic renal insufficiency
• Hemoglobin (Hgb) concentration <8.5 g/dL.
• Absolute neutrophil count (ANC) < 1x 10^9/L.
• Platelet count <50 x 10^9/L. Prior Therapy
• Use of inhaled prostanoids.
• Chronic use of oral anticoagulants (ie, vitamin K antagonist such as warfarin or novel oral anticoagulant [NOAC]/direct oral anticoagulant [DOAC]).
• Chronic use of any prohibited medication. NOTE: Additional inclusion/exclusion criteria may apply, per protocol.
Drug: GB002 (seralutinib), Device: Generic Dry Powder Inhaler
Pulmonary Arterial Hypertension, Lung/Thoracic
seralutinib
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
Comparison of Methods of Pulmonary Blood Flow Augmentation in Neonates: Shunt Versus Stent (The COMPASS Trial) (COMPASS)
COMPASS is a prospective multicenter randomized interventional trial. Participants with ductal-dependent pulmonary blood flow will be randomized to receive either a systemic-to-pulmonary artery shunt or ductal artery stent. Block randomization will be performed by center and by single vs. two ventricle status. Participants will be followed through the first year of life.
Call 214-648-5005
studyfinder@utsouthwestern.edu, madison.munson@childrens.com
Surendranath Veeram Reddy
All
1 Day to 30 Days old
N/A
NCT05268094
STU-2023-0190
Inclusion Criteria:
• Neonates with Congenital Heart Disease (CHD) and ductal-dependent pulmonary blood flow requiring only a stable source of pulmonary blood flow as the initial palliation, for whom the clinical decision is made at the enrolling center that this is best achieved by either DAS or SPS.
• Age ≤ 30 days at time of index procedure (DAS or SPS).
Exclusion Criteria:
• 1. Any patient for whom the clinical decision at the enrolling center is that an initial intervention other than DAS or SPS is indicated (e.g., Right Ventricle-Pulmonary Artery (RV-PA) conduit, Right Ventricular Outflow Tract (RVOT) stent, primary complete anatomic repair, etc.).
• Pulmonary Atresia with Intact Ventricular Septum (PA/IVS) where Right Ventricle (RV) decompression is planned.
• Presence of MAPCAs: defined as an aortopulmonary collateral that is expected to require unifocalization.
• Non-confluent Pulmonary Arteries (i.e., isolated Pulmonary Artery (PA) of ductal origin).
• Acutely jeopardized branch Pulmonary Arteries (>75% narrowing of proximal PA based on screening cross sectional imaging [Computed Tomography Angiography (CTA) or cardiovascular Magnetic Resonance (cMR)]).
• Bilateral Patent Ductus Arteriosis (PDA). 7. Patient who, at the time of enrollment, is deemed not to be a candidate for eventual Glenn or Complete Surgical Repair (CSR) for any reason.
• Birth weight <2.0 kg. 9. Gestational age <34 weeks at birth. 10. Patient for whom additional intervention is expected concomitant with, or prior to, DAS or SPS (e.g., atrial septostomy, aortic arch intervention, or RV outflow tract intervention) - except for branch PA arterioplasty or stent/balloon angioplasty.
• Major co-morbidities which, in the opinion of the investigator, would negatively alter expected 1-year survival (e.g., intracranial hemorrhage, renal failure, etc.).
• Specific known genetic anomaly which, in the opinion of the investigator, would be expected to significantly alter clinical course in the first year of life (e.g., Trisomy 13/18, CHARGE, VACTERL).
• Patient who does not plan to return to the enrolling center or another participating center for Glenn/CSR.
Device: Ductal Arterial Stent, Procedure: Systemic-to-Pulmonary Artery Shunt
Congenital Heart Disease in Children
Congenital Heart Disease, Ductal Dependent Pulmonary Blood Flow, Ductal Artery Shunt, Systemic-to-Pulmonary Artery Shunt
Children’s Health
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
A Study of CIN-107 in Adults With Primary Aldosteronism
This is a multicenter, open-label study in adult patients with PA to evaluate the effectiveness and safety of CIN-107 after up to 12 weeks of treatment, at doses from 2 to 8 mg per day, for the management of blood pressure in patients with primary aldosteronism (PA).
Call 214-648-5005
studyfinder@utsouthwestern.edu, Natalie.Booker@UTSouthwestern.edu
Oksana Hamidi
All
18 Years and over
Phase 2
NCT04605549
STU-2021-0884
Inclusion Criteria:
• Have been diagnosed with PA.
• Are taking mineralocorticoid receptor antagonist (MRA) to control BP; or are newly diagnosed with PA and have not started MRA treatment.
• Are willing and able to cease dosing of MRA for up to 4 weeks in patients taking MRA.
• Are willing to be compliant with the contraception and reproduction restrictions of the study.
• Have increased SBP by ≥ 20 mmHg or have SBP ≥ 160 mmHg after dosing of MRA treatment is ceased for up to 4 weeks duration, or have SBP ≥ 150 mmHg for patients who are newly diagnosed with PA and have not taken an MRA in the past 12 weeks.
Exclusion Criteria:
• At Screening Visit, have a single occurrence of mean seated SBP > 180 mmHg or DBP > 110 mmHg if not taking an MRA; or have a mean seated SBP ≥ 160 mmHg or DBP ≥ 100 mmHg if currently taking an MRA.
• Have a body mass index > 45 kg/m2.
• Have had a previous surgical intervention for an adrenal adenoma or have a planned adrenal carcinoma, adrenalectomy, renal nerve denervation, or adrenal ablative procedure during the course of the study.
• Have a documented estimated glomerular filtration rate < 45 mL/min/1.73 m2.
• Have a planned dialysis, kidney transplantation or any major surgical procedure during the course of the study.
• Have known documented New York Heart Association class III or IV chronic heart failure.
• Have had a stroke, transient ischemic attack, hypertensive encephalopathy, acute coronary syndrome, or hospitalization for heart failure within 6 months before the Screening Visit.
• Have known current severe left ventricular outflow obstruction.
• Have had major cardiac surgery within 6 months before the Screening Visit.
• Have a history of, or currently experiencing, clinically significant arrhythmias.
• Have had a prior solid organ transplant or cell transplant.
• Are positive for HIV antibody, hepatitis C virus RNA, or hepatitis B surface antigen.
• Have typical consumption of > 14 alcoholic drinks weekly.
Drug: CIN-107 2 mg dosing, Drug: CIN-107 4 mg dosing, Drug: CIN-107 8 mg dosing
Primary Aldosteronism, Hyperaldosteronism, Other Endocrine System
Primary Aldosteronism
UT Southwestern
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