StudyFinder



Search Results

Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.

168 Study Matches

A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, and LGMD2C/R5, ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), or Limb-girdle muscular dystrophy type 2C (LGMD2C/R5). These enrolled participants will be followed to evaluate mobility and pulmonary function for up to 3 years after enrollment. Additional participant data will be collected from the time the individual began experiencing LGMD symptoms to the present.

Call 214-648-5005
studyfinder@utsouthwestern.edu, Elaine.Most@UTSouthwestern.edu

Kaitlin Batley
162753
All
4 Years and over
This study is NOT accepting healthy volunteers
NCT04475926
STU-2020-0865
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Male or female participant ≥ 4 years of age with confirmed genetic diagnosis of LGMD2E/R4, LGMD2D/R3, or LGMD2C/R5.
Exclusion Criteria:

• Demonstrates cognitive delay or impairment that could confound motor development, in the opinion of the Investigator.
• Has a medical condition, in the opinion of the Investigator, that might compromise participants ability to comply with study requirements.
• Is participating in other interventional study(ies) at the time of enrollment in this study.
Limb-girdle Muscular Dystrophy
North Star Assessment for Dysferlinopathy (NSAD), Performance of Upper Limb (PUL), Pulmonary function tests (PFTs), Ambulatory, Non-Ambulatory, Limb-girdle, LGMD, sarcoglycanopathy, β -sarcoglycan, Muscular Dystrophy, α -sarcoglycan, γ -sarcoglycan, LGMD-2D/R3, LGMD-2E/R4, LGMD-2C/R5, Clinical Outcomes Assessment
UT Southwestern; Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

RAndomized Therapy In Status Epilepticus (RAISE)

This study will evaluate the effectiveness and safety of an investigational product, IV ganaxolone, to treat subjects with status epilepticus.

Call 214-648-5005
studyfinder@utsouthwestern.edu, Karla.CastroOchoa@UTSouthwestern.edu

Rana Said
66861
All
12 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04391569
STU-2020-0740
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• clinical and/or electrographic seizures
Exclusion Criteria:

• life expectancy of less than 24 hours
• anoxic brain injury or an uncontrolled metabolic condition as primary cause of SE
• treatment of current SE episode with IV anesthetics
Drug: Ganaxolone, Drug: Placebo
Epilepsy, Status Epilepticus, Convulsive Status EPILEPTICUS, Non-Convulsive Status Epilepticus
seizure
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

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
124198
All
10 Years to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04801771
STU-2021-0286
Show full eligibility criteria
Hide eligibility criteria
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
Obstructive Sleep Apnea, Down Syndrome, Ear, Nose, Throat
Hypoglossal nerve stimulation
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

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@childrens.com

Thomas Zellers
18301
All
Not specified
This study is NOT accepting healthy volunteers
NCT04860765
STU-2021-0535
Show full eligibility criteria
Hide eligibility criteria
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
Pulmonary Valve Insufficiency, Cardiovascular, Complex Congenital Heart Defect, Dysfunctional RVOT Conduit, Pulmonary Valve Degeneration, Pulmonary Valve, Obstruction
Transcatheter pulmonary valve replacement, Transcatheter pulmonary valve implantation, SAPIEN 3
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

Call 214-648-5005
studyfinder@utsouthwestern.edu, Kristy.Riddle@UTSouthwestern.edu

Susan Iannaccone
13463
Male
4 Years to 7 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT05096221
STU-2020-0932
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Is ambulatory and from 4 to under 8 years of age at time of randomization.
• Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
• Ability to cooperate with motor assessment testing.
• Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
• rAAVrh74 antibody titers are not elevated as per protocol-specified requirements.
Exclusion Criteria:

• Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
• Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
• Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer. Other inclusion or exclusion criteria could apply.
Genetic: SRP-9001, Genetic: Placebo
Duchenne Muscular Dystrophy, Other
Muscular Dystrophies, Duchenne Muscular Dystrophy, DMD, North Star Ambulatory Assessment (NSAA), Ambulatory, Pediatric, Gene-Delivery
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

A Study to Test if Fremanezumab is Effective in Preventing Episodic 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 episodic migraine (EM). Secondary objectives are to further demonstrate the efficacy of Fremanezumab as compared to placebo for the preventive treatment of EM, to evaluate the safety and tolerability of Fremanezumab in the preventive treatment of EM 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 up to 36 months.

Call 214-648-5005
studyfinder@utsouthwestern.edu, Kiley.Poppino@UTSouthwestern.edu

Deryk Walsh
94400
All
6 Years to 17 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04458857
STU-2020-0990
Show full eligibility criteria
Hide eligibility criteria
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 ≤=14 headache 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 or parent/caregiver maintain a prospectively collected headache diary
• 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 or 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, Brain and Nervous System, Cardiovascular
episodic migraine
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

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
94400
All
6 Years to 17 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04530110
STU-2020-1130
Show full eligibility criteria
Hide eligibility criteria
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
I'm interested
Share via email
See this study on ClinicalTrials.gov

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
94400
All
6 Years to 17 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04464707
STU-2020-0683
Show full eligibility criteria
Hide eligibility criteria
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
I'm interested
Share via email
See this study on ClinicalTrials.gov

A Study of Mavorixafor in Participants With Severe Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders

This Phase 1b study will determine the safety and tolerability of mavorixafor and its effect on absolute neutrophil count.

Call 214-648-5005
studyfinder@utsouthwestern.edu, Laurie.Rodgers-Augustyniak@childrens.com

Kathryn Dickerson
156007
All
12 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT04154488
STU-2021-1051
Show full eligibility criteria
Hide eligibility criteria
Key
Inclusion Criteria:

• For all participants:
• Sign the informed consent form (ICF) and be willing and able to comply with the protocol.
• Weigh ≥15 kg
• Agree to use a highly effective form of contraception.
• Participants may be eligible for the study whether they are on or off G-CSF treatment.
• Participants who are on granulocyte-colony stimulating factor (G-CSF) must be on a stable dose for at least 14 days prior to enrollment and for the duration of the study.
• Participants who are not on G-CSF must be off for at least 14 days prior to enrollment and for the duration of the study.
• Have an absolute neutrophil count (ANC) < 1000 cells/µL at the screening and baseline visits. Participants on G-CSF are allowed if their screening and baseline ANC is ≥1000 cells/microliter (uL). Participants with cyclical neutropenia are required to have ANC <1000 cells/ µL only at the baseline visit.
• Have been diagnosed with chronic neutropenia for at least 6 months prior to screening that is not attributable to medications, active or recent (within 3 months) infections, or malignant cause. Key
Exclusion Criteria:

• Known systemic hypersensitivity to the mavorixafor drug substance or its inactive ingredients.
• Is pregnant or nursing.
• Known history of a positive serology or viral load for human immunodeficiency virus (HIV) or a known history of acquired immune deficiency syndrome.
• At screening, has laboratory test results meeting one or more of the following criteria:
• Positive hepatitis C virus (HCV) antibodies with confirmation by HCV-ribonucleic acid polymerase chain reaction reflex testing.
• Positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb). Note: If a participant tests negative for HBsAg but positive for HBcAb, the participant would be considered eligible if the participant tests positive for antibody to HBsAg reflex testing.
• At screening, has laboratory test results meeting one or more of the following criteria:
• Hemoglobin <9.0 grams/deciliter (g/dL).
• Platelet count <30,000/μL with prior or current history of clinical bleeding events or malignancy or other bone marrow pathologies (e.g., myelodysplastic syndrome).
• Mild/moderate renal impairment, defined as 30 to 60 milliliter/minute (mL/min) estimated glomerular filtration rate.
• Serum aspartate transaminase >2.5 * upper limit of normal (ULN).
• Serum alanine transaminase >2.5 * ULN.
• Total bilirubin >1.5 * ULN (unless due to Gilbert's syndrome, in which case total bilirubin greater than or equal to (≥) 3.0 * ULN and direct bilirubin >1.5 * ULN).
• Within 2 weeks before Day 1, received any of the following treatments:
• Glucocorticoids (>5 mg prednisone equivalent per day).
• Medication prohibited based on cytochrome P450 (CYP) and/or transporter-based (such as, P-glycoprotein ([P-gp]) potential for drug-drug interaction.
• At the planned initiation of study drug, has an infection requiring use of antibiotics (systemic or inhaled) or took systemic antibiotics within 4 weeks before Day 1.
• Has any other medical or personal condition that, in the opinion of the Investigator, may potentially compromise the safety or compliance of the participant, or may preclude the participant's successful completion of the clinical study.
• Inability to ingest capsules of study drug as presented.
• Has a history of hematologic malignancy.
• Diagnosed or have suspected congenital long QT syndrome. Any history of clinically significant ventricular arrhythmias (such as ventricular tachycardia, ventricular fibrillation, or torsades de pointes); any history of arrhythmia will be discussed with the sponsor's medical monitor before participant's entry into the study.
• Prolonged corrected QT interval using Fridericia's formula on pre-entry electrocardiogram (ECG) (>450 milliseconds [ms]).
Drug: Mavorixafor
Neutropenia
Chronic congenital neutropenia, Chronic idiopathic neutropenia, SCN, CIN, CXCR4, Mavorixafor, Neutropenia glycogen storage disease type 1b, GSD1b, G6PC3, SLC37A4, GATA2
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

A Research Study to See How Well the New Weekly Medicine IcoSema, Which is a Combination of Insulin Icodec and Semaglutide, Controls Blood Sugar Level in People With Type 2 Diabetes Compared to Weekly Insulin Icodec (COMBINE 1)

This study will compare the new medicine IcoSema, which is a combination of insulin icodec and semaglutide, taken once a week, to insulin icodec taken once a week in people with type 2 diabetes. The study will look at how well IcoSema controls blood sugar level in people with type 2 diabetes compared to insulin icodec. Participants will either get IcoSema or insulin icodec. Which treatment participants get is decided by chance. IcoSema and insulin icodec are both new medicines that doctors cannot prescribe. Participants will get IcoSema or insulin icodec, which participants must inject once a week with a pen, which has a small needle, in a skin fold in the thigh, upper arm, or stomach. The study will last for about 1 year and 1 month. Participants will have 21 clinic visits, 31 phone/video calls with the study doctor, and 4 contacts with the site that can either be clinic visits or phone/video calls At 11 clinic visits participants will have blood samples taken. At 7 clinic visits participants cannot eat or drink (except for water) for 8 hours before the visit. Women cannot take part if pregnant, breast-feeding or plan to get pregnant during the study period. Not applicable for China: Participants will be asked to wear a sensor that measures their blood sugar level all the time during a 5 week period at the end of the study.

Call 214-648-5005
studyfinder@utsouthwestern.edu, Saubia.Mian@UTSouthwestern.edu

Ildiko Lingvay
55880
All
Not specified
Phase 3
This study is NOT accepting healthy volunteers
NCT05352815
STU-2022-0220
Show full eligibility criteria
Hide eligibility criteria
Key inclusion criteria
• Male or female and age above or equal to 18 years at the time of signing informed consent.
• Diagnosed with type 2 diabetes mellitus 180 days or more before screening.
• HbA1c of 7.0 10.0% (53.0 85.8 mmol/mol) (both inclusive) as assessed by central laboratory on the day of screening.
• Treated with once daily or twice daily basal insulin (neutral protamine hagedorn insulin, insulin degludec, insulin detemir, insulin glargine 100 units/mL, or insulin glargine 300 units/mL) 20- 80 units/day for 90 days or more before screening. Short term bolus insulin treatment for a maximum of 14 days before screening is allowed, as is prior insulin treatment for gestational diabetes. The treatment can be with or without any of the following anti diabetic drugs with stable doses for 90 days or more before screening:
• Metformin
• Sulfonylureas (a)
• Meglitinides (glinides) (a)
• DPP 4 inhibitors (a)
• Sodium glucose co transporter 2 inhibitors
• Alpha glucosidase inhibitors
• Thiazolidinediones
• Marketed oral combination products only including the products listed above.
• Body mass index (BMI) below or equal to 40.0 kg/m^2. (a) Sulfonylureas, meglitinides (glinides) and DPP 4 inhibitors must be discontinued at randomisation. Key exclusion criteria
• Female who is pregnant, breast-feeding or intends to become pregnant or is of childbearing potential and not using a highly effective contraceptive method.
• Anticipated initiation or change in concomitant medication (for more than 14 consecutive days) known to affect weight or glucose metabolism (e.g. treatment with orlistat, thyroid hormones, or systemic corticosteroids).
• Treatment with any medication for the indication of diabetes or obesity other than stated in the inclusion criteria within 90 days before screening.
• Any episodes (as declared by the participant or in the medical records.) of diabetic ketoacidosis within 90 days before screening.
• Presence or history of pancreatitis (acute or chronic) within 180 days before screening.
• Any of the following: Myocardial infarction, stroke, hospitalization for unstable angina pectoris or transient ischaemic attack within 180 days before screening.
• Chronic heart failure classified as being in New York Heart Association Class IV at screening.
• Recurrent severe hypoglycaemic episodes within the last year (12 months) as judged by the investigator.
• Uncontrolled and potentially unstable diabetic retinopathy or maculopathy. Verified by a fundus examination performed within the past 90 days before screening or in the period between screening and randomisation. Pharmacological pupil dilation is a requirement unless using a digital fundus photography camera specified for non-dilated examination.
Drug: IcoSema, Drug: Insulin icodec
Diabetes Mellitus, Type 2
UT Southwestern
I'm interested
Share via email
See this study on ClinicalTrials.gov

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
48082
All
11 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03459287
STU-2021-0761
Show full eligibility criteria
Hide eligibility criteria
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
I'm interested
Share via email
See this study on ClinicalTrials.gov

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, Alexandria.Silver@UTSouthwestern.edu

Susan Iannaccone
13463
Male
7 Years to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04004065
STU-2021-1143
Show full eligibility criteria
Hide eligibility criteria
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.
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
I'm interested
Share via email
See this study on ClinicalTrials.gov

Posoleucel (ALVR105) for the Treatment of Adenovirus Infection in Pediatric and Adult Participants Receiving Standard of Care Following Allogeneic Hematopoietic Cell Transplantation

This study will assess the safety and efficacy of Posoleucel for the treatment of adenovirus (AdV) infection in pediatric and adult allo-HCT recipients receiving standard of care (SoC).

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Yeh-Chung Chang
118958
All
1 Year and over
Phase 3
This study is NOT accepting healthy volunteers
NCT05179057
STU-2021-1116
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Male or female >1 year of age.
• Has undergone allogeneic cell transplantation ≥21 days prior to randomization and demonstrated engraftment with an absolute neutrophil count >500/mm^3, AND has one of the following:
• AdV viremia DNA ≥10,000 copies/mL at screening, OR
• Two consecutive and rising AdV viremia DNA results of ≥1,000 copies/mL at screening, AND
• has absolute lymphocyte count <180/mm3, OR
• has received T cell depletion.
• Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and refrain from donating sperm or eggs for at least 90 days after treatment completion.
• Willing and able to provide signed informed consent.
• Has an HLA type matching with at least 1 suitably matched and available posoleucel VST line for infusion.
Exclusion Criteria:

• Grade >2 acute GVHD
• Ongoing therapy with high-dose systemic corticosteroids
• Grade 4 diarrhea
• Uncontrolled viral (other than AdV), bacterial, or fungal infection(s)
• Requirement for fraction of inspired oxygen >0.5 to maintain arterial oxygen saturation >90% (via pulse oximetry) or need for mechanical ventilation.
• Prior therapy with anti-thymocyte globulin, alemtuzumab (Campath®), or other immunosuppressive T cell monoclonal antibodies within 28 days prior to randomization.
• Prior donor lymphocyte infusion or CD34+ stem cell infusion within 21 days prior to randomization.
• Use of vasopressors within 7 days prior to randomization.
• Use of any investigational antiviral agent, including brincidofovir, within 7 days prior to randomization.
• Lactating female unwilling to discontinue nursing prior to randomization.
• Severe allergy to any component of posoleucel or history of severe prior reactions to blood product transfusions.
• Positive for SARS-CoV-2 virus at screening.
Drug: Posoleucel, Drug: Placebo
Adenovirus Infection, Other Hematopoietic
Allogeneic Hematopoietic Cell Transplant, Adenoviremia, Adenovirus, Stem cell transplant, Posoleucel, ALVR105
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

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
13826
All
12 Years to 100 Years old
Phase 2
This study is also accepting healthy volunteers
NCT05423483
STU-2022-0396
Show full eligibility criteria
Hide eligibility criteria
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
I'm interested
Share via email
See this study on ClinicalTrials.gov

Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders (NACHO-COBI)

This is a research study of a drug called cobimetinib in children and adults diagnosed with Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or does not respond to treatment. Cobimetinib blocks activation of a protein called Mitogen-activated protein kinase (MEK) that is part of incorrect growth signals in histiocytosis cells. Four different groups of patients will be enrolled.

Call 833-722-6237
canceranswerline@utsouthwestern.edu

Erin Butler
104034
All
Not specified
Phase 2
This study is NOT accepting healthy volunteers
NCT04079179
STU-2021-0830
Show full eligibility criteria
Hide eligibility criteria
INCLUSION CRITERIA: Age at study entry
• For Group 1: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment
• For Group 2: Participant may be at least 6 months of age at the time of enrollment
• For Group 3: Participant must be at least 6 months of age and less than 21 years of age at the time of enrollment
• For Group 4: Participant must be 21 years of age or older at the time of enrollment
• Participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet which may be taken by mouth or other enteral route such as nasogastric or gastric tube.
• Biopsy proven LCH -AND
• Failure of at least front-line therapy for LCH with evaluable disease. -OR
• Diagnosis of LCH-associated neurodegenerative disease with radiologic or clinical progression within the past 3 months. -OR
• Biopsy proven JXG, ECD, RDD, histiocytic sarcoma, or other histiocytic lesion (newly diagnosed or relapsed/refractory disease) with evaluable active disease. Performance Level: -Karnofsky ≥ 50% for patients > 16 years of age and Lansky ≥ 50% for patients ≤ 16 years of age. Adequate Hematologic Function Defined as:
• ANC ≥ 0.75 x 10^9/L (unsupported/without growth factor stimulant)
• Platelet count ≥ 75 x 10^9/L (unsupported/without transfusion within the past 7 days).
• Patients with marrow disease must have platelet count of >/= 75 x 10^9/L (transfusion support allowed) and must not be refractory to platelet transfusions.
• Hemoglobin ≥ 8 g/dL (unsupported/without transfusion within the past 7 days)
• Patients with marrow disease must have hemoglobin ≥ 8 g/dL (transfusion support allowed). Adequate Renal Function Defined as:
• Calculated creatinine clearance (or radioisotope GFR) ≥ 70 mL/min/1.73m^2 or serum creatinine based on age/gender as follows: Maximum Serum Creatinine (mg/dL) Age 2 to < 6 years: Male 0.8 mg/d, Female 0.8; 6 to < 10 years: Male 1 mg/dL,Female 1; 10 to < 13 years: Male 1.2 mg/dL; Female 1.2; 13 to < 16 years: Male 1.5 mg/dL ; Female 1.4; ≥ 16 years: Male 1.7 mg/dL; Female 1.4; Adequate Liver Function Defined as:
• Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age
• AST and ALT ≤ 3x ULN (≤ 5 x ULN for participants with liver involvement)
• Serum albumin ≥ 2 g/dL. For patients with liver disease caused by histiocytic disorder: • Patients may be enrolled with abnormal bilirubin, AST, ALT and albumin with documentation of histiocytic liver disease. Adequate Cardiac Function Defined as:
• Fractional shortening (FS) of ≥ 30% or ejection fraction of ≥ 50% by echocardiogram at baseline, as determined by echocardiography or multigated acquisition scan (MUGA) within 28 days prior to enrollment. Depending on institutional standard, either FS or LVEF is adequate for enrollment if only one value is measured; if both values are measured, then both values must meet criteria above Pregnancy/Birth Control
• Female patients of childbearing potential require a negative urine or serum pregnancy test for eligibility and again at database registration, if more than 2 weeks has elapsed.
• Female patients of childbearing potential must agree to follow the contraceptive requirements using two forms of effective contraceptive methods for the duration of the study treatment. Male patients with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) must agree to use two forms of effective methods of contraception (one of which must be a barrier method) during the treatment period and for at least 3 months after the last dose of the study drug to avoid pregnancy and/or potential adverse effects on a developing embryo. Agreement to true abstinence (not periodic abstinence or withdrawal method) is an acceptable method of birth control. EXCLUSION CRITERIA:
• Prior and Concomitant Use of Drugs with CYP3A4 inducing/inhibiting activity: Patient taking strong inducers or inhibitors of CYP3A4 within 14 days prior to study enrollment, including but not limited to the following: erythromycin, clarithromycin, ketoconazole, azithromycin, itraconazole, grapefruit juice or St. John's wort.
• Prior Therapy Restrictions Completion of previous chemotherapy, immunotherapy, radiotherapy, or targeted therapy for LCH (or other histiocytic disorder) at least 28 days (except where specified below) prior to study enrollment, with resolution of all associated toxicity to ≤ Grade 1 prior to study enrollment (exception for alopecia and ototoxicity which do not need to be resolved ≤ Grade 1). Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the laboratory eligibility criteria are met, the patient is considered to have recovered adequately.
• Radiation therapy within the 28 days prior to enrollment.
• Any prior treatment with Cobimetinib.
• Treatment with a long-acting hematopoietic growth factor within 14 days prior to initiation of study drug or a short-acting hematopoietic growth factor within 7 days prior to enrollment.
• Treatment with hormonal therapy (except hormone replacement therapy or oral contraceptives), immunotherapy, biologic therapy, investigational therapy, or herbal cancer therapy within 28 days or < 5 half-lives, whichever is longer, prior to study enrollment.
• Treatment with high-dose chemotherapy and stem-cell rescue (autologous stem cell transplant) or allogeneic stem cell transplant within 90 days prior to enrollment. Anti-GVHD agents post-transplant: Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial.
• For patients with brain tumors (intracranial masses), use of anticoagulants within 7 days prior to enrollment.
• Corticosteroid therapy <0.5 mg/kg/day averaged during the month prior to study enrollment is permissible but must be discontinued fourteen (14) days prior to enrollment. Patients with documented brain lesions receiving corticosteroids for management of cerebral edema must be on a stable dose for fourteen (14) days prior to enrollment.
• Patient has received treatment with investigational therapy within 4 weeks prior to initiation of study drug.
• Patients taking anticoagulants or have a pre-existing bleeding disorder unrelated to histiocytic disease.
• Exclusions for other illness
• Other active malignancy or history of secondary malignancy.
• Refractory nausea and vomiting, malabsorption, external biliary shunt
• Infection: Patients who have a known active infection (excluding documented fungal infection of the nail beds) within 28 days prior to enrollment that has not completely resolved.
• Major surgical procedure or significant traumatic injury within 28 days prior to enrollment, or anticipation of need for major surgical procedure during the course of the study. Placement of a vascular access device or minor surgery is permitted within fourteen (14) days prior to study enrollment (provided that the wound has healed).
• History of significant bowel resection that would preclude adequate absorption or other significant malabsorptive disease.
• History of pneumonitis.
• Ophthalmologic considerations: Patients with known significant ophthalmologic conditions or known risk factors for retinal vein occlusion are not eligible. Specifically, patients with a history of retinal vein occlusion (RVO), retinal detachment, retinal pathology on ophthalmologic exam, retinopathy of prematurity, central serous chorioretinopathy (CSSCR), neovascular retinopathy, intraocular pressure > 21 mmHg, and predisposing factors to RVO (e.g., uncontrolled hypertension, diabetes, or hyperlipidemia, coagulopathy) will be excluded. Patients with longstanding and stable ophthalmologic findings secondary to existing conditions are eligible with appropriate written documentation and approval from Study Chair.
• History of solid organ transplantation: Patients who have received a prior solid organ transplantation are not eligible.
• Any other disease, metabolic or psychological dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that in the opinion of the investigator contraindicates use of an investigational drug or places the patient at unacceptable risk from treatment complications.
• History of clinically significant cardiac dysfunction, including the following:
• Clinically significant cardiac arrhythmias including brady-arrhythmias and/or patients who require anti-arrhythmic therapy (with the exception of beta blockers or digoxin). Patients with controlled atrial fibrillation are not excluded.
• Unstable arrhythmia
• Unstable angina, or new-onset angina within 3 months prior to initiation of study treatment
• Symptomatic congestive heart failure, defined as New York Heart Association Class II or higher
• Myocardial infarction within 3 months prior to initiation of study treatment
• Known chronic human immunodeficiency virus (HIV).
• History of Grade ≥ 2 CNS hemorrhage or history of any CNS hemorrhage within 28 days of enrollment.
• Female patients who are pregnant or lactating. Pregnant or lactating women will not be entered on this study because there is no available information regarding human fetal or teratogenic toxicities.
Drug: Cobimetinib
Histiocytic Sarcoma, Juvenile Xanthogranuloma, Brain and Nervous System, Bones and Joints, Liver, Lung/Thoracic, Other Hematopoietic, Langerhan's Cell Histiocytosis, Erdheim-Chester Disease, Rosai Dorfman Disease, Neuro-Degenerative Disease, Histiocytic Disorders, Malignant
Cobimetinib, Langerhans Cell Histiocytosis (LCH)
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

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
109868
All
1 Day to 30 Days old
N/A
This study is NOT accepting healthy volunteers
NCT05268094
STU-2023-0190
Show full eligibility criteria
Hide eligibility criteria
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
I'm interested
Share via email
See this study on ClinicalTrials.gov

Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia (CONnECT)

This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal Hyperplasia.

Call 214-648-5005
studyfinder@utsouthwestern.edu, Michelle.Murphy@UTSouthwestern.edu

Perrin White
17917
All
16 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT05063994
STU-2021-1015
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:

• Male or female participants must be aged 16 years or older at the time of signing the informed consent/assent.
• In participants aged <18 years, height velocity must be less than 2 cm/year in the last year and puberty must be completed (Tanner stage V).
• Participants with known classic CAH due to 21 hydroxylase deficiency diagnosed in childhood with documented (at any time) elevated 17-OHP and with or without elevated A4 and currently treated with hydrocortisone, prednisone, prednisolone or dexamethasone (or a combination of the aforementioned glucocorticoids) and on stable glucocorticoid therapy for a minimum of 3 months.
• Participants who are receiving fludrocortisone must be on a documented stable dose for a minimum of 3 months prior to enrollment and must have stable renin levels at screening.
• Female participants of childbearing potential and all male participants must agree to the use of an accepted method of contraception during the study.
• A female participant is eligible to participate if she is not pregnant, not breastfeeding, and she is either not a woman of childbearing potential (WOCBP) or has a negative pregnancy test at entry into the study. Note: females presenting with oligomenorrhea or amenorrhea who are aged ≤55 years should be considered potentially fertile and therefore should undergo pregnancy testing like all other female participants.
• Capable of giving signed informed consent/assent which includes compliance with requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Exclusion Criteria:

• Clinical or biochemical evidence of hepatic or renal disease e.g. creatinine >2 times the upper limit of normal (ULN) or elevated liver function tests (alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >2 times the ULN).
• History of bilateral adrenalectomy.
• History of malignancy (other than basal cell carcinoma successfully treated >26 weeks prior to entry into the study).
• Participants who have type 1 diabetes or receive regular insulin, have uncontrolled diabetes, or have a screening HbA1c greater than 8%..
• Persistent signs of adrenal insufficiency or the participant does not tolerate treatment at the end of the 4-week run-in period.
• Participants with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the study.
• Participants on regular daily inhaled, topical, nasal or oral steroids for any indication other than CAH.
• Co-morbid condition requiring daily administration of a medication or consumption of any material that interferes with the metabolism of glucocorticoids.
• Participants who are receiving <10 mg hydrocortisone dose at screening or the hydrocortisone dose equivalent.
• Participants anticipating regular prophylactic use of additional steroids e.g. for strenuous exercise.
• Participation in another clinical study of an investigational or licensed drug or device within the 12 weeks prior to screening.
• Inclusion in any natural history or translational research study that would require evaluation of androgen levels during the study period outside of this protocol's assessments.
• Participants who have previously been exposed to Chronocort in any Diurnal study.
• Participants who routinely work night shifts and so do not sleep during the usual night-time hours.
• Participants, who in the opinion of the Investigator, will be unable to comply with the requirements of the protocol.
• Participants with a known hypersensitivity to any of the components of the Chronocort capsules, the Cortef tablets, or the placebo capsules.
• Participants with congenital galactosemia, malabsorption of glucose and galactose, or who are lactase deficient.
• Participants with a body weight of 45 kg or less.
Drug: Chronocort, Drug: Cortef
Congenital Adrenal Hyperplasia
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov

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
67555
All
1 Year to 21 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT05476770
STU-2022-1157
Show full eligibility criteria
Hide eligibility criteria
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
Lymphoma, B-cell, Hodgkin Lymphoma, Acute Undifferentiated Leukemia, MDS, Lymphoblastic Lymphoma, Mixed Phenotype Acute Leukemia, AML, Leukemia, Other, Hodgkins Lymphoma, Leukemia, Not Otherwise Specified, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, ALL, Hematologic Malignancy, BPDCN, Lymphoma, T-Cell
Children’s Health
I'm interested
Share via email
See this study on ClinicalTrials.gov