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ClinicalTrials.gov public records Last synced Oct 23, 2026, 9:12 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 937–960 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 2 Interventional Results available

Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers

NCT00141765
View directory record Official record
Conditions
Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer, Brain Tumor, Recurrent
Interventions
Myeloablative Chemotherapy, Stem Cell Rescue
Procedure
Lead sponsor
University of Michigan Rogel Cancer Center
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2010
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 20, 2014 · Synced Oct 23, 2026, 9:12 AM EDT
Terminated Not applicable Interventional

PK/PD of Vitamin D3 in Adults With CF

NCT03734744
View directory record Official record
Conditions
Vitamin D Deficiency, Cystic Fibrosis
Interventions
Vitamin D3
Dietary Supplement
Lead sponsor
University of Southern California
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 17, 2022 · Synced Oct 23, 2026, 9:12 AM EDT
Terminated Phase 4 Interventional Results available

Safety and Efficacy of Pimecrolimus Cream 1% in Atopic Disease Modification

NCT00124709
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Pimecrolimus, Corticosteroid
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
3 Months to 18 Months
Enrollment
1,091 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
36
States / cities
Birmingham, Alabama • Fayetteville, Arkansas • Jonesboro, Arkansas + 33 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2011 · Synced Oct 23, 2026, 9:12 AM EDT
Completed No phase listed Observational

CureDuchenne Link®: A Resource for Research

NCT04972604
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Interventions
Not listed
Lead sponsor
CureDuchenne
Other
Eligibility
4 Weeks and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
10
States / cities
Little Rock, Arkansas • Newport Beach, California • Orange, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2026 · Synced Oct 23, 2026, 9:12 AM EDT
Completed Phase 2 Interventional Results available

Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma

NCT01553149
View directory record Official record
Conditions
Neurofibromatosis Type 1, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Childhood Visual Pathway Glioma
Interventions
Lenalidomide, Pharmacological Study
Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Up to 21 Years
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2023
U.S. locations
113
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 90 more
Source: ClinicalTrials.gov public record
Updated Jan 7, 2025 · Synced Oct 23, 2026, 9:12 AM EDT
Recruiting Phase 1 Interventional

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

NCT04532047
View directory record Official record
Conditions
MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions
Aldurazyme (laronidase)
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Oct 23, 2026, 9:12 AM EDT
Completed Phase 1 Interventional Results available

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

NCT01065558
View directory record Official record
Conditions
Lesch-Nyhan Disease
Interventions
Ecopipam
Drug
Lead sponsor
Psyadon Pharma
Industry
Eligibility
6 Years to 65 Years · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
2
States / cities
San Diego, California • Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 18, 2013 · Synced Oct 23, 2026, 9:12 AM EDT
Completed Phase 4 Interventional

A Study to See How Well Lebrikizumab Works in Adults and Adolescents With Moderate Atopic Dermatitis (Eczema) and High Itch Burden

NCT07006792
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Lebrikizumab
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
12 Years and older
Enrollment
233 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
35
States / cities
Birmingham, Alabama • Litchfield Park, Arizona • Phoenix, Arizona + 29 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 23, 2026, 9:12 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Atorvastatin ± Aspirin in Lynch Syndrome Syndrome

NCT04379999
View directory record Official record
Conditions
Lynch Syndrome
Interventions
Atorvastatin 20mg, Atorvastatin 20mg AND Aspirin 325 mg
Drug
Lead sponsor
Fox Chase Cancer Center
Other
Eligibility
18 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 7, 2025 · Synced Oct 23, 2026, 9:12 AM EDT
Recruiting Early Phase 1 Interventional

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

NCT06313398
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Hemoglobinopathy
Interventions
Biotin-labeled red blood cells
Biological
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 23, 2026, 9:12 AM EDT
Terminated Not applicable Interventional Results available

Immunoadsorption Therapy for Patients With Non-Ischemic Dilated Cardiomyopathy (DCM)

NCT01478087
View directory record Official record
Conditions
Cardiomyopathy, Dilated
Interventions
Mysorba
Device
Lead sponsor
Asahi Kasei Medical Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
2
States / cities
Rochester, Minnesota • Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 15, 2013 · Synced Oct 23, 2026, 9:12 AM EDT
Completed Phase 1 Interventional

A First-in-Human Study of RPT193 in Healthy Volunteers and Patients With Atopic Dermatitis

NCT04271514
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
RPT193, Placebo
Drug
Lead sponsor
RAPT Therapeutics, Inc., a member of the GSK plc group of companies
Industry
Eligibility
18 Years to 65 Years
Enrollment
103 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
13
States / cities
Anniston, Alabama • Scottsdale, Arizona • San Diego, California + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2021 · Synced Oct 23, 2026, 9:12 AM EDT
Recruiting Phase 1Phase 2 Interventional

Oral Deucrictibant for Prophylactic and Acute Treatment in Hereditary Angioedema Patients

NCT07046806
View directory record Official record
Conditions
Hereditary Angioedema (HAE), Angioedema, Bradykinin-mediated Angioedema, C1 Inhibitor Deficiency
Interventions
Deucrictibant XR tablet, Placebo comparator to XR tablet, Deucrictibant 20 mg capsule, Placebo comparator to 20 mg capsule
Drug
Lead sponsor
Institute for Asthma and Allergy
Network
Eligibility
18 Years to 75 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Wheaton, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2025 · Synced Oct 23, 2026, 9:12 AM EDT
Recruiting Phase 1Phase 2 Interventional

Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists

NCT05463133
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Sirolimus, Cyclophosphamide, Alemtuzumab, Busulfan, Pheripheral blood stem cells, Emapalumab-Izsg, Tociluzumab, Total Body Irradiation
Drug · Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
4 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 23, 2026, 9:12 AM EDT
Active, not recruiting No phase listed Observational

An Observational Study Using Artificial Intelligence (AI) Algorithms on Electrocardiography (ECG), Point-of-care Ultrasound (POCUS), and Transthoracic Echocardiophy (TTE) to Estimate the Under-diagnosis of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Across a Diverse Range of US Health Systems.

NCT07062848
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
AI Toolkit for ATTR-CM Diagnosis
Diagnostic Test
Lead sponsor
Yale University
Other
Eligibility
50 Years to 95 Years
Enrollment
1,500,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
12
States / cities
San Francisco, California • New Haven, Connecticut • Chicago, Illinois + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 23, 2026, 9:12 AM EDT
Recruiting No phase listed Observational

Polycystic Kidney Disease Data Repository

NCT00792155
View directory record Official record
Conditions
Polycystic Kidney Disease
Interventions
Not listed
Lead sponsor
The Rogosin Institute
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2030
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 23, 2026, 9:12 AM EDT
Recruiting Phase 1Phase 2 Interventional

Treatment of Refractory BK Infections With Related Donor BK Specific Cytotoxic T-cells (CTLs)

NCT04197596
View directory record Official record
Conditions
Viral Infection, Primary Immune Deficiency Disorder
Interventions
BK CTL
Biological
Lead sponsor
New York Medical College
Other
Eligibility
1 Month to 79 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
7
States / cities
San Francisco, California • Baltimore, Maryland • St Louis, Missouri + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Oct 23, 2026, 9:12 AM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

NCT06546670
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
ITU512, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
12 Years to 55 Years
Enrollment
161 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2030
U.S. locations
9
States / cities
Birmingham, Alabama • Miami, Florida • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 23, 2026, 9:12 AM EDT
Active, not recruiting Phase 3 Interventional

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

NCT03837483
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
OTL-103
Genetic
Lead sponsor
Fondazione Telethon
Other
Eligibility
Up to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Oct 23, 2026, 9:12 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Universal Endometrial Cancer DNA Sequencing for Detection of Lynch Syndrome and Personalized Care

NCT03460483
View directory record Official record
Conditions
Endometrial Adenocarcinoma, Endometrial Carcinoma, Lynch Syndrome, Relatives
Interventions
Genetic Counseling, Genetic Testing, Laboratory Biomarker Analysis, Mutation Carrier Screening
Other · Procedure
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
1,001 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2025
U.S. locations
9
States / cities
Akron, Ohio • Canton, Ohio • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 3, 2025 · Synced Oct 23, 2026, 9:12 AM EDT
Not listed Not applicable Interventional

Identification of Undiagnosed Gaucher Disease

NCT01716741
View directory record Official record
Conditions
Gaucher Disease
Interventions
Enzyme analysis
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 30, 2012 · Synced Oct 23, 2026, 9:12 AM EDT
Terminated Phase 2 Interventional Results available

A Phase II Study of Bintrafusp Alfa (M7824) in Checkpoint Inhibitor Naive and Refractory Subjects With Urothelial Carcinoma

NCT04501094
View directory record Official record
Conditions
Urothelial Cancer
Interventions
Bintrafusp alfa (M7824)
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 22, 2022 · Synced Oct 23, 2026, 9:12 AM EDT
Completed No phase listed Observational

A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

NCT04721366
View directory record Official record
Conditions
Gaucher Disease
Interventions
Standard of Care
Other
Lead sponsor
Takeda
Industry
Eligibility
Up to 5 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 29, 2024 · Synced Oct 23, 2026, 9:12 AM EDT
Completed No phase listed Observational

Serum Brain Natriuretic Peptide Levels and Pulmonary Hypertension in Pediatric Sickle Cell Patients

NCT01023451
View directory record Official record
Conditions
Pulmonary Hypertension
Interventions
Not listed
Lead sponsor
Akron Children's Hospital
Other
Eligibility
7 Years to 21 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 26, 2014 · Synced Oct 23, 2026, 9:12 AM EDT