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ClinicalTrials.gov public records Last synced Oct 23, 2026, 1:44 PM EDT

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

Showing 385–408 of 10,333 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital, Hereditary, and Neonatal Diseases and Abnormalities Clear all
Active, not recruiting Phase 3 Interventional

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

NCT05881408
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
Male only
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
14
States / cities
Little Rock, Arkansas • Palo Alto, California • Sacramento, California + 11 more
Source: ClinicalTrials.gov public record
Updated May 22, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Not applicable Interventional Results available

Clinical Study on a New Flowable Composite as a Restorative in Adult Teeth

NCT01369108
View directory record Official record
Conditions
Dental Caries
Interventions
Flowable composite, Conventional composite restorative
Device
Lead sponsor
Solventum US LLC
Industry
Eligibility
19 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Oct 2, 2024 · Synced Oct 23, 2026, 1:44 PM EDT
Completed No phase listed Observational

Neurocognitive and Psychosocial Outcomes in Survivors of Childhood Leukemia With Down Syndrome

NCT02859389
View directory record Official record
Conditions
Leukemia, Down Syndrome
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 18, 2019 · Synced Oct 23, 2026, 1:44 PM EDT
Terminated Phase 2 Interventional

Hospital-Based Program for Treatment of Severe Cardiopulmonary Disease With Inhaled Nitric Oxide

NCT00041574
View directory record Official record
Conditions
Pulmonary Hypertension, Lung Disease, Sickle Cell Disease, Cardiac Transplant, Lung Transplant
Interventions
Inhaled Nitric Oxide
Drug
Lead sponsor
Mallinckrodt
Industry
Eligibility
5 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2005
U.S. locations
3
States / cities
Denver, Colorado • Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Not applicable Interventional

Marfan Syndrome Moderate Exercise Pilot

NCT04641325
View directory record Official record
Conditions
Marfan Syndrome
Interventions
Exercise Intervention Group
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
12 Years to 21 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Aug 14, 2023 · Synced Oct 23, 2026, 1:44 PM EDT
Recruiting No phase listed Observational

BBD Longitudinal Study of Osteogenesis Imperfecta

NCT02432625
View directory record Official record
Conditions
Osteogenesis Imperfecta
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2031
U.S. locations
11
States / cities
Phoenix, Arizona • Los Angeles, California • Wilmington, Delaware + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

HDClarity: a Multi-site Cerebrospinal Fluid Collection Initiative to Facilitate Therapeutic Development for Huntington's Disease

NCT02855476
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
University College, London
Other
Eligibility
11 Years to 75 Years
Enrollment
2,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2017
U.S. locations
6
States / cities
Englewood, Colorado • Washington D.C., District of Columbia • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Not applicable Interventional

Investigating the Feasibility and Implementation of Whole Genome Sequencing in Patients With Suspected Genetic Disorder

NCT03829176
View directory record Official record
Conditions
Hereditary Disease, Genetic Predisposition to Disease
Interventions
Whole Genome Sequencing
Genetic
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
3 Months and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 18, 2020 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Enhancing At-risk Latina Women's Use of Genetic Counseling for Hereditary Breast and Ovarian Cancer

NCT03075540
View directory record Official record
Conditions
Hereditary Breast and Ovarian Cancer
Interventions
Genetic Counseling and Testing YouTube Video
Behavioral
Lead sponsor
Georgetown University
Other
Eligibility
21 Years and older · Female only
Enrollment
88 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Alexandria, Virginia
Source: ClinicalTrials.gov public record
Updated Dec 12, 2019 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

RDEA3170 PK/PD Study

NCT02608710
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 4.5 mg, RDEA3170 6 mg, RDEA3170 12 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Sep 2, 2016 · Synced Oct 23, 2026, 1:44 PM EDT
Terminated Phase 2 Interventional Results available

Panhematin for Prevention of Acute Attacks of Porphyria

NCT02922413
View directory record Official record
Conditions
Acute Intermittent Porphyria, Hereditary Coproporphyria, Variegate Porphyria
Interventions
Hemin for injection, Placebo
Biological · Other
Lead sponsor
The University of Texas Medical Branch, Galveston
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

NCT01856868
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(-)-epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 22, 2021 · Synced Oct 23, 2026, 1:44 PM EDT
Recruiting No phase listed Observational

Genetic Disorders of Obesity Program Database

NCT05747976
View directory record Official record
Conditions
Obesity, Childhood, Genetic Disease
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Not yet recruiting Not applicable Interventional

Evaluation of 3D Printed Myoelectric Prosthetic Devices in Children With Upper Congenital Limb Deficiency

NCT05547308
View directory record Official record
Conditions
Limb Deformities, Congenital, Upper Extremity Deformities, Congenital, Congenital Abnormalities
Interventions
3D myoelectric prosthetic device
Device
Lead sponsor
Orlando Health, Inc.
Other
Eligibility
7 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Sep 21, 2022 · Synced Oct 23, 2026, 1:44 PM EDT
Active, not recruiting Phase 3 Interventional

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
avalglucosidase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
0 Days to 12 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Stanford, California • Minneapolis, Minnesota • Hawthorne, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Recruiting Phase 4 Interventional

De-implementing Inhaled Nitric Oxide for Congenital Diaphragmatic Hernia

NCT05213676
View directory record Official record
Conditions
Congenital Diaphragmatic Hernia
Interventions
Inhaled Nitric Oxide (iNO) use, De-implementation of Inhaled Nitric Oxide (iNO) use
Drug · Other
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
0 Months to 1 Month
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
19
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Irvine, California + 16 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Phase 3 Interventional

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

NCT05153317
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
ELX/TEZ/IVA, IVA
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
13
States / cities
Tucson, Arizona • Palo Alto, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Phase 1 Interventional

Treatment of Dysglycemia Using Sitagliptin in Adolescents With Cystic Fibrosis

NCT01721382
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Sitagliptin
Drug
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
13 Years to 20 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Jun 14, 2016 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Not applicable Interventional

Auditory Neural Function in Implanted Patients With Usher Syndrome

NCT04906135
View directory record Official record
Conditions
Usher Syndrome, Cochlear Implantation
Interventions
Experimental manipulation of stimulation parameters
Other
Lead sponsor
Ohio State University
Other
Eligibility
1 Year to 85 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
4
States / cities
Boston, Massachusetts • Columbus, Ohio • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 29, 2025 · Synced Oct 23, 2026, 1:44 PM EDT
Active, not recruiting Phase 2 Interventional

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

NCT04239625
View directory record Official record
Conditions
Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
Interventions
ALK-001
Drug
Lead sponsor
Alkeus Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 70 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
8
States / cities
Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Phase 2 Interventional

Radiolabeled Monoclonal Antibody, Cyclophosphamide, and Total Body Irradiation Followed By Donor Stem Cell Transplantation in Treating Patients With Advanced Acute Myeloid Leukemia

NCT00003868
View directory record Official record
Conditions
Leukemia
Interventions
cyclophosphamide, methotrexate, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, iodine I 131 monoclonal antibody BC8, radiation therapy
Drug · Procedure · Radiation
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
2 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2005
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Aug 24, 2010 · Synced Oct 23, 2026, 1:44 PM EDT
Terminated No phase listed Observational

Assessing Clinical Endpoints and Biomarkers in Myotonic Dystrophy Type-1 and Type 2 (ASCEND-DM)

NCT03867435
View directory record Official record
Conditions
Myotonic Dystrophy Type-1, Myotonic Dystrophy Type-2
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
11 Years to 70 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 23, 2026, 1:44 PM EDT
Completed Phase 1 Interventional Results available

Rasburicase in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer or Other Disease Undergoing Donor Stem Cell Transplant

NCT00513474
View directory record Official record
Conditions
Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions
busulfan, cyclophosphamide, cyclosporin-A, etoposide, methotrexate, rasburicase, sirolimus, tacrolimus, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, total-body irradiation, fludarabine, allopurinol
Drug · Procedure · Radiation
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 25, 2017 · Synced Oct 23, 2026, 1:44 PM EDT
Active, not recruiting Phase 3 Interventional

Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1

NCT05680818
View directory record Official record
Conditions
Autosomal Dominant Hypocalcemia (ADH)
Interventions
Encaleret, Standard of Care
Drug · Dietary Supplement
Lead sponsor
Calcilytix Therapeutics, Inc., a BridgeBio company
Industry
Eligibility
16 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
11
States / cities
Oakland, California • Aurora, Colorado • Indianapolis, Indiana + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 23, 2026, 1:44 PM EDT