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ClinicalTrials.gov public records Last synced Oct 15, 2026, 4:55 PM EDT

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Showing 697–720 of 10,531 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Diseases Clear all
Not listed No phase listed Observational Accepts healthy volunteers

Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders

NCT03812055
View directory record Official record
Conditions
Lysosomal Storage Diseases
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 22, 2019 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Not applicable Interventional Results available

Team-Based Connected Health (TCH) to Improve Clinical Outcomes and Access in Atopic Dermatitis

NCT03981926
View directory record Official record
Conditions
Atopic Dermatitis Eczema, Atopic Dermatitis, Eczema
Interventions
Team-Based Connected Health (TCH)
Other
Lead sponsor
University of Southern California
Other
Eligibility
1 Year and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
2
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Oct 6, 2025 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Feasibility of a Mobile Medication Plan Application in CF Patient Care

NCT03637504
View directory record Official record
Conditions
Cystic Fibrosis, Cystic Fibrosis in Children
Interventions
MedActionPlan®
Behavioral
Lead sponsor
Boston Children's Hospital
Other
Eligibility
12 Years and older
Enrollment
105 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2021
U.S. locations
5
States / cities
Birmingham, Alabama • Tucson, Arizona • Stanford, California + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2021 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Reproductive Capacity and Iron Burden in Thalassemia

NCT02308904
View directory record Official record
Conditions
THALASSEMIA MAJOR
Interventions
Blood Draw/Semen Exam, Retrospective data/Chart Review/Relevant Clinical Results, Pituitary MRI
Other
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
12 Years and older
Enrollment
59 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Dec 23, 2020 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Not applicable Interventional

A Comparison Between Full Thickness and Partial Thickness Corneal Transplantation for Corneal Edema

NCT00346138
View directory record Official record
Conditions
Fuchs Dystrophy, Corneal Edema
Interventions
Corneal transplantation
Procedure
Lead sponsor
Mayo Clinic
Other
Eligibility
21 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 15, 2010 · Synced Oct 15, 2026, 4:55 PM EDT
Withdrawn No phase listed Observational

Microbiota of the Respiratory Flora in Children With Cystic Fibrosis During the First Year of Life

NCT00977158
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Throat Swab
Procedure
Lead sponsor
Tufts Medical Center
Other
Eligibility
1 Day to 3 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
2
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2015 · Synced Oct 15, 2026, 4:55 PM EDT
Recruiting Phase 3 Interventional

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

NCT07160634
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SGT-003, Placebo
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
7 Years to 11 Years · Male only
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2034
U.S. locations
3
States / cities
Little Rock, Arkansas • Flower Mound, Texas • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 15, 2026, 4:55 PM EDT
Terminated Phase 2 Interventional Results available

Study of Ataluren (PTC124) in Hemophilia A and B

NCT00947193
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
7
States / cities
Peoria, Illinois • Indianapolis, Indiana • Worcester, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2020 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

NCT02412787
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Idursulfase-IT, Elaprase
Drug
Lead sponsor
Shire
Industry
Eligibility
Up to 18 Years · Male only
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 19, 2025 · Synced Oct 15, 2026, 4:55 PM EDT
Recruiting Phase 2 Interventional

A Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study

NCT06594393
View directory record Official record
Conditions
Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa
Interventions
TCP-25 gel, Vehicle (placebo)
Drug
Lead sponsor
Xinnate AB
Industry
Eligibility
4 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 15, 2026, 4:55 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Epacadostat in Combination With Pembrolizumab and Azacitidine in Subjects With Metastatic Colorectal Cancer

NCT03182894
View directory record Official record
Conditions
Metastatic Colorectal Cancer
Interventions
Epacadostat (INCB024360) in Combination with Pembrolizumab (MK-3475) and Azacitidine (VIDAZA)
Drug
Lead sponsor
James J Lee
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients

NCT02273596
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
4
States / cities
Los Angeles, California • New Haven, Connecticut • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2021 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Trial to Compare eConsent With Standard Consent Among Prospective Biobank Participants

NCT04131062
View directory record Official record
Conditions
Lynch Syndrome
Interventions
Electronic Consent (iPad)
Behavioral
Lead sponsor
Geisinger Clinic
Other
Eligibility
18 Years and older
Enrollment
703 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2022
U.S. locations
1
States / cities
Danville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 27, 2022 · Synced Oct 15, 2026, 4:55 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

NCT04849741
View directory record Official record
Conditions
Alexander Disease
Interventions
zilganersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 65 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2034
U.S. locations
4
States / cities
Palo Alto, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 15, 2026, 4:55 PM EDT
Active, not recruiting Phase 2 Interventional

A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

NCT06668805
View directory record Official record
Conditions
Noonan Syndrome
Interventions
Vosoritide Injection
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 11 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2041
U.S. locations
21
States / cities
Los Angeles, California • Orange, California • Aurora, Colorado + 18 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Not applicable Interventional

Assessing the Effects of Auricular Acupressure on Newborns With NAS

NCT03890562
View directory record Official record
Conditions
Neonatal Abstinence Syndrome
Interventions
Auricular acupressure
Other
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
Up to 2 Months
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced Oct 15, 2026, 4:55 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics

NCT00001373
View directory record Official record
Conditions
Familial Mediterranean Fever (FMF), Autoinflammation, Periodic Fever, Fever, Genetic Diseases, ROSAH, ALPK1
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Months to 115 Years
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1994
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Inhaled NO as an Anti-inflammatory and Anti-reperfusion Agent in Infants and Children Undergoing Cardiopulmonary Bypass

NCT00585013
View directory record Official record
Conditions
Congenital Heart Disease
Interventions
Nitric Oxide
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Up to 16 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Nov 4, 2015 · Synced Oct 15, 2026, 4:55 PM EDT
Completed No phase listed Observational

Stress, Hypothalamic-pituitary-adrenal (HPA) Dysfunction, and Relapse in Alcoholism

NCT00744588
View directory record Official record
Conditions
Alcohol Dependence
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 60 Years · Male only
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 3, 2013 · Synced Oct 15, 2026, 4:55 PM EDT
Recruiting Phase 3 Interventional

Open-label Extension Study to Evaluate Metreleptin in Patients With Partial Lipodystrophy

NCT06679270
View directory record Official record
Conditions
Familial Partial Lipodystrophy
Interventions
Metreleptin
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
13 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
7
States / cities
Birmingham, Alabama • Boca Raton, Florida • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 15, 2026, 4:55 PM EDT
Completed No phase listed Observational

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)

NCT02057705
View directory record Official record
Conditions
Myotubular Myopathy
Interventions
Not listed
Lead sponsor
Valerion Therapeutics, LLC
Industry
Eligibility
Not listed
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 7, 2018 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Early Phase 1 Interventional

Ketones & Mitochondrial Heteroplasmy

NCT01252979
View directory record Official record
Conditions
MELAS Syndrome, Mitochondrial Diseases
Interventions
Medium-Chain Triglycerides
Dietary Supplement
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 6, 2012 · Synced Oct 15, 2026, 4:55 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Microfluidic Assessment of Clinical Outcomes in Preterm Newborns

NCT03291496
View directory record Official record
Conditions
Neonatal SEPSIS
Interventions
Blood Collection Preterm, Blood Collection Term, Adult Blood collection
Other
Lead sponsor
University of Florida
Other
Eligibility
23 Weeks to 42 Weeks
Enrollment
293 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2026
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 13, 2026 · Synced Oct 15, 2026, 4:55 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

A Brief Laboratory-Based Hypnosis Session for Pain in Sickle Cell Disease

NCT02620488
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Guided Imagery
Behavioral
Lead sponsor
University of California, Los Angeles
Other
Eligibility
13 Years and older
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated May 3, 2017 · Synced Oct 15, 2026, 4:55 PM EDT