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ClinicalTrials.gov public records Last synced Oct 22, 2026, 7:20 AM EDT

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Showing 577–600 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Active, not recruiting No phase listed Observational

Cerliponase Alfa Observational Study in the US

NCT04476862
View directory record Official record
Conditions
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa, Administration Kit
Drug · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
16
States / cities
Little Rock, Arkansas • Orange, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Nitric Oxide Production in MELAS Syndrome

NCT01339494
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Arginine and citrulline supplementations
Dietary Supplement
Lead sponsor
Baylor College of Medicine
Other
Eligibility
3 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 5, 2016 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Phase 4 Interventional

Comparison of Two Drugs, Cidofovir and Ganciclovir, in Treating Patients With AIDS Who Have CMV Retinitis

NCT00000894
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Cidofovir, Probenecid, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
12
States / cities
Los Angeles, California • Miami, Florida • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Feb 23, 2011 · Synced Oct 22, 2026, 7:20 AM EDT
Recruiting Not applicable Interventional

Plasma Rich in Growth Factors in Corneal Endothelial Transplantation

NCT06261346
View directory record Official record
Conditions
Fuchs' Endothelial Dystrophy, Corneal Edema
Interventions
Endothelial keratoplasty, PRGF
Procedure · Biological
Lead sponsor
University of Miami
Other
Eligibility
18 Years to 80 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Miami, Florida • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Oct 22, 2026, 7:20 AM EDT
Terminated Phase 1 Interventional

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

NCT00215527
View directory record Official record
Conditions
Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
Interventions
laronidase
Drug
Lead sponsor
Patricia I. Dickson, M.D.
Individual
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2011
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Feb 21, 2013 · Synced Oct 22, 2026, 7:20 AM EDT
Recruiting Phase 4 Interventional

Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency

NCT07135427
View directory record Official record
Conditions
Alpha 1-Antitrypsin, COPD, Antibody Deficiency
Interventions
20-valent pneumococcal conjugate vaccine
Biological
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
19 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 10, 2025 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Phase 3 Interventional Results available

CLINICAL PHASE III STUDY TO MONITOR THE SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (OCTANORM) IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES, INCLUDING (BUT NOT LIMITED TO) THOSE WHO HAVE COMPLETED THE SCGAM-01 TRIAL

NCT03907241
View directory record Official record
Conditions
Primary Immunodeficiency
Interventions
Octanorm 16.5%
Drug
Lead sponsor
Octapharma
Industry
Eligibility
2 Years to 75 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
6
States / cities
Irvine, California • San Diego, California • Centennial, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2020 · Synced Oct 22, 2026, 7:20 AM EDT
Completed No phase listed Observational

Exploring How to Adapt an Evidence-Based Mindful Self-Compassion Program for Young Adults With Li-Fraumeni Syndrome

NCT07032922
View directory record Official record
Conditions
Li-Fraumeni Syndrome
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 29 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 10, 2025 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Phase 3 Interventional Results available

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

NCT01140451
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
191 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
15
States / cities
Birmingham, Alabama • Long Beach, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2020 · Synced Oct 22, 2026, 7:20 AM EDT
Terminated Phase 1 Interventional Results available

A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

NCT03362502
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
PF-06939926
Genetic
Lead sponsor
Pfizer
Industry
Eligibility
4 Years and older · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
22
States / cities
Los Angeles, California • Durham, North Carolina • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Not applicable Interventional Results available

Open-label Vitamin D Trial for Patients With Cystic Fibrosis and Allergic Bronchopulmonary Aspergillosis

NCT01222273
View directory record Official record
Conditions
Cystic Fibrosis, Allergic Bronchopulmonary Aspergillosis
Interventions
cholecalciferol (Vitamin D3)
Dietary Supplement
Lead sponsor
University of Pittsburgh
Other
Eligibility
12 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 29, 2018 · Synced Oct 22, 2026, 7:20 AM EDT
Terminated Phase 1 Interventional

Neuroimaging GABA Physiology in Fragile X Syndrome

NCT04308954
View directory record Official record
Conditions
Fragile X Syndrome (FXS), Idiopathic Intellectual Developmental Disorder (IDD)
Interventions
[18F]flumazenil
Drug
Lead sponsor
Stanford University
Other
Eligibility
18 Years to 30 Years · Male only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Jan 28, 2021 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Phase 1 Interventional

Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex

NCT05062070
View directory record Official record
Conditions
Epidermolysis Bullosa Simplex
Interventions
TolaSure Topical Gel, Vehicle Topical Gel
Drug
Lead sponsor
BioMendics, LLC
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Redwood City, California
Source: ClinicalTrials.gov public record
Updated Sep 11, 2023 · Synced Oct 22, 2026, 7:20 AM EDT
Active, not recruiting No phase listed Observational

Analysis of Specimens From Individuals With Pulmonary Fibrosis

NCT00084305
View directory record Official record
Conditions
Pulmonary Fibrosis, Healthy Volunteers, Hermansky-Pudlak Syndrome (HPS)
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 115 Years
Enrollment
315 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Oct 22, 2026, 7:20 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Proxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum Disorders

NCT03440905
View directory record Official record
Conditions
Zellweger Spectrum
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
92 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Phase 2 Interventional Results available

Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial Hypercholesterolemia

NCT00477594
View directory record Official record
Conditions
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases, Hyperlipoproteinemia Type II, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Infant, Newborn, Diseases, Metabolic Disorder, Congenital Abnormalities, Hypercholesterolemia, Hyperlipoproteinemias, Dyslipidemias, Lipid Metabolism Disorders
Interventions
mipomersen sodium
Drug
Lead sponsor
Kastle Therapeutics, LLC
Industry
Eligibility
12 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
5
States / cities
Chicago, Illinois • Auburn, Maine • Biddeford, Maine + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2016 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Phase 3 Interventional Results available

Phase 3 Study of Aztreonam for Inhalation Solution (AZLI) in a Continuous Alternating Therapy Regimen for the Treatment of Chronic Pseudomonas Aeruginosa Infection in Patients With CF

NCT01641822
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
AZLI, Placebo to match AZLI, Tobramycin inhalation solution
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
6 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
71
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 62 more
Source: ClinicalTrials.gov public record
Updated May 9, 2016 · Synced Oct 22, 2026, 7:20 AM EDT
Completed No phase listed Observational

Positional Cloning of the Gene(s) Responsible for Alagille Syndrome

NCT00001642
View directory record Official record
Conditions
Alagille Syndrome
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
Not listed
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Phase 4 Interventional Results available

Developing New Treatments for Tourette Syndrome: Therapeutic Trials With Modulators of Glutamatergic Neurotransmission

NCT01018056
View directory record Official record
Conditions
Tourette Syndrome
Interventions
D-serine, Riluzole, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
8 Years to 17 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 30, 2018 · Synced Oct 22, 2026, 7:20 AM EDT
Suspended Not applicable Interventional

Clinical Evaluation of the AccuCinch® Ventricular Restoration System in Patients Who Present With Symptomatic Heart Failure With Reduced Ejection Fraction (HFrEF): The CORCINCH-HF Study

NCT04331769
View directory record Official record
Conditions
Heart Failure With Reduced Ejection Fraction (HFrEF), Dilated Cardiomyopathy
Interventions
AccuCinch Ventricular Restoration System, Guideline-Directed Medical Therapy
Device · Drug
Lead sponsor
Ancora Heart, Inc.
Industry
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
96
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 75 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 22, 2026, 7:20 AM EDT
Active, not recruiting Not applicable Interventional

Effect of Exercise on Body Composition and Bone Health in Patients With Thalassemia

NCT07064941
View directory record Official record
Conditions
Thalassemia
Interventions
Exercise
Behavioral
Lead sponsor
University of California, San Francisco
Other
Eligibility
14 Years to 40 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
2
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 22, 2026, 7:20 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Prenatal Transplantation for Fetuses With Fanconi Anemia

NCT07408583
View directory record Official record
Conditions
Fanconi Anemia, Anemia, Hypoplastic, Congenital, Congenital Bone Marrow Failure Syndromes, Bone Marrow Failure Disorders, Genetic Diseases, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA Repair-Deficiency Disorders, Cancer Predisposition Syndrome
Interventions
IUHSCT for FA-affected fetuses
Biological
Lead sponsor
Agnieszka Czechowicz
Other
Eligibility
Not listed
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2028 – 2033
U.S. locations
2
States / cities
San Francisco, California • Stanford, California
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Oct 22, 2026, 7:20 AM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

NCT00873041
View directory record Official record
Conditions
Non-transfusion Dependent Thalassemia
Interventions
deferasirox, placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
10 Years and older
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 9, 2013 · Synced Oct 22, 2026, 7:20 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

NCT06065605
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
2
States / cities
Rockville, Maryland • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Oct 22, 2026, 7:20 AM EDT