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ClinicalTrials.gov public records Last synced Oct 22, 2026, 5:25 AM EDT

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Showing 553–576 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed No phase listed Observational

Observational Hemodynamic Monitoring During LVAD Implantation Among Individuals With Advanced Heart Failure

NCT03464981
View directory record Official record
Conditions
Heart Failure, Circulatory Disorders Postprocedural Complication, Cardiomyopathy, Congestive, Surgery
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Sep 25, 2020 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Phase 2 Interventional Results available

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

NCT02693119
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
elamipretide (MTP-131) 1% topical ophthalmic solution, Vehicle topical ophthalmic solution
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 30, 2021 · Synced Oct 22, 2026, 5:25 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Development of a Blood Test for Marfan Syndrome

NCT02148900
View directory record Official record
Conditions
Marfan Syndrome, Marfan Related Disorders, Control Subjects
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
4 Years to 26 Years
Enrollment
336 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2017
U.S. locations
5
States / cities
Chicago, Illinois • Omaha, Nebraska • Portland, Oregon + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 5, 2017 · Synced Oct 22, 2026, 5:25 AM EDT
Recruiting No phase listed Observational

Evaluation of a Non-Invasive Device for Early Detection of Atopic Dermatitis Flares

NCT07645820
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Nevisense electrical impedance spectroscopy
Device
Lead sponsor
Castle Biosciences Incorporated
Industry
Eligibility
12 Years to 89 Years
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
13
States / cities
Phoenix, Arizona • Los Angeles, California • Rockville, Maryland + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 22, 2026, 5:25 AM EDT
Active, not recruiting No phase listed Observational

AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD

NCT05683379
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
AAV8 DetectCDx
Diagnostic Test
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
0 Years to 25 Years · Male only
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Oct 22, 2026, 5:25 AM EDT
Recruiting No phase listed Observational

Remote Assessments and Genetic Determinants of Myotonic Dystrophy

NCT07505342
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1), DM1
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 88 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Phase 2 Interventional Results available

MK-3795 (PT2385) for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma (MK-3795-003)

NCT03108066
View directory record Official record
Conditions
VHL Gene Mutation, VHL, VHL Syndrome, VHL Gene Inactivation, Von Hippel, Von Hippel-Lindau Disease, Von Hippel's Disease, Von Hippel-Lindau Syndrome, Modifiers of, Clear Cell Renal Cell Carcinoma, Clear Cell RCC, ccRCC
Interventions
MK-3795
Drug
Lead sponsor
Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 24, 2024 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Phase 2 Interventional

Nutrients and Hormones: Effects in Boys With Disordered Growth

NCT00139451
View directory record Official record
Conditions
Constitutional Growth Delay, Nutrition Disorders, Growth Disorders
Interventions
Pediasure, GH
Drug
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
7 Years to 10 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 12, 2011 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease

NCT00911495
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
GMI-1070
Drug
Lead sponsor
GlycoMimetics Incorporated
Industry
Eligibility
18 Years to 45 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
3
States / cities
Oakland, California • Sacramento, California • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 11, 2020 · Synced Oct 22, 2026, 5:25 AM EDT
Active, not recruiting Phase 2 Interventional

Gene Therapy for Fanconi Anemia, Complementation Group A

NCT04248439
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
2
States / cities
Stanford, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT02579239
View directory record Official record
Conditions
Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy
Interventions
ATYR1940, Placebo
Biological
Lead sponsor
aTyr Pharma, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
3
States / cities
Irvine, California • Baltimore, Maryland • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 19, 2023 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Phase 1Phase 2 Interventional

Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy

NCT00470977
View directory record Official record
Conditions
Coats' Disease, Idiopathic Retinal Telangiectasia, Retinal Angiomatous Proliferation, Polypoidal Choroidal Vasculopathy, Pseudoxanthoma Elasticum, Pathological Myopia, Multi-focal Choroiditis, Rubeosis Iridis, Von Hippel Lindau Disease, BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder)
Interventions
ranibizumab injection (0.5 mg)
Drug
Lead sponsor
Manhattan Eye, Ear & Throat Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 25, 2012 · Synced Oct 22, 2026, 5:25 AM EDT
Completed No phase listed Observational

Study of Hepatic Function in Patients With Spinal and Bulbar Muscular Atrophy

NCT02124057
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy (SBMA), Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 18, 2022 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating Rocatinlimab in Moderate-to-severe Atopic Dermatitis (ROCKET-IGNITE)

NCT05398445
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Rocatinlimab, Placebo
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years to 100 Years
Enrollment
769 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Anaheim, California + 44 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Oct 22, 2026, 5:25 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Children With CLN3 Batten Disease

NCT03770572
View directory record Official record
Conditions
CLN3, Batten Disease
Interventions
Low dose CLN-301, High dose CLN-301
Genetic
Lead sponsor
Neela Therapeutics
Industry
Eligibility
3 Years to 10 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 22, 2026, 5:25 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

A Study to Test the Diagnostic Potential of Brillouin Microscopy for Corneal Ectasia

NCT02118922
View directory record Official record
Conditions
Keratoconus, Ectasia, Crosslinking, Fuchs' Endothelial Dystrophy
Interventions
Brillouin imaging
Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
20 Years to 75 Years
Enrollment
168 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Not applicable Interventional Results available

Massive Iron Deposit Assessment

NCT01572922
View directory record Official record
Conditions
Iron Overload, Excessive Body Iron Burden
Interventions
R2*-UTE, R2*-GRE, Liver biopsy
Device · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
142 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 11, 2019 · Synced Oct 22, 2026, 5:25 AM EDT
Completed No phase listed Observational

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

NCT05255445
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Pediatric Cancer
Interventions
Red Blood Cell (RBC) Transfusion
Biological
Lead sponsor
Westat
Other
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
Oakland, California • San Francisco, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 17, 2024 · Synced Oct 22, 2026, 5:25 AM EDT
Recruiting Phase 2 Interventional

A Phase II, Placebo Controlled, Clinical Trial of Topical TolaSure Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex

NCT07027345
View directory record Official record
Conditions
Epidermolysis Bullosa Simplex
Interventions
5% TolaSure Topical Gel, Topical Placebo Gel
Drug
Lead sponsor
BioMendics, LLC
Industry
Eligibility
2 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
2
States / cities
Palo Alto, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 22, 2026, 5:25 AM EDT
Withdrawn Phase 2 Interventional

Alipogene Tiparvovec for the Treatment of LPLD Patients

NCT02904772
View directory record Official record
Conditions
LPL Deficiency
Interventions
alipogene tiparvovec, Prednisolone, Cyclosporins, Mycophenolate mofetil
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 17, 2017 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Phase 2 Interventional Results available

Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2

NCT01419639
View directory record Official record
Conditions
Neurofibromatosis Type II
Interventions
Everolimus (RAD001) , Afinitor®
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
3 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 18, 2017 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Not applicable Interventional

A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia

NCT03013777
View directory record Official record
Conditions
Dysthymia, Anxiety Disorders, Familial Dysautonomia, Paroxysmal Hypertension, Autosomal Recessive Disease
Interventions
8-week CBT Program
Behavioral
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 29, 2019 · Synced Oct 22, 2026, 5:25 AM EDT
Recruiting Phase 2 Interventional

Cardamom and Topical Roseomonas in Atopic Dermatitis

NCT06096857
View directory record Official record
Conditions
Atopic Dermatitis, Eczema
Interventions
Roseomonas mucosa (RSM2015) and Cardamom seeds, Placebo (sucrose)
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
2 Years to 100 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 22, 2026, 5:25 AM EDT
Completed Not applicable Interventional

PACE CF, a 3-session Mind-body Pain Management Program for Adults With Cystic Fibrosis

NCT06606457
View directory record Official record
Conditions
Cystic Fibrosis (CF), Pain Management
Interventions
Pain Acknowledgment Coping and Empowerment in CF (PACE CF)
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 12, 2025 · Synced Oct 22, 2026, 5:25 AM EDT