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ClinicalTrials.gov public records Last synced Oct 22, 2026, 4:14 PM EDT

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

Showing 697–720 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Withdrawn No phase listed Observational

Early Detection of Graft-Versus-Host Disease in Patients Undergoing a Donor Bone Marrow Transplant

NCT00898612
View directory record Official record
Conditions
Lymphoma
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Scottsdale, Arizona
Source: ClinicalTrials.gov public record
Updated Mar 10, 2014 · Synced Oct 22, 2026, 4:14 PM EDT
Completed No phase listed Observational

Microbial Community Composition and Dynamics in Lungs of Cystic Fibrosis Sibling Pairs

NCT00590330
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Year to 16 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 13, 2011 · Synced Oct 22, 2026, 4:14 PM EDT
Terminated Phase 2 Interventional

Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa

NCT03578029
View directory record Official record
Conditions
Junctional Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa
Interventions
RGN-137, Placebo
Drug
Lead sponsor
Lenus Therapeutics, LLC
Industry
Eligibility
4 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
5
States / cities
Evanston, Illinois • Brooklyn, New York • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 22, 2022 · Synced Oct 22, 2026, 4:14 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

NCT02583672
View directory record Official record
Conditions
Gaucher Disease Type 1
Interventions
N-acetylcysteine
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
2
States / cities
Minneapolis, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT)

NCT06071377
View directory record Official record
Conditions
Sickle Cell Trait
Interventions
Biologic Specimen Collection
Other
Lead sponsor
National Alliance for Sickle Cell Centers
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
8
States / cities
Birmingham, Alabama • Wilmington, Delaware • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

NCT06389877
View directory record Official record
Conditions
Alpha 1-Antitrypsin Deficiency
Interventions
BEAM-302
Drug
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
192 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Birmingham, Alabama • Boston, Massachusetts • Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Not listed Not applicable Interventional

Identification of Undiagnosed Lysosomal Acid Lipase Deficiency

NCT01716728
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
Interventions
enzyme analysis
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 13, 2013 · Synced Oct 22, 2026, 4:14 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Determine the Relative Bioavailability of Capsule and Tablet Formulations of GDC-0941 and the Effect of Food and Proton Pump Inhibition on GDC-0941 Tablet Pharmacokinetics in Healthy Volunteers

NCT00999128
View directory record Official record
Conditions
Healthy Volunteers
Interventions
GDC-0941, rabeprazole
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
1
States / cities
South San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 9, 2017 · Synced Oct 22, 2026, 4:14 PM EDT
Not listed Not applicable Interventional

Overcoming Barriers to Accessing Genetic Medicine

NCT05064241
View directory record Official record
Conditions
Genetic Disease, Genetic Predisposition
Interventions
Access to Genetic Counselor for answering questions
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 3, 2024 · Synced Oct 22, 2026, 4:14 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Prenatal Test for Fetal Aneuploidy Detection

NCT01256606
View directory record Official record
Conditions
Aneuploidy, Trisomy 21
Interventions
Not listed
Lead sponsor
Roche Sequencing Solutions
Industry
Eligibility
18 Years to 60 Years · Female only
Enrollment
4,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2011
U.S. locations
6
States / cities
Oakland, California • Sacramento, California • San Diego, California + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 10, 2013 · Synced Oct 22, 2026, 4:14 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
Conditions
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Up to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 3, 2023 · Synced Oct 22, 2026, 4:14 PM EDT
Completed No phase listed Observational

Duchenne Muscular Dystrophy Video Assessment Registry

NCT05712447
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
The Emmes Company, LLC
Industry
Eligibility
2 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Korean Translation and Validation of the K-HEPI in NYC

NCT05544578
View directory record Official record
Conditions
Disaster, Vulnerable Populations
Interventions
Nurses Taking On Readiness Measures (N- TORM)
Behavioral
Lead sponsor
Hunter College of City University of New York
Other
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 15, 2024 · Synced Oct 22, 2026, 4:14 PM EDT
Recruiting Phase 1 Interventional

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

NCT06371417
View directory record Official record
Conditions
Antiphospholipid Syndrome (APS), Bullous Pemphigoid (BP), Behçet's Syndrome (BS), Dermatomyositis (DM), Immune-mediated Necrotizing Myopathy (IMNM), Immune Thrombocytopenia (ITP)
Interventions
RAY121
Drug
Lead sponsor
Chugai Pharmaceutical
Industry
Eligibility
18 Years to 85 Years
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
11
States / cities
Orange, California • Baltimore, Maryland • Lake Success, New York + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Recruiting No phase listed Observational

Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants

NCT05589714
View directory record Official record
Conditions
Inherited Retinal Degeneration, Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
Jaeb Center for Health Research
Other
Eligibility
4 Years and older
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
19
States / cities
Little Rock, Arkansas • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Recruiting Phase 1 Interventional

131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)

NCT07015684
View directory record Official record
Conditions
Sickling Disorder Due to Hemoglobin S
Interventions
131I-apamistmab, Sirolimus, Campath, Total Body Irradiation, Exchange Transfusion, Planar gamma imaging
Drug · Radiation · Procedure
Lead sponsor
Columbia University
Other
Eligibility
12 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Completed Phase 1 Interventional

Inhaled Sodium Pyruvate for the Treatment of Cystic Fibrosis.

NCT00308243
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Sodium Pyruvate in 0.9% Sodium Chloride Solution
Drug
Lead sponsor
Emphycorp
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 19, 2011 · Synced Oct 22, 2026, 4:14 PM EDT
Completed Phase 3 Interventional Results available

A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)

NCT03879135
View directory record Official record
Conditions
Von Willebrand Disease (VWD)
Interventions
rVWF, rFVIII
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
0 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
8
States / cities
Little Rock, Arkansas • Aurora, Colorado • Gainesville, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced Oct 22, 2026, 4:14 PM EDT
Active, not recruiting Phase 3 Interventional

A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A

NCT05685238
View directory record Official record
Conditions
Haemophilia A, Haemophilia A With Inhibitors
Interventions
Mim8
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Not listed
Enrollment
451 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
15
States / cities
Los Angeles, California • Aurora, Colorado • Miami, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Completed No phase listed Observational

Study of Megakaryocytes From Patients With Abnormal Platelet Vesicles

NCT00086476
View directory record Official record
Conditions
Blood Coagulation Disorders
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 22, 2026, 4:14 PM EDT
Terminated Phase 2 Interventional Results available

A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome

NCT03458416
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Cannabidiol Oral Solution
Drug
Lead sponsor
Radius Pharmaceuticals, Inc.
Industry
Eligibility
8 Years to 17 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Tacoma, Washington
Source: ClinicalTrials.gov public record
Updated Jun 22, 2023 · Synced Oct 22, 2026, 4:14 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Treatment to Lift Facial Lax Skin and Improve Facial Wrinkles Using Precise and Lift Applicators

NCT06333067
View directory record Official record
Conditions
Lax Skin
Interventions
Sofwave
Device
Lead sponsor
Sofwave Medical LTD
Industry
Eligibility
35 Years to 80 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
2
States / cities
La Mesa, California • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 13, 2025 · Synced Oct 22, 2026, 4:14 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Histological Evaluation of Human Skin Bx to Assess the Effects of APR Tx as an Adjunct Procedure in Facelift Surgery

NCT06243744
View directory record Official record
Conditions
Skin Laxity, Tissue Degeneration, Tissue Breakdown, Collagen Degeneration, Collagen Shrinkage
Interventions
Renuvion APR System
Device
Lead sponsor
Apyx Medical
Industry
Eligibility
18 Years to 75 Years
Enrollment
7 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
2
States / cities
Bradenton, Florida • Sarasota, Florida
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 22, 2026, 4:14 PM EDT
Completed Phase 2 Interventional Results available

Cixutumumab in Treating Patients With Relapsed or Refractory Solid Tumors

NCT00831844
View directory record Official record
Conditions
Adult Rhabdomyosarcoma, Adult Synovial Sarcoma, Childhood Hepatoblastoma, Childhood Synovial Sarcoma, Previously Treated Childhood Rhabdomyosarcoma, Recurrent Adrenocortical Carcinoma, Recurrent Adult Soft Tissue Sarcoma, Recurrent Childhood Liver Cancer, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive, Neuroectodermal Tumor, Recurrent Neuroblastoma, Recurrent Osteosarcoma, Recurrent Retinoblastoma, Recurrent Wilms Tumor and Other Childhood Kidney Tumors
Interventions
cixutumumab, laboratory biomarker analysis
Biological · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
7 Months to 30 Years
Enrollment
116 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
93
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Downey, California + 78 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2015 · Synced Oct 22, 2026, 4:14 PM EDT