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ClinicalTrials.gov public records Last synced Oct 20, 2026, 10:12 PM EDT

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

Showing 289–312 of 8,005 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed No phase listed Observational

Effects of Low-density Lipoprotein (LDL) Apheresis on Inflammatory and Lipid Markers

NCT01138371
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
LDL Apheresis
Procedure
Lead sponsor
Emory University
Other
Eligibility
3 Years to 70 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 17, 2013 · Synced Oct 20, 2026, 10:12 PM EDT
Completed Phase 2 Interventional

Niacin to Improve Blood Flow in People With Sickle Cell Disease

NCT00508989
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Niacin-ER, Placebo, L-NMMA, Acetylcholine
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2015
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 10, 2020 · Synced Oct 20, 2026, 10:12 PM EDT
Completed No phase listed Observational

Review of Kidney Biopsies of Dent Disease Patients

NCT02022189
View directory record Official record
Conditions
Dent Disease
Interventions
Renal Biopsy
Procedure
Lead sponsor
Mayo Clinic
Other
Eligibility
Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Sep 22, 2015 · Synced Oct 20, 2026, 10:12 PM EDT
Terminated Phase 4 Interventional

Intraduodenal Aspiration Study to Assess the Bioavailability of Oral Pancrecarb® Compared to Placebo Control

NCT00744250
View directory record Official record
Conditions
Exocrine Pancreatic Insufficiency, Chronic Pancreatitis, Cystic Fibrosis
Interventions
Pancrelipase
Drug
Lead sponsor
Digestive Care, Inc.
Industry
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 22, 2013 · Synced Oct 20, 2026, 10:12 PM EDT
Completed Not applicable Interventional Results available

A Translational Approach to Gitelman Syndrome

NCT00822107
View directory record Official record
Conditions
Gitelman Syndrome
Interventions
Hydrochlorothiazide
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
21 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 5, 2019 · Synced Oct 20, 2026, 10:12 PM EDT
Active, not recruiting No phase listed Observational

A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)

NCT05981092
View directory record Official record
Conditions
Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
6
States / cities
Palo Alto, California • Aurora, Colorado • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 20, 2026, 10:12 PM EDT
Completed Early Phase 1 Interventional Results available

RHD Genotype Matched Red Cells for Anti-D

NCT04156906
View directory record Official record
Conditions
Sickle Cell Disease, Anti-D Antibodies
Interventions
D+ RH genotype matched red cell units for transfusion
Biological
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
8 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 2, 2025 · Synced Oct 20, 2026, 10:12 PM EDT
Completed No phase listed Observational

Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)

NCT00111384
View directory record Official record
Conditions
Neurofibromatosis Type 1, Legius Syndrome
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
313 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 20, 2026, 10:12 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer

NCT06764485
View directory record Official record
Conditions
Metastatic Castration-resistant Prostate Cancer
Interventions
BMS-986365, Enzalutamide, Abiraterone, Docetaxel, Predinsone/Prednisolone
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older · Male only
Enrollment
960 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
48
States / cities
Birmingham, Alabama • Gilbert, Arizona • Anaheim, California + 34 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 20, 2026, 10:12 PM EDT
Not listed No phase listed Observational

Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

NCT01772043
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
4 Years and older · Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
8
States / cities
Sacramento, California • Stanford, California • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2015 · Synced Oct 20, 2026, 10:12 PM EDT
Completed Not applicable Interventional

Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1

NCT05377008
View directory record Official record
Conditions
Neurocognitive Deficit, Mental Health Impairment, Neurofibromatosis 1
Interventions
OPTIMAL-SSI
Behavioral
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
6 Years to 16 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 22, 2023 · Synced Oct 20, 2026, 10:12 PM EDT
Completed Early Phase 1 Interventional

A Pilot Study of the Safety and Efficacy of Abatacept Injections in the Treatment of Behcet's Syndrome

NCT01693640
View directory record Official record
Conditions
Female Patients With Behcet's Syndrome
Interventions
Abatacept
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older · Female only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 28, 2019 · Synced Oct 20, 2026, 10:12 PM EDT
Recruiting Phase 2 Interventional Accepts healthy volunteers

Efficacy of Guselkumab in Treating Hailey Hailey Disease

NCT06651489
View directory record Official record
Conditions
Hailey Hailey Disease
Interventions
Guselkumab
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 90 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 20, 2026, 10:12 PM EDT
Completed No phase listed Observational

Clinical Care of Autosomal Polycystic Kidney Disease: Retrospective Analysis and Prospective PKD Genotyping

NCT02161068
View directory record Official record
Conditions
ADPKD
Interventions
Not listed
Lead sponsor
The Rogosin Institute
Other
Eligibility
18 Years and older
Enrollment
79 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 2, 2022 · Synced Oct 20, 2026, 10:12 PM EDT
Terminated Not applicable Interventional Results available

Neuromodulation for Children With Cystic Fibrosis Experiencing Chronic Abdominal Pain

NCT05515250
View directory record Official record
Conditions
Cystic Fibrosis, Irritable Bowel Syndrome, Chronic Pain
Interventions
Use of neurostimulation device
Device
Lead sponsor
St. Louis University
Other
Eligibility
11 Years to 18 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 9, 2024 · Synced Oct 20, 2026, 10:12 PM EDT
Recruiting Not applicable Interventional

Milk for Diabetes Prevention

NCT06513026
View directory record Official record
Conditions
Lactose Intolerance, Lactose Intolerant, Lactase Persistence, Pre-Diabetes, Diabetes Mellitus, Type 2
Interventions
Lactose-Containing Milk, Lactose-Free Milk
Dietary Supplement
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
18 Years to 70 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Oct 20, 2026, 10:12 PM EDT
Terminated Phase 2 Interventional Results available

A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease

NCT02494778
View directory record Official record
Conditions
Huntington's Disease
Interventions
Pridopidine
Drug
Lead sponsor
Prilenia
Industry
Eligibility
21 Years and older
Enrollment
248 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
11
States / cities
Los Angeles, California • Englewood, Colorado • Washington D.C., District of Columbia + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2021 · Synced Oct 20, 2026, 10:12 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Simulines Non-Inferiority Pivotal Study

NCT02736825
View directory record Official record
Conditions
Skin Laxity
Interventions
Ulthera System with standard transducer, Ulthera System with prototype 2 simulines transducer
Device
Lead sponsor
Ulthera, Inc
Industry
Eligibility
30 Years to 70 Years
Enrollment
262 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2017
U.S. locations
8
States / cities
Beverly Hills, California • Roseville, California • Sacramento, California + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 19, 2020 · Synced Oct 20, 2026, 10:12 PM EDT
Recruiting No phase listed Observational

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

NCT06192576
View directory record Official record
Conditions
Niemann-Pick Diseases, Acid Sphingomyelinase Deficiency
Interventions
Olipudase alfa
Drug
Lead sponsor
Sanofi
Industry
Eligibility
Up to 2 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
6
States / cities
Chicago, Illinois • Cincinnati, Ohio • Columbus, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 20, 2026, 10:12 PM EDT
Terminated Not applicable Interventional Results available

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

NCT02156674
View directory record Official record
Conditions
Maroteaux-Lamy Syndrome
Interventions
Naglazyme®
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
3 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2020 · Synced Oct 20, 2026, 10:12 PM EDT
Completed No phase listed Observational

Journey of Patients With Vasculitis From First Symptom to Diagnosis

NCT03410290
View directory record Official record
Conditions
Vasculitis, Systemic Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis, Eosinophilic Granulomatous Vasculitis, Temporal Arteritis, Giant Cell Arteritis, Granulomatosis With Polyangiitis, Wegener Granulomatosis, Henoch Schonlein Purpura, IgA Vasculitis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis, Urticarial Vasculitis
Interventions
Online Questionnaire
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
456 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 6, 2018 · Synced Oct 20, 2026, 10:12 PM EDT
Withdrawn No phase listed Observational

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 8, 2015 · Synced Oct 20, 2026, 10:12 PM EDT
Terminated Phase 3 Interventional Results available

Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031

NCT03573882
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Voxelotor
Drug
Lead sponsor
Pfizer
Industry
Eligibility
12 Years to 65 Years
Enrollment
179 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
34
States / cities
Little Rock, Arkansas • Oakland, California • Miami, Florida + 20 more
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Oct 20, 2026, 10:12 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599
View directory record Official record
Conditions
Gaucher Disease, Gaucher Disease, Type 1
Interventions
LY3884961
Genetic
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
2
States / cities
Durham, North Carolina • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 20, 2026, 10:12 PM EDT