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ClinicalTrials.gov public records Last synced Oct 8, 2026, 1:33 PM EDT

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Showing 73–96 of 9,262 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Diseases Genetic, Genetic Clear all
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 8, 2026, 1:33 PM EDT
Completed No phase listed Observational

Studies in Patients With Tuberous Sclerosis Complex

NCT03276195
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Translational Genomics Research Institute
Other
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 29, 2023 · Synced Oct 8, 2026, 1:33 PM EDT
Recruiting Not applicable Interventional

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

NCT07521930
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 2 Interventional Results available

Topical Tazarotene in Treating Patients With Basal Cell Skin Cancer and Basal Cell Nevus Syndrome on the Chest and Back

NCT00783965
View directory record Official record
Conditions
Skin Carcinoma
Interventions
tazarotene, placebo
Drug · Other
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
18 Years to 75 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2014
U.S. locations
3
States / cities
Oakland, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 11, 2016 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 2 Interventional Results available

Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients

NCT03474965
View directory record Official record
Conditions
Sickle Cell Disease (SCD)
Interventions
Crizanlizumab
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
117 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
12
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2025 · Synced Oct 8, 2026, 1:33 PM EDT
Recruiting Phase 4 Interventional

Hemlibra in Mild Hemophilia A

NCT04567511
View directory record Official record
Conditions
Factor VIII Deficiency, Congenital
Interventions
Emicizumab
Drug
Lead sponsor
Indiana Hemophilia &Thrombosis Center, Inc.
Other
Eligibility
5 Years to 45 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Mar 15, 2024 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 2 Interventional Results available

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

NCT01520363
View directory record Official record
Conditions
Rett Syndrome
Interventions
dextromethorphan, placebo
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
1 Year to 10 Years
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 4, 2018 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 1Phase 2 Interventional

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE

NCT05030831
View directory record Official record
Conditions
ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
Interventions
INZ-701
Drug
Lead sponsor
Inozyme Pharma
Industry
Eligibility
18 Years to 69 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 5, 2025 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 3 Interventional Results available

Trial of Netarsudil for Prevention of Corticosteroid-induced Intraocular Pressure Elevation

NCT03248037
View directory record Official record
Conditions
Fuchs' Endothelial Dystrophy, Bullous Keratopathy
Interventions
Netarsudil, Placebo
Drug
Lead sponsor
Cornea Research Foundation of America
Other
Eligibility
18 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 12, 2021 · Synced Oct 8, 2026, 1:33 PM EDT
Terminated Phase 3 Interventional Results available

Evaluating the Effects of Tradipitant vs. Placebo in Atopic Dermatitis (EPIONE2)

NCT04140695
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Tradipitant, Placebo
Drug
Lead sponsor
Vanda Pharmaceuticals
Industry
Eligibility
18 Years to 70 Years
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
51
States / cities
Hoover, Alabama • Tempe, Arizona • Encino, California + 43 more
Source: ClinicalTrials.gov public record
Updated May 14, 2024 · Synced Oct 8, 2026, 1:33 PM EDT
Active, not recruiting Phase 2 Interventional

Open-Label Study of AG10 in Patients with Cardiomyopathy

NCT03536767
View directory record Official record
Conditions
Amyloid Cardiomyopathy
Interventions
AG10
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
9
States / cities
Palo Alto, California • San Francisco, California • New Haven, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2025 · Synced Oct 8, 2026, 1:33 PM EDT
Active, not recruiting No phase listed Observational

Validating Innovative Biosensors for Rett Autonomic Symptom Tracking

NCT06338267
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
2
States / cities
Chicago, Illinois • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 3 Interventional

Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

NCT00037557
View directory record Official record
Conditions
Hemophilia B
Interventions
BeneFIX
Drug
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
Up to 5 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
6
States / cities
Aurora, Colorado • Detroit, Michigan • New Brunswick, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2007 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 3 Interventional

Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care

NCT00023192
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
T-Cell Depleted & CD34+Select/w/StemCell Enriched Product
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 8, 2026, 1:33 PM EDT
Completed No phase listed Observational

A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)

NCT06064656
View directory record Official record
Conditions
Fibrodysplasia Ossificans Progressiva (FOP)
Interventions
Non-Interventional
Other
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Tarrytown, New York
Source: ClinicalTrials.gov public record
Updated Jul 3, 2025 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 2 Interventional Results available

Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease

NCT02561702
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Placebo, Mexiletine
Other · Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 26, 2018 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of EUR-1008 (APT-1008) Pancreatic Enzyme Product in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency

NCT00297167
View directory record Official record
Conditions
Cystic Fibrosis, Exocrine Pancreatic Insufficiency
Interventions
EUR-1008 (APT-1008), Placebo
Drug
Lead sponsor
Forest Laboratories
Industry
Eligibility
7 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006
U.S. locations
1
States / cities
Tyler, Texas
Source: ClinicalTrials.gov public record
Updated Mar 16, 2017 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 3 Interventional Results available

Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia

NCT00006400
View directory record Official record
Conditions
Hematologic Diseases, Anemia, Sickle Cell
Interventions
Hydroxyurea, Placebo
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
9 Months to 18 Months
Enrollment
193 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
14
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2020 · Synced Oct 8, 2026, 1:33 PM 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 8, 2026, 1:33 PM EDT
Completed No phase listed Observational

Retrospective Study to Capture Dosing and Treatment Outcome Data in Participants With Severe Congenital Protein C Deficiency Who Were Treated With Protein C Concentrate Under an Emergency Use IND

NCT00161720
View directory record Official record
Conditions
Protein C Deficiency
Interventions
Protein C Concentrate (Human) Vapor Heated
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005
U.S. locations
10
States / cities
Los Angeles, California • Denver, Colorado • Fort Carson, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2021 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers

Effect of Genetics on Metabolism of Efavirenz

NCT00245986
View directory record Official record
Conditions
Metabolism
Interventions
Blood draw
Procedure
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 55 Years
Enrollment
350 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 8, 2026, 1:33 PM EDT
Withdrawn Not applicable Interventional

FFP Versus PCC in Intracranial Hemorrhage

NCT02429453
View directory record Official record
Conditions
Intracranial Hemorrhage, Traumatic, Intracranial Hemorrhage, Spontaneous
Interventions
Four Factor Prothrombin Complex Concentrate, Fresh Frozen Plasma
Drug · Biological
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Nov 16, 2016 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral ABP-745 Administration in Healthy Volunteers

NCT06258213
View directory record Official record
Conditions
Gout, Acute Gout Flare, Gout Flares
Interventions
ABP-745, Placebo
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 55 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Aug 17, 2025 · Synced Oct 8, 2026, 1:33 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Pilot Mouthwash Study of Pioglitazone and Simvastatin in Healthy Volunteers

NCT00531882
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Pioglitazone, Simvastatin, Ibuprofen
Drug
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2009
U.S. locations
2
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 5, 2017 · Synced Oct 8, 2026, 1:33 PM EDT