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Showing 1–24 of 9,374 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Genetic Diseases Clear all
Completed Phase 2 Interventional

A Study of HQK-1001 in Patients With Sickle Cell Disease

NCT01322269
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S
Interventions
HQK-1001
Drug
Lead sponsor
HemaQuest Pharmaceuticals Inc.
Industry
Eligibility
12 Years to 60 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2011
U.S. locations
9
States / cities
Oakland, California • Miami, Florida • Augusta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 18, 2013 · Synced Sep 4, 2026, 7:10 PM EDT
Completed Phase 1 Interventional Results available

Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

NCT02530905
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
SRP-4045, Placebo
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 21 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Chicago, Illinois • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 16, 2021 · Synced Sep 4, 2026, 7:10 PM EDT
Completed No phase listed Observational Results available

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

NCT00937794
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Neurobehavioral testing, Visual and auditory assessments
Behavioral · Other
Lead sponsor
Shire
Industry
Eligibility
32 Months to 18 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Sep 4, 2026, 7:10 PM EDT
Not listed Not applicable Interventional

Histological Study of the Effects of a 2910 nm Fiber Laser Technology

NCT06317766
View directory record Official record
Conditions
Skin Laxity
Interventions
2910 nm Fiber Laser Treatment; Pre-Clinical Histology Evaluation, 2910 nm Fiber Laser Treatment; Part 2 Histology Evaluation
Device
Lead sponsor
FA Corporation
Industry
Eligibility
18 Years to 75 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 18, 2024 · Synced Sep 4, 2026, 7:10 PM EDT
Not yet recruiting Not applicable Interventional

Reach and Engagement of GENetic Education, Risk Assessment, and TEsting Feasibility Trial

NCT07787156
View directory record Official record
Conditions
Pancreatic Ductal Adenocarcinoma (PDAC), Family History of Pancreas Cancer, Genetic Testing, Multi-cancer Early Detection
Interventions
Telephone education session, Online educational materials
Behavioral
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Sep 4, 2026, 7:10 PM EDT
Completed No phase listed Observational

Educational Needs of Patients With Systemic Vasculitis

NCT02190929
View directory record Official record
Conditions
Behcet's Disease, Churg-Strauss Syndrome, Vasculitis, Central Nervous System, Giant Cell Arteritis, Wegener Granulomatosis, Henoch-Schoenlein Purpura, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
386 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jul 14, 2014 · Synced Sep 4, 2026, 7:10 PM EDT
Not listed No phase listed Observational

Studies in Porphyria I: Characterization of Enzyme Defects

NCT00004331
View directory record Official record
Conditions
Porphyria
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1992
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Sep 4, 2026, 7:10 PM EDT
Completed Phase 2 Interventional Results available

A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease

NCT02006472
View directory record Official record
Conditions
Huntington's Disease
Interventions
Pridopidine, Placebo
Drug · Other
Lead sponsor
Prilenia
Industry
Eligibility
21 Years and older
Enrollment
408 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
16
States / cities
La Jolla, California • Los Angeles, California • Englewood, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2021 · Synced Sep 4, 2026, 7:10 PM 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 16, 2017 · Synced Sep 4, 2026, 7:10 PM EDT
Recruiting Phase 3 Interventional

Compassionate Use Study of Epi-ON Corneal Collagen Crosslinking Performed Using UVA Exposure on Eyes With Ectatic Corneal Diseases for Subjects With Down Syndrome

NCT07135167
View directory record Official record
Conditions
Down Syndrome (DS), Keratoconus, Pellucid Marginal Degeneration, Forme Fruste Keratoconus (FFK)
Interventions
Epi-ON corneal cross-linking (CXL)
Combination Product
Lead sponsor
Woolfson Eye Institute
Other
Eligibility
8 Years and older
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
12
States / cities
Atlanta, Georgia • Canton, Georgia • Cumming, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2026 · Synced Sep 4, 2026, 7:10 PM EDT
Completed Phase 4 Interventional Results available

Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A

NCT00289536
View directory record Official record
Conditions
Hemophilia A
Interventions
Antihemophilic factor, recombinant, manufactured protein-free
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
12 Years to 65 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
8
States / cities
Little Rock, Arkansas • Los Angeles, California • Peoria, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 9, 2021 · Synced Sep 4, 2026, 7:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

NS-050/NCNP-03 in Boys With DMD (Meteor50)

NCT06053814
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-050/NCNP-03, Placebo
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 15 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
6
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Sep 4, 2026, 7:10 PM EDT
Completed No phase listed Observational

Study of Usher Syndromes, Type 1 and Type 2

NCT00001347
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 4, 2026, 7:10 PM EDT
Completed No phase listed Observational Results available

Genetic and Biochemical Markers of Interferon-Induced Depression.

NCT00252538
View directory record Official record
Conditions
Depression
Interventions
Not listed
Lead sponsor
US Department of Veterans Affairs
Federal
Eligibility
18 Years and older
Enrollment
133 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
3
States / cities
Long Beach, California • Minneapolis, Minnesota • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 10, 2014 · Synced Sep 4, 2026, 7:10 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy Participants

NCT06345755
View directory record Official record
Conditions
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Interventions
VX-407, Placebo, Midazolam
Drug
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
18 Years to 55 Years
Enrollment
159 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2025
U.S. locations
1
States / cities
Lenexa, Kansas
Source: ClinicalTrials.gov public record
Updated Jul 13, 2025 · Synced Sep 4, 2026, 7:10 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

NCT06181136
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA
Interventions
DNL126
Drug
Lead sponsor
Denali Therapeutics Inc.
Industry
Eligibility
0 Years to 18 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
4
States / cities
Oakland, California • Iowa City, Iowa • Chapel Hill, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 4, 2026, 7:10 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Risk Factors, Costs, and Impacts of ED Boarding

NCT07532564
View directory record Official record
Conditions
Stroke (CVA) or TIA, Diabetes, Asthma (Diagnosis), COPD (Chronic Obstructive Pulmonary Disease), Sepsis, Hypertension, Heart Failure, Pneumonia, Urinary Tract Infection (Diagnosis), Chest Pain, Psychiatric Disorder, Sickle Cell Disease (SCD)
Interventions
Not listed
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
18 Years and older
Enrollment
30,486 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Sep 4, 2026, 7:10 PM EDT
Terminated Phase 3 Interventional Results available

Study of Aztreonam for Inhalation in Children With Cystic Fibrosis and New Infection of the Airways by Pseudomonas Aeruginosa Bacteria

NCT03219164
View directory record Official record
Conditions
Pseudomonas Aeruginosa Respiratory Tract Infection/Colonization, Cystic Fibrosis
Interventions
AZLI, Placebo
Drug
Lead sponsor
Gilead Sciences
Industry
Eligibility
3 Months to 18 Years
Enrollment
149 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
12
States / cities
San Francisco, California • Washington D.C., District of Columbia • St. Petersburg, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2022 · Synced Sep 4, 2026, 7:10 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients

NCT05616793
View directory record Official record
Conditions
LCA5
Interventions
AAV8.hLCA5
Biological
Lead sponsor
Opus Genetics, Inc
Industry
Eligibility
4 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced Sep 4, 2026, 7:10 PM EDT
Completed Not applicable Interventional

Cluster Randomized Trial Comparing Interventions to Enhance Genetic Counseling Among Breast Cancer Patients

NCT01789684
View directory record Official record
Conditions
Breast Cancer
Interventions
Active Provider Intervention
Other
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older · Female only
Enrollment
3,780 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jan 31, 2021 · Synced Sep 4, 2026, 7:10 PM EDT
Completed Not applicable Interventional

MD2Me - Texting to Promote Chronic Disease Management

NCT01253733
View directory record Official record
Conditions
Cystic Fibrosis, Inflammatory Bowel Disease, Type 1 Diabetes, Chronic Disease Management
Interventions
MD2ME
Behavioral
Lead sponsor
Huang, Jeannie, M.D.
Individual
Eligibility
14 Years to 22 Years
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jan 25, 2016 · Synced Sep 4, 2026, 7:10 PM EDT
Completed No phase listed Observational

Post Traumatic Stress Disorder (PTSD), Sleep Disordered Breathing And Genetics: Effects On Cognition

NCT01120847
View directory record Official record
Conditions
Stress Disorders, Post-Traumatic
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
55 Years to 90 Years
Enrollment
324 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2017
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Feb 27, 2020 · Synced Sep 4, 2026, 7:10 PM EDT
Terminated Phase 2Phase 3 Interventional Results available

T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

NCT00176826
View directory record Official record
Conditions
Hemophagocytic Lymphohistiocytosis, X-Linked Lymphoproliferative Disorders, Chediak-Higashi Syndrome, Griscelli Syndrome, Immunologic Diseases, Langerhans-Cell Histiocytosis, Hematologic Diseases
Interventions
Stem Cell Transplant, Myeloablative conditioning regimen
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2015
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 22, 2018 · Synced Sep 4, 2026, 7:10 PM EDT
Completed Phase 4 Interventional Results available

Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE

NCT01457430
View directory record Official record
Conditions
Hereditary Angioedema
Interventions
Icatibant
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
5
States / cities
La Jolla, California • Los Angeles, California • Wheaton, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 17, 2016 · Synced Sep 4, 2026, 7:10 PM EDT