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ClinicalTrials.gov public records Last synced Oct 21, 2026, 1:12 AM EDT

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Showing 1–24 of 9,413 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Disorders Clear all
Completed Phase 1Phase 2 Interventional Results available

A First in Human Study of RT001 in Patients With Friedreich's Ataxia

NCT02445794
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Low dose cohort, High dose cohort
Drug
Lead sponsor
Biojiva LLC
Industry
Eligibility
18 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Long Beach, California • Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Nov 27, 2020 · Synced Oct 21, 2026, 1:12 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia

NCT00630253
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Anti-Thymocyte Globulin, Cyclophosphamide, Fludarabine, Hematopoietic Stem Cell Transplantation, Methylprednisolone, Filgrastim, Cyclosporine, Mycophenolate Mofetil
Biological · Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 59 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 12, 2021 · Synced Oct 21, 2026, 1:12 AM EDT
Not yet recruiting Phase 1 Interventional

High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

NCT07612345
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
Vitamin C
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 21, 2026, 1:12 AM EDT
Recruiting Phase 2 Interventional

Cognitive Remediation Intervention to Prepare for Transition of Care

NCT05336201
View directory record Official record
Conditions
Sickle Cell Disease, Cognitive Impairment, Adolescent Behavior, Self Efficacy, Health-Related Behavior, Coping Skills
Interventions
CREADY (Cognitive-Remediation of Executive and Adaptive Deficits in Youth)
Behavioral
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
10 Years to 18 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 21, 2026, 1:12 AM EDT
Completed No phase listed Observational

Blood Sample Donations to Study the Role of Genes in Pain

NCT00341367
View directory record Official record
Conditions
Pain
Interventions
Not listed
Lead sponsor
National Institute on Alcohol Abuse and Alcoholism (NIAAA)
NIH
Eligibility
Not listed
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2010
U.S. locations
1
States / cities
South Portland, Maine
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 21, 2026, 1:12 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease

NCT04536792
View directory record Official record
Conditions
Healthy Volunteers, Anemia, Sickle Cell
Interventions
AG-946, AG-946 Matched Placebo
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
122 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
8
States / cities
La Jolla, California • Boston, Massachusetts • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2024 · Synced Oct 21, 2026, 1:12 AM EDT
Active, not recruiting Phase 3 Interventional

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Cipaglucosidase Alfa, Miglustat
Biological · Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
0 Years to 17 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
9
States / cities
Gainesville, Florida • Jacksonville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 21, 2026, 1:12 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Retinal Imaging in Patients With Inherited Retinal Degenerations

NCT00254605
View directory record Official record
Conditions
Retinitis Pigmentosa
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
13 Years and older
Enrollment
400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2028
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jun 5, 2026 · Synced Oct 21, 2026, 1:12 AM EDT
Not listed Not applicable Interventional

Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders

NCT00006056
View directory record Official record
Conditions
Chediak-Higashi Syndrome, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, Virus-Associated Hemophagocytic Syndrome
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, filgrastim, methotrexate, allogeneic hematopoietic stem cell transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 1:12 AM EDT
Completed No phase listed Observational

Natural History and Functional Status Study of Patients With Lafora Disease

NCT03876522
View directory record Official record
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Oct 21, 2026, 1:12 AM EDT
Completed Phase 3 Interventional Results available

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

NCT03417245
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
fitusiran, factor concentrates
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
12 Years and older · Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
8
States / cities
Little Rock, Arkansas • Gainesville, Florida • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2022 · Synced Oct 21, 2026, 1:12 AM EDT
Terminated No phase listed Observational

Genetic Study of Brain Tumors in Young Children

NCT00010101
View directory record Official record
Conditions
Central Nervous System Tumor, Pediatric
Interventions
Not listed
Lead sponsor
Pediatric Brain Tumor Consortium
Network
Eligibility
Up to 3 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
4
States / cities
Washington D.C., District of Columbia • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2011 · Synced Oct 21, 2026, 1:12 AM EDT
Completed Phase 2 Interventional Results available

Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

NCT06334419
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Gaboxadol, Placebo
Drug
Lead sponsor
Craig Erickson
Other
Eligibility
18 Years to 40 Years · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 21, 2026, 1:12 AM EDT
Completed Not applicable Interventional

Voriconazole Compared With Itraconazole in Preventing Fungal Infections in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

NCT00079222
View directory record Official record
Conditions
Cancer
Interventions
itraconazole, voriconazole
Drug
Lead sponsor
Jonsson Comprehensive Cancer Center
Other
Eligibility
12 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Aug 3, 2020 · Synced Oct 21, 2026, 1:12 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetic Studies in Breast Cancer

NCT00582322
View directory record Official record
Conditions
Breast Cancer, Ovarian Cancer
Interventions
Not listed
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
Female only
Enrollment
483 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1992 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 20, 2009 · Synced Oct 21, 2026, 1:12 AM EDT
Completed Phase 2 Interventional

Imaging and Genetic Biomarkers of Parkinson Disease (PD) Onset and Progression in High-risk Families

NCT00273351
View directory record Official record
Conditions
Parkinson Disease, Parkinsonian Syndrome
Interventions
[123I]B-CIT
Drug
Lead sponsor
Institute for Neurodegenerative Disorders
Other
Eligibility
21 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated May 9, 2014 · Synced Oct 21, 2026, 1:12 AM EDT
Terminated No phase listed Observational Results available

Collecting and Studying Tissue Samples From Patients With HIV-Associated Malignancies

NCT01567722
View directory record Official record
Conditions
Lung Cancer, Lymphoma
Interventions
DNA analysis, RNA analysis, gene expression analysis, polymorphism analysis, biologic sample preservation procedure, flow cytometry, medical chart review
Genetic · Other
Lead sponsor
AIDS Malignancy Consortium
Network
Eligibility
18 Years and older
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
14
States / cities
La Jolla, California • Los Angeles, California • Washington D.C., District of Columbia + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 16, 2023 · Synced Oct 21, 2026, 1:12 AM EDT
Terminated No phase listed Observational

Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

NCT01541722
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
Not listed
Lead sponsor
Mark Batshaw
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
10
States / cities
Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2015 · Synced Oct 21, 2026, 1:12 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

GLASSIA Infusion Rate Study

NCT01651351
View directory record Official record
Conditions
Alpha1-antitrypsin Deficiency, Healthy Volunteers
Interventions
Alpha1-proteinase inhibitor, Placebo: Human albumin 2.5%
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 65 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated May 25, 2021 · Synced Oct 21, 2026, 1:12 AM EDT
Withdrawn Phase 1Phase 2 Interventional Accepts healthy volunteers

Evaluation of 20% Betulinic Acid Ointment for Treatment of Dysplastic Nevi (Moderate to Severe Dysplasia)

NCT00346502
View directory record Official record
Conditions
Dysplastic Nevus Syndrome
Interventions
20% betulinic acid ointment, BA
Drug
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
18 Years and older
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Oct 21, 2026, 1:12 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Improving the Diagnosis of Common Variable Immune Deficiency

NCT03335605
View directory record Official record
Conditions
CVI - Common Variable Immunodeficiency
Interventions
Not listed
Lead sponsor
University of California, Los Angeles
Other
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2022
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Oct 21, 2026, 1:12 AM EDT
Completed Phase 1 Interventional

The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes

NCT01319331
View directory record Official record
Conditions
Diabetes, Type 1 Diabetes
Interventions
Alpha 1-Antitrypsin (AAT, Aralast NP)
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
6 Years to 45 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 24, 2017 · Synced Oct 21, 2026, 1:12 AM EDT
Terminated Phase 3 Interventional Accepts healthy volunteers Results available

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

NCT04221451
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease
Interventions
venglustat GZ402671, placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
2 Years and older
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2024
U.S. locations
5
States / cities
Los Angeles, California • Atlanta, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 21, 2026, 1:12 AM EDT
Not listed No phase listed Observational

MMRF Molecular Profiling Protocol

NCT02884102
View directory record Official record
Conditions
Multiple Myeloma, Plasma Malignancy
Interventions
genetic sequencing
Genetic
Lead sponsor
Multiple Myeloma Research Foundation
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
19
States / cities
Scottsdale, Arizona • Duarte, California • San Francisco, California + 15 more
Source: ClinicalTrials.gov public record
Updated May 6, 2025 · Synced Oct 21, 2026, 1:12 AM EDT