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ClinicalTrials.gov public records Last synced Oct 21, 2026, 10:32 PM EDT

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Showing 457–480 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Completed Phase 2 Interventional Results available

Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome

NCT02057692
View directory record Official record
Conditions
Alagille Syndrome
Interventions
LUM001, Placebo
Drug
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Eligibility
12 Months to 18 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
12
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 26, 2019 · Synced Oct 21, 2026, 10:32 PM EDT
Recruiting No phase listed Observational

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011
View directory record Official record
Conditions
Pompe Disease
Interventions
Cipaglucosidase alfa, Miglustat, Alglucosidase alfa or Avalglucosidase alfa, Untreated
Biological · Drug · Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2034
U.S. locations
15
States / cities
Little Rock, Arkansas • Irvine, California • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 21, 2026, 10:32 PM EDT
Completed Not applicable Interventional Results available

Comparison of MRI and Molecular Breast Imaging in Breast Diagnostic Evaluation

NCT00591864
View directory record Official record
Conditions
Breast Cancer
Interventions
Molecular Breast Imaging
Device
Lead sponsor
Mayo Clinic
Other
Eligibility
25 Years and older · Female only
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 9, 2014 · Synced Oct 21, 2026, 10:32 PM EDT
Completed No phase listed Observational

Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)

NCT01125709
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Cooperative International Neuromuscular Research Group
Network
Eligibility
6 Years to 18 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
3
States / cities
Washington D.C., District of Columbia • St Louis, Missouri • Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 11, 2013 · Synced Oct 21, 2026, 10:32 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Dissecting the Genetics of Fetal Alcohol Spectrum Disorders

NCT04072809
View directory record Official record
Conditions
Fetal Alcohol Spectrum Disorders
Interventions
No intervention
Other
Lead sponsor
Indiana University
Other
Eligibility
1 Month to 110 Years
Enrollment
751 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated May 6, 2023 · Synced Oct 21, 2026, 10:32 PM EDT
Withdrawn Not applicable Interventional

Effect of Topical and Systemic Decolonization of Staphylococcus Aureus (SA) in Pediatric Cystic Fibrosis (CF) Patients

NCT01229553
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
decolonization
Drug
Lead sponsor
State University of New York - Upstate Medical University
Other
Eligibility
2 Months to 23 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
1
States / cities
Syracuse, New York
Source: ClinicalTrials.gov public record
Updated Jun 11, 2015 · Synced Oct 21, 2026, 10:32 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major

NCT00480506
View directory record Official record
Conditions
Thalassemia Major
Interventions
Not listed
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2005
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 23, 2012 · Synced Oct 21, 2026, 10:32 PM EDT
Completed No phase listed Observational

Natural Disease Progression in Participants With Choroideremia

NCT04795206
View directory record Official record
Conditions
Choroideremia
Interventions
No Intervention
Other
Lead sponsor
Biogen
Industry
Eligibility
10 Years and older
Enrollment
1,178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 8, 2021 · Synced Oct 21, 2026, 10:32 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 21, 2026, 10:32 PM EDT
Completed Phase 1Phase 2 Interventional

Stem Cell Gene Therapy for Sickle Cell Disease

NCT02247843
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
βAS3-FB vector transduced peripheral blood CD34+ cells
Biological
Lead sponsor
Donald B. Kohn, M.D.
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Oct 21, 2026, 10:32 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 21, 2026, 10:32 PM EDT
Not listed Phase 1 Interventional

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

NCT03814408
View directory record Official record
Conditions
Fanconi Anemia Complementation Group A
Interventions
RP-L102
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Year to 12 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Nov 24, 2020 · Synced Oct 21, 2026, 10:32 PM EDT
Terminated Phase 2 Interventional Results available

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

NCT03943290
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
Interventions
ACE-083
Drug
Lead sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
19
States / cities
Orange, California • Sacramento, California • Aurora, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2022 · Synced Oct 21, 2026, 10:32 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

NCT03333590
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
rAAVrh74.MCK.GALGT2
Biological
Lead sponsor
Kevin Flanigan
Other
Eligibility
4 Years and older · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 13, 2025 · Synced Oct 21, 2026, 10:32 PM EDT
Completed Phase 3 Interventional Results available

Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)

NCT00288704
View directory record Official record
Conditions
Familial Cold Autoinflammatory Syndrome (FCAS), Familial Cold Urticaria, Muckle-Wells Syndrome (MWS), Genetic Diseases, Inborn
Interventions
rilonacept 160 mg, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
7 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
24
States / cities
Little Rock, Arkansas • Palm Desert, California • Upland, California + 21 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2011 · Synced Oct 21, 2026, 10:32 PM EDT
Active, not recruiting Phase 2 Interventional

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

NCT05418049
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Baclofen, Memantine, Roflumilast, Placebo
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 45 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 21, 2026, 10:32 PM EDT
Completed Not applicable Interventional Results available

Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria

NCT01428258
View directory record Official record
Conditions
Phenylketonuria
Interventions
GMP Diet/GMP Medical Foods, AA Diet/AA Medical Foods
Other
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
12 Years to 45 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
2
States / cities
Boston, Massachusetts • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Aug 24, 2018 · Synced Oct 21, 2026, 10:32 PM EDT
Terminated Phase 1 Interventional

Evaluation of K9 in Subjects With Thyroid Eye Disease (TED)

NCT06467435
View directory record Official record
Conditions
Thyroid Eye Disease
Interventions
Kamuvudine-9
Drug
Lead sponsor
Peter Timoney
Other
Eligibility
18 Years to 75 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Oct 21, 2026, 10:32 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

NCT01780168
View directory record Official record
Conditions
Oxidative Phosphorylation Deficiencies, Electron Transport Chain Disorders, Mitochondrial, Mitochondrial Disorders, Leigh Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
4 Weeks to 115 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 21, 2026, 10:32 PM EDT
Completed Phase 3 Interventional Results available

Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B

NCT02234310
View directory record Official record
Conditions
Hemophilia B
Interventions
rFIXFc
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
11
States / cities
Sacramento, California • Washington D.C., District of Columbia • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 25, 2022 · Synced Oct 21, 2026, 10:32 PM EDT
Not yet recruiting Not applicable Interventional Accepts healthy volunteers

Evaluate the Safety and Efficacy of a Topical Serum (AGE Serum) in Improving Visible Signs of Aging

NCT07216352
View directory record Official record
Conditions
Laxity; Skin
Interventions
AGE Serum
Other
Lead sponsor
Goldman, Butterwick, Fitzpatrick and Groff
Other
Eligibility
25 Years to 50 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Oct 28, 2025 · Synced Oct 21, 2026, 10:32 PM EDT
Enrolling by invitation Phase 2Phase 3 Interventional

HELIOS: Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP or XLP

NCT05883748
View directory record Official record
Conditions
Erythropoietic Protoporphyria
Interventions
DISC-1459
Drug
Lead sponsor
Disc Medicine, Inc
Industry
Eligibility
12 Years and older
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
11
States / cities
Birmingham, Alabama • San Francisco, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 9, 2026 · Synced Oct 21, 2026, 10:32 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Tolerability of SYNB1020-CP-001

NCT03179878
View directory record Official record
Conditions
Healthy Volunteer, Urea Cycle Disorder
Interventions
Placebo, SYNB1020
Drug
Lead sponsor
Synlogic
Industry
Eligibility
18 Years to 64 Years
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Brooklyn, Maryland
Source: ClinicalTrials.gov public record
Updated May 12, 2021 · Synced Oct 21, 2026, 10:32 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Development of the ORCA Communication Measure for Rett Syndrome

NCT04920110
View directory record Official record
Conditions
Rett Syndrome
Interventions
Not listed
Lead sponsor
Rett Syndrome Research Trust
Other
Eligibility
18 Years and older
Enrollment
272 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 22, 2022 · Synced Oct 21, 2026, 10:32 PM EDT