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ClinicalTrials.gov public records Last synced Oct 25, 2026, 5:25 PM EDT

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Showing 1273–1296 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Enrolling by invitation Phase 2 Interventional

An Open-label Long-term Follow-up Study of SAT-3247 for Participants With Duchenne Muscular Dystrophy Including Those Who Participated in SAT-3247-CL-101

NCT06867107
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
SAT-3247
Drug
Lead sponsor
Satellos Bioscience, Inc.
Industry
Eligibility
16 Years to 40 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
4
States / cities
Los Angeles, California • St Louis, Missouri • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Pivotal Study to Evaluate the Safety and Efficacy of a Micro-coring Device Treating Moderate to Severe Facial Wrinkles

NCT03573271
View directory record Official record
Conditions
Laxity; Skin
Interventions
MCD
Device
Lead sponsor
Cytrellis Biosystems, Inc.
Industry
Eligibility
40 Years to 70 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
5
States / cities
Sacramento, California • Miami, Florida • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 9, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Phase 2 Interventional

Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

NCT06023199
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Voxelotor
Drug
Lead sponsor
Inova Health Care Services
Other
Eligibility
18 Years to 65 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Falls Church, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Not listed Phase 1 Interventional

An Open-label Trial of Intravenous Immune Globulin (IVIG)in Treating Spinocerebellar Ataxias

NCT02287064
View directory record Official record
Conditions
Spinocerebellar Ataxias
Interventions
Intravenous Immune Globulin (IVIG)
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2015
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 8, 2016 · Synced Oct 25, 2026, 5:25 PM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

A Cross-Sectional Natural History Study to Evaluate Sweat Volume and Other Phenotypic and Genetic Characteristics in Patients Affected by XLHED

NCT03912792
View directory record Official record
Conditions
XLHED
Interventions
Not listed
Lead sponsor
Dermelix Biotherapeutics, LLC.
Industry
Eligibility
Up to 80 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Lincolnshire, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 2, 2019 · Synced Oct 25, 2026, 5:25 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Oral Metformin for Treatment of ABCA4 Retinopathy

NCT04545736
View directory record Official record
Conditions
ABCA4 Retinopathy, Stargardt Disease, Retinal Dystrophy, Retinal Degeneration
Interventions
Metformin hydrochloride
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
12 Years to 100 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2029
U.S. locations
2
States / cities
Bethesda, Maryland • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Oct 22, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Recruiting Phase 2 Interventional

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

NCT03579875
View directory record Official record
Conditions
Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion, Telomere Biology Disorder, Bone Marrow Failure, Dyskeratosis Congenita, Telomere Biology Disorders
Interventions
Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU), Methylprednisolone (MP), Donor mobilized PBSC infusion, G-CSF, Cyclophosphamide (CY) (Plan 2), Rituximab, Busulfan, Alemtuzumab, Melphalan
Drug · Device
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 65 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

CF And Effects of Drugs Mixed Ex Vivo With Sputum for Mucolytic Treatment

NCT01533636
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 65 Years
Enrollment
114 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2026
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 10, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Phase 1 Interventional

A Phase I Study of Vorinostat and Bortezomib in Children With Refractory of Recurrent Solid Tumors, Including CNS Tumors and Lymphomas

NCT01132911
View directory record Official record
Conditions
Lymphoma, Sarcoma, WilmsTumor, Neuroblastoma
Interventions
Vorinostat (SAHA), Velcade (PS-341, Bortezomib)
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 21 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 25, 2026, 5:25 PM EDT
Recruiting Phase 2 Interventional

A Study of CSTI-500 in Patients With Prader-Willi Syndrome

NCT07348601
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
CSTI-500
Drug
Lead sponsor
ConSynance Therapeutics
Industry
Eligibility
13 Years to 50 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Oct 21, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Phase 3 Interventional Results available

Study of Abrocitinib Compared With Dupilumab in Adults With Moderate to Severe Atopic Dermatitis on Background Topical Therapy

NCT04345367
View directory record Official record
Conditions
Atopic Dermatitis
Interventions
Abrocitinib 200 mg, Dupilumab 300 mg
Drug · Combination Product
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
727 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
49
States / cities
Birmingham, Alabama • Phoenix, Arizona • Fountain Valley, California + 39 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2022 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Phase 3 Interventional Results available

A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH)

NCT00730236
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
AEGR-733
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 20, 2018 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Phase 1Phase 2 Interventional

Phase 1 and 2 Study of PX-866 and Cetuximab

NCT01252628
View directory record Official record
Conditions
Incurable Metastatic Colorectal Carcinoma, Incurable Progressive, Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
Interventions
PX-866 (SCCHN), Cetuximab (SCCHN), PX-866 (CRC), Cetuximab (CRC)
Drug
Lead sponsor
Cascadian Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
31
States / cities
Birmingham, Alabama • Escondido, California • Monterey, California + 24 more
Source: ClinicalTrials.gov public record
Updated May 16, 2018 · Synced Oct 25, 2026, 5:25 PM EDT
Completed No phase listed Observational

Using Positron Emission Tomography to Predict Intracranial Tumor Growth in Neurofibromatosis Type II Patients

NCT01222728
View directory record Official record
Conditions
Neoplasms, Nervous System Disease, Vestibular Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 50 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Phase 1Phase 2 Interventional

A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency

NCT02488044
View directory record Official record
Conditions
Arginase I Deficiency, Hyperargininemia
Interventions
AEB1102
Drug
Lead sponsor
Aeglea Biotherapeutics
Industry
Eligibility
2 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
6
States / cities
Stanford, California • Gainesville, Florida • Detroit, Michigan + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2019 · Synced Oct 25, 2026, 5:25 PM EDT
Completed No phase listed Observational

Clinical, Cellular, and Molecular Investigation Into Oculocutaneous Albinism

NCT00808106
View directory record Official record
Conditions
Oculocutaneous Albinism
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Year to 80 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 6, 2020 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Phase 1 Interventional Results available

Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease

NCT03664804
View directory record Official record
Conditions
Huntington's Disease
Interventions
No Study Drug was Administered in this Study
Other
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years to 65 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
6
States / cities
Englewood, Colorado • Washington D.C., District of Columbia • Wichita, Kansas + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 3, 2025 · Synced Oct 25, 2026, 5:25 PM EDT
Completed No phase listed Observational

G551D Observational Study- Expanded to Additional Genotypes and Extended for Long Therm Follow up (GOAL-e2)

NCT01521338
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
Not listed
Lead sponsor
Steven M Rowe
Other
Eligibility
6 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
36
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 31 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2021 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Not applicable Interventional Results available

Evaluation of the Gastrointestinal Manifestation of Fabry's Disease

NCT02798458
View directory record Official record
Conditions
Fabry's Disease
Interventions
Smartpill, Endoscopic Mucosal Resection
Device · Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Not applicable Interventional Results available

Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome

NCT01444898
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
Exenatide
Drug
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
13 Years to 20 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Sep 29, 2016 · Synced Oct 25, 2026, 5:25 PM EDT
Completed Phase 2 Interventional Results available

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898
View directory record Official record
Conditions
Metachromatic Leukodystrophy (MLD)
Interventions
SHP611
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Months to 72 Months
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
11
States / cities
Torrance, California • Aurora, Colorado • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Recruiting Phase 2 Interventional

A Study Evaluating Ficerafusp Alfa (BCA101) QW in Combination With Pembrolizumab vs Alternative Ficerafusp Alfa Dosing in 1L R or M HNSCC

NCT07807163
View directory record Official record
Conditions
Metastatic Head and Neck Squamous Cell Carcinoma, Recurrent Head and Neck Squamous Cell Carcinoma
Interventions
Ficerafusp alfa, Pembrolizumab (KEYTRUDA®)
Drug
Lead sponsor
Bicara Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
7
States / cities
Fort Myers, Florida • Orlando, Florida • St. Petersburg, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Recruiting Phase 2 Interventional

A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1

NCT06541847
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
HLX-1502
Drug
Lead sponsor
Healx Limited
Industry
Eligibility
12 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
14
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 25, 2026, 5:25 PM EDT
Terminated Phase 3 Interventional

Carotid B-mode Ultrasound Study to Compare Anti-Atherosclerotic Effect of Torcetrapib/Atorvastatin to Atorvastatin.

NCT00134238
View directory record Official record
Conditions
Mixed Hyperlipidemia
Interventions
torcetrapib/atorvastatin, atorvastatin
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 70 Years
Enrollment
755 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
40
States / cities
Gilbert, Arizona • Phoenix, Arizona • Scottsdale, Arizona + 37 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2012 · Synced Oct 25, 2026, 5:25 PM EDT