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ClinicalTrials.gov public records Last synced Oct 22, 2026, 4:03 AM EDT

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Showing 505–528 of 8,007 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetics/Birth Defects Clear all
Terminated Phase 2 Interventional

Open Label Extension Study of HT-100 in Patients With DMD

NCT01978366
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
HT-100
Drug
Lead sponsor
Processa Pharmaceuticals
Industry
Eligibility
6 Years to 20 Years · Male only
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
Sacramento, California • Baltimore, Maryland • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2020 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Nurses Taking On Readiness Measures - Mercer County, New Jersey

NCT05455580
View directory record Official record
Conditions
Disaster, Vulnerable Populations
Interventions
Nurses Taking On Readiness Measures (N-TORM)
Behavioral
Lead sponsor
Hunter College of City University of New York
Other
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 5, 2024 · Synced Oct 22, 2026, 4:03 AM EDT
Active, not recruiting Phase 1 Interventional

The Impact of Botox on Neuroimmune Interactions in Atopic Dermatitis

NCT06928246
View directory record Official record
Conditions
Atopic Dermatitis (AD)
Interventions
Onabotulinum Toxin Type A - Phase 1b, Onabotulinum Toxin Type A - Phase 2
Drug
Lead sponsor
Daniel Kaplan
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 22, 2026, 4:03 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers

A Comprehensive Care Plan for Pediatric Patients With Vaso-Occlusive Crises

NCT02698761
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Crisis, Vaso-Occlusive Crises
Interventions
Comprehensive Care Plan
Other
Lead sponsor
Valley Anesthesiology Consultants
Other
Eligibility
12 Years to 18 Years
Enrollment
3 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2018
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated May 3, 2018 · Synced Oct 22, 2026, 4:03 AM EDT
Suspended Phase 1Phase 2 Interventional

RGX-111 Gene Therapy in Patients With MPS I

NCT03580083
View directory record Official record
Conditions
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Interventions
RGX-111
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 22, 2026, 4:03 AM EDT
Recruiting Phase 1 Interventional

Testing for Safety and Colorectal Cancer Preventive Effects of ONC201

NCT05630794
View directory record Official record
Conditions
Colorectal Adenomatous Polyp, Colorectal Carcinoma, Familial Adenomatous Polyposis, Multiple Adenomatous Polyps
Interventions
Biopsy Procedure, Biospecimen Collection, Colonoscopy, Dordaviprone Hydrochloride, Endoscopic Procedure, Questionnaire Administration, Sigmoidoscopy
Procedure · Drug · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
5
States / cities
Ann Arbor, Michigan • St Louis, Missouri • Cleveland, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Phase 2Phase 3 Interventional

Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases

NCT01019876
View directory record Official record
Conditions
Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
Interventions
Fludarabine, Cyclophosphamide, Busulfan, Alemtuzumab, Rabbit Anti-thymocyte Globulin, Horse Anti-thymocyte Globulin
Drug
Lead sponsor
Columbia University
Other
Eligibility
Up to 30 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 31, 2024 · Synced Oct 22, 2026, 4:03 AM EDT
Terminated Phase 2 Interventional Results available

IL-11 in Adults With Von Willebrand Disease Undergoing Surgery

NCT00524225
View directory record Official record
Conditions
Von Willebrand Disease
Interventions
Neumega (Oprelvekin, Interleukin 11, IL-11)
Drug
Lead sponsor
Margaret Ragni
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 11, 2019 · Synced Oct 22, 2026, 4:03 AM EDT
Terminated Phase 1 Interventional Results available

A Research Trial of Aralast in New Onset Diabetes (RETAIN)

NCT01183468
View directory record Official record
Conditions
Diabetes Mellitus, Type 1
Interventions
Aralast NP 45 mg dose, Aralast NP 90 mg dose, Aralast NP 180 mg dose
Biological
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
8 Years to 35 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
15
States / cities
San Diego, California • Aurora, Colorado • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2018 · Synced Oct 22, 2026, 4:03 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Study of Metabolic Effects of Pregnancy in Women With Cystic Fibrosis

NCT00014768
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
glucose, insulin, leucine
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older · Female only
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2001
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 22, 2026, 4:03 AM EDT
Terminated No phase listed Observational

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT01675674
View directory record Official record
Conditions
Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
Interventions
Dried blood spot test for MPS
Other
Lead sponsor
National MPS Society
Other
Eligibility
6 Months to 18 Years
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
New Brunswick, New Jersey • New York, New York
Source: ClinicalTrials.gov public record
Updated May 24, 2013 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Phase 2 Interventional Results available

Ziv-Aflibercept in Treating and Computed Tomography Perfusion Imaging in Predicting Response in Patients With Pancreatic Neuroendocrine Tumors That Are Metastatic or Cannot Be Removed by Surgery

NCT02101918
View directory record Official record
Conditions
Multiple Endocrine Neoplasia Type 1, Pancreatic Neuroendocrine Carcinoma
Interventions
Computed Tomography Perfusion Imaging, Laboratory Biomarker Analysis, Ziv-Aflibercept
Radiation · Other · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
3
States / cities
Tampa, Florida • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 23, 2020 · Synced Oct 22, 2026, 4:03 AM EDT
Withdrawn No phase listed Observational

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 8, 2015 · Synced Oct 22, 2026, 4:03 AM EDT
Completed No phase listed Observational

Gene Therapy for Patients With Leukocyte Adherence Deficiency (Follow-Up of Phase 1 Trial)

NCT00023010
View directory record Official record
Conditions
Leukocyte Adhesion Deficiency Syndrome
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
Not listed
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Phase 2 Interventional Results available

Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)

NCT00745420
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Hematopoietic Stem Cell Transplantation
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
3 Years to 19 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2016
U.S. locations
19
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Miami, Florida + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2022 · Synced Oct 22, 2026, 4:03 AM EDT
Terminated Phase 4 Interventional

Safety Study of Galantamine in Tic Disorders

NCT00226824
View directory record Official record
Conditions
Tourette's Syndrome, Motor Tic Disorder, Vocal Tic Disorder
Interventions
galantamine
Drug
Lead sponsor
Parkinson's Disease and Movement Disorders Center
Other
Eligibility
18 Years to 50 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Albany, New York
Source: ClinicalTrials.gov public record
Updated Aug 25, 2009 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Phase 2 Interventional Results available

Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)

NCT02153723
View directory record Official record
Conditions
Rett Syndrome
Interventions
Glatiramer Acetate
Drug
Lead sponsor
Montefiore Medical Center
Other
Eligibility
10 Years and older · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Nov 5, 2018 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Phase 3 Interventional Results available

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood

NCT05147662
View directory record Official record
Conditions
Treatment of Bleeding, Prophylaxis of Bleeding, Hemophilia A, Children
Interventions
Damoctocog alfa pegol (Jivi, BAY94-9027)
Biological
Lead sponsor
Bayer
Industry
Eligibility
7 Years to 11 Years · Male only
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Jul 2, 2026 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Phase 2 Interventional Results available

Trial of Sertraline to Treat Children With Fragile X Syndrome

NCT01474746
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Sertraline, Placebo
Drug
Lead sponsor
Randi J. Hagerman, MD
Other
Eligibility
24 Months to 68 Months
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Sep 25, 2017 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Phase 2 Interventional

High-Dose Intravenous Immunoglobulin to Treat Cerebellar Degeneration

NCT00034242
View directory record Official record
Conditions
Spinocerebellar Degenerations
Interventions
high-dose intravenous immunoglobulin (IVIG)
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 22, 2026, 4:03 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety, Tolerability and PK of ATTO-1310 in Healthy Volunteers and Patients With Atopic Dermatitis and Patients With Chronic Pruritus

NCT06787586
View directory record Official record
Conditions
Normal Volunteers, Atopic Dermatitis (AD), Atopic Eczema, Chronic Pruritus
Interventions
ATTO-1310, ATTO-1310 Placebo
Drug
Lead sponsor
Attovia Therapeutics Inc
Industry
Eligibility
18 Years to 85 Years
Enrollment
108 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
11
States / cities
Encinitas, California • Rocklin, California • Sacramento, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 22, 2026, 4:03 AM EDT
Recruiting No phase listed Observational

Duchenne Electronic Health Record Study

NCT07609394
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy, Dystrophinopathy, Dystrophinopathy Symptomatic Female Carrier
Interventions
Observational study with patients who may be treated with various disease-modifying therapies
Other
Lead sponsor
The Duchenne Registry
Other
Eligibility
Not listed
Enrollment
2,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2072
U.S. locations
10
States / cities
Little Rock, Arkansas • Sacramento, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 22, 2026, 4:03 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403
View directory record Official record
Conditions
MPS-IH (Hurler Syndrome)
Interventions
Experimental: OTL-203, Active Comparator: Allo-HSCT
Genetic
Lead sponsor
Orchard Therapeutics
Industry
Eligibility
28 Days to 30 Months
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 22, 2025 · Synced Oct 22, 2026, 4:03 AM EDT
Completed No phase listed Observational

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

NCT01971957
View directory record Official record
Conditions
Sjogren-Larsson Syndrome (SLS)
Interventions
Not listed
Lead sponsor
University of Nebraska
Other
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2023
U.S. locations
2
States / cities
Omaha, Nebraska • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Oct 22, 2026, 4:03 AM EDT