- Conditions
- Choroideremia, CHM (Choroideremia) Gene Mutations
- Interventions
- AAV2-hCHM
- Biological
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2022
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Philadelphia, Pennsylvania
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1–24
of 838
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Brain and Central Nervous System Tumors, Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific
- Interventions
- filgrastim, sargramostim, therapeutic autologous lymphocytes, O6-benzylguanine, carmustine, temozolomide, in vitro-treated peripheral blood stem cell transplantation
- Biological · Drug · Procedure
- Lead sponsor
- Case Comprehensive Cancer Center
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2007
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- Gene-Transduced Autologous CD34+ Stem Cells
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Months to 20 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2011
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Sickle Cell Disease, Pulmonary Disease, Renal Disease, Heart Disease
- Interventions
- Not listed
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 4 Years to 65 Years
- Enrollment
- 377 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 5
- States / cities
- Washington D.C., District of Columbia • Atlanta, Georgia • Baltimore, Maryland + 2 more
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Risdiplam
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 3 Months to 24 Months
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2029
- U.S. locations
- 10
- States / cities
- Little Rock, Arkansas • Madera, California • Palo Alto, California + 7 more
- Conditions
- Esophageal Neoplasms
- Interventions
- CD40 Gene Transfer
- Genetic
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 18 Years to 75 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2010
- U.S. locations
- 2
- States / cities
- Ridgewood, New Jersey • New York, New York
- Conditions
- Aplastic Anemias, Hemoglobinopathies, Bone Marrow Failure
- Interventions
- Not listed
- Lead sponsor
- Medical College of Wisconsin
- Other
- Eligibility
- Not listed
- Enrollment
- 375 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026 – 2031
- U.S. locations
- 14
- States / cities
- Duarte, California • Gainesville, Florida • Atlanta, Georgia + 10 more
- Conditions
- Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
- Interventions
- No intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 3
- States / cities
- Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- rAAVrh74.MCK.GALGT2, PLACEBO (Saline)
- Biological · Other
- Lead sponsor
- Kevin Flanigan
- Other
- Eligibility
- 9 Years and older · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Fabry Disease, Lysosomal Storage Diseases
- Interventions
- FLT190
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 4
- States / cities
- Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
- Conditions
- Hemophilia B
- Interventions
- HEMGENIX, Factor IX (FIX)
- Genetic · Biological
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2043
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Prostate Cancer
- Interventions
- PROSTVAC-F (Fowlpox)/TRICOM, PROSTVAC-V (Vaccinia)/TRICOM, Enzalutamide (Xtandi)
- Biological · Drug
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 100 Years · Male only
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2020
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Parkinson's Disease, Multiple System Atrophy, Parkinson Variant, Multiple System Atrophy
- Interventions
- This is an observational study.
- Other
- Lead sponsor
- AskBio Inc
- Industry
- Eligibility
- 35 Years to 75 Years
- Enrollment
- 73 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 7
- States / cities
- Irvine, California • Boca Raton, Florida • Boston, Massachusetts + 4 more
- Conditions
- Phenylketonurias, PAH Deficiency, Phenylketonuria
- Interventions
- HMI-103
- Drug
- Lead sponsor
- Homology Medicines, Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 2
- States / cities
- Topeka, Indiana • Lancaster, Pennsylvania
- Conditions
- GSD1
- Interventions
- DTX401, steroid regimen
- Genetic · Drug
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2021
- U.S. locations
- 3
- States / cities
- Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
- Conditions
- Heart Failure With Reduced Ejection Fraction
- Interventions
- YAP101 (AAV9-Sav-shRNA)
- Combination Product
- Lead sponsor
- Medley Therapeutics
- Industry
- Eligibility
- 18 Years to 79 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Granulomatous Disease, Chronic, X-linked
- Interventions
- Lentiviral G1XCGD Gene Therapy
- Biological
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 23 Months and older · Male only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2024
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Bethesda, Maryland • Boston, Massachusetts
- Conditions
- Coronary Artery Disease, Ischemia, Angina Refractory, Cardiovascular Diseases, Heart Diseases
- Interventions
- AdVEGFXC1
- Biological
- Lead sponsor
- XyloCor Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2023
- U.S. locations
- 16
- States / cities
- Stanford, California • Washington D.C., District of Columbia • Daytona Beach, Florida + 11 more
- Conditions
- X-Linked Chronic Granulomatous Disease
- Interventions
- EN-374
- Genetic
- Lead sponsor
- Ensoma
- Industry
- Eligibility
- 3 Months and older · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 8
- States / cities
- Los Angeles, California • San Francisco, California • St. Petersburg, Florida + 5 more
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Long-term Follow-Up
- Other
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 3
- States / cities
- San Francisco, California • New Brunswich, New Jersey • Philadelphia, Pennsylvania
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- PF-06939926, Placebo
- Genetic · Other
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 4 Years to 7 Years · Male only
- Enrollment
- 114 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2039
- U.S. locations
- 17
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 8 more
- Conditions
- Pompe Disease
- Interventions
- rAAV1-CMV-GAA (study agent) Administration, RMST
- Drug · Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2015
- U.S. locations
- 1
- States / cities
- Gainesville, Florida
- Conditions
- Advanced Solid Tumor
- Interventions
- RP3, Nivolumab
- Biological
- Lead sponsor
- Replimune, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 123 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2026
- U.S. locations
- 3
- States / cities
- Iowa City, Iowa • Pittsburgh, Pennsylvania • Houston, Texas
- Conditions
- Parkinson's Disease
- Interventions
- Surgical infusion of AAV-GAD into the subthalamic nucleus
- Genetic
- Lead sponsor
- Neurologix, Inc.
- Industry
- Eligibility
- 25 Years to 75 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2005
- U.S. locations
- 1
- States / cities
- Manhasset, New York