- Conditions
- Leukodystrophy, Globoid Cell
- Interventions
- PBKR03
- Biological
- Lead sponsor
- Gemma Biotherapeutics
- Industry
- Eligibility
- 1 Month to 9 Months
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2030
- U.S. locations
- 4
- States / cities
- Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
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 73–96
of 1,603
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Lysosomal Storage Diseases
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Homozygous Familial Hypercholesterolemia, HoFH
- Interventions
- HDL Therapeutics PDS-2 System
- Device
- Lead sponsor
- HDL Therapeutics
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD II)
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2020
- U.S. locations
- 4
- States / cities
- Valhalla, New York • Cincinnati, Ohio • Salt Lake City, Utah + 1 more
- Conditions
- Propionic Acidemia
- Interventions
- ornithine alpha ketoglutarate, glutamine, disodium citrate
- Drug
- Lead sponsor
- Nicola Longo
- Other
- Eligibility
- 5 Years to 12 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- Conditions
- Cardiac Amyloidosis
- Interventions
- F-18 florbetapir
- Drug
- Lead sponsor
- The Cleveland Clinic
- Other
- Eligibility
- 18 Years to 95 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2019
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Gout, Gouty Arthritis, Hyperuricemia, Gout Chronic, Refractory Gout, Uncontrolled Gout, Tophaceous Gout
- Interventions
- AR882 75 mg, XOI Low Dose, XOI High Dose
- Drug
- Lead sponsor
- Arthrosi Therapeutics
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 8
- States / cities
- Margate, Florida • Miami, Florida • Tampa, Florida + 5 more
- Conditions
- Phenylketonuria
- Interventions
- sapropterin dihydrochloride, Moxifloxacin, Moxifloxacin placebo
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 56 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- Fargo, North Dakota
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
- Interventions
- Cholic Acids
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1992 – 2009
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- VTS-270
- Drug
- Lead sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
- Industry
- Eligibility
- 2 Years to 25 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Cytomegalovirus Infections, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Mucopolysaccharidosis I
- Interventions
- JR-171
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Hyperuricemia, Gout
- Interventions
- Vitamin C
- Dietary Supplement
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 135 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Adrenomyeloneuropathy, Adrenoleukodystrophy
- Interventions
- Lorenzo's oil
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 240 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2007
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Pompe Disease (Late-onset)
- Interventions
- No Intervention
- Other
- Lead sponsor
- Astellas Gene Therapies
- Industry
- Eligibility
- 16 Years to 69 Years
- Enrollment
- 119 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 10
- States / cities
- Atlanta, Georgia • Kansas City, Kansas • Ann Arbor, Michigan + 7 more
- Conditions
- Unresectable or Metastatic Malignant Gastrointestinal Stromal Tumor (GIST)
- Interventions
- Imatinib mesylate
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 148 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2013
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Portland, Oregon • Philadelphia, Pennsylvania
- Conditions
- Hunter Syndrome
- Interventions
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg)
- Other · Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- 3 Years to 18 Years · Male only
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- Conditions
- CLN7
- Interventions
- AAV9/CLN7
- Genetic
- Lead sponsor
- Benjamin Greenberg
- Other
- Eligibility
- 1 Year to 18 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Ornithine Transcarbamylase Deficiency
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Amyloidosis Cardiac
- Interventions
- Sonotherapy
- Combination Product
- Lead sponsor
- Brigham and Women's Hospital
- Other
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 70 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Inherited Mitochondrial Disease, Including Leigh Syndrome
- Interventions
- Cysteamine Bitartrate
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 6 Years to 17 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 5
- States / cities
- San Diego, California • Stanford, California • Akron, Ohio + 2 more
- Conditions
- X-linked Hypophosphatemia
- Interventions
- burosumab
- Biological
- Lead sponsor
- Kyowa Kirin, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 5
- States / cities
- San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
- Interventions
- Melphalan
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 17 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2021
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Phenylketonuria
- Interventions
- JNT-517 Tablet, Placebo Tablet: BID
- Drug
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Gainesville, Florida • Tampa, Florida + 7 more