- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
- Interventions
- Thiotepa
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Up to 17 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 1
- States / cities
- San Francisco, California
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–24
of 1,597
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Generalized Lipodystrophy
- Interventions
- Mibavademab, Placebo
- Drug
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 2
- States / cities
- Ann Arbor, Michigan • Dallas, Texas
- Conditions
- Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
- Interventions
- AR882 Dose 1, AR882 Dose 2, Allopurinol Tablet
- Drug
- Lead sponsor
- Arthrosi Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 42 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 10
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Sun City, Arizona + 7 more
- Conditions
- Gas, Bloating, Distension, Constipation, Diarrhea
- Interventions
- APP Diary, Paper Diary
- Other
- Lead sponsor
- Augusta University
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2024
- U.S. locations
- 1
- States / cities
- Augusta, Georgia
- Conditions
- Fed
- Interventions
- CIPRO®XR
- Drug
- Lead sponsor
- Dr. Reddy's Laboratories Limited
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Tay Sachs Disease, Sandhoff Disease, Late Onset Tay Sachs Disease
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 2
- States / cities
- Tampa, Florida • Minneapolis, Minnesota
- Conditions
- Gout
- Interventions
- Lesinurad, Placebo, Allopurinol
- Drug
- Lead sponsor
- Ardea Biosciences, Inc.
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 610 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 88
- States / cities
- Mobile, Alabama • Chandler, Arizona • Mesa, Arizona + 76 more
- Conditions
- Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
- Interventions
- Acoramidis, Placebo oral tablet
- Drug
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 587 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 40
- States / cities
- La Jolla, California • Los Angeles, California • San Francisco, California + 31 more
- Conditions
- Hunter Syndrome
- Interventions
- Idursulfase-IT, Elaprase
- Drug
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 3 Years to 18 Years · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2024
- U.S. locations
- 7
- States / cities
- Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
- Interventions
- Whole-body Electrical Muscle Stimulation Exercise
- Device
- Lead sponsor
- University of Missouri-Columbia
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 1
- States / cities
- Columbia, Missouri
- 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
- Acute Gouty Arthritis, Gout Flare, Gout Flares, Acute Gout Flare
- Interventions
- ABP-745 Dose A, ABP-745 Dose B, Colchicine, Placebo
- Drug
- Lead sponsor
- Atom Therapeutics Co., Ltd
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 380 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 14
- States / cities
- Peoria, Arizona • La Mesa, California • Norcross, Georgia + 11 more
- Conditions
- Gout, Hyperuricemia
- Interventions
- SAP-001
- Drug
- Lead sponsor
- Shanton Pharma Pte. Ltd.
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2025
- U.S. locations
- 2
- States / cities
- Tampa, Florida • Saint Paul, Minnesota
- Conditions
- Gout
- Interventions
- Allopurinol, Dotinurad
- Drug
- Lead sponsor
- Crystalys Therapeutics
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 95
- States / cities
- Birmingham, Alabama • Foley, Alabama • Gilbert, Arizona + 86 more
- Conditions
- Mucopolysaccharidosis (MPS), Hunter Syndrome
- Interventions
- Not listed
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years to 18 Years · Male only
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 3
- States / cities
- Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina
- 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
- Wilson Disease
- Interventions
- zinc acetate
- Drug
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years and older
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1993
- U.S. locations
- 1
- States / cities
- Ann Arbor, Michigan
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis
- Interventions
- Mycophenolate mofetil, Liquid Placebo
- Drug
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 6 Years to 25 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Phenylketonuria
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2017
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- VTS-270
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- Up to 6 Months
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- MPS I
- Interventions
- SB-318
- Biological
- Lead sponsor
- Sangamo Therapeutics
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2021
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Hunter Syndrome
- Interventions
- Not listed
- Lead sponsor
- Shire
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,443 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2023
- U.S. locations
- 1
- States / cities
- Lexington, Massachusetts
- Conditions
- Enteric Hyperoxaluria, Primary Hyperoxaluria, Hyperoxalemia
- Interventions
- ALLN-177
- Drug
- Lead sponsor
- Allena Pharmaceuticals
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 4
- States / cities
- Scottsdale, Arizona • Little Rock, Arkansas • New Haven, Connecticut + 1 more
- Conditions
- Acute Gout
- Interventions
- apremilast, indomethacin SR
- Drug
- Lead sponsor
- Dartmouth-Hitchcock Medical Center
- Other
- Eligibility
- 18 Years and older · Male only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010
- U.S. locations
- 1
- States / cities
- Lebanon, New Hampshire