- Conditions
- Fabry Disease
- Interventions
- Fabrazyme (agalsidase beta)
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 58 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2004
- U.S. locations
- 14
- States / cities
- Los Angeles, California • San Francisco, California • Coral Springs, Florida + 11 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 505–528
of 1,510
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease, Sandhoffs Disease, Wolman Disease, I-Cell Disease, Sanfilippo Syndrome, GM1 Gangliosidosis
- Interventions
- Clofarabine, Total body Irradiation, Melphalan, Hematopoietic Stem Cell Transplantation, Alemtuzumab, mycophenylate mofetil, Cyclosporine A, Hydroxyurea
- Drug · Procedure · Biological + 1 more
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Familial Hypercholesterolemia - Homozygous
- Interventions
- Inclisiran, Placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 12 Years to 17 Years
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Boca Raton, Florida
- 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
- Cystinosis
- Interventions
- RP103, Cystagon®
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 5
- States / cities
- San Francisco, California • Stanford, California • Atlanta, Georgia + 2 more
- Conditions
- Pseudohypoparathyroidism Type 1a, Obesity
- Interventions
- Setmelanotide
- Drug
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Hypophosphatasia
- Interventions
- Asfotase alfa
- Drug
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 3
- States / cities
- St Louis, Missouri • Durham, North Carolina • Nashville, Tennessee
- 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
- MELAS Syndrome
- Interventions
- Idebenone, Placebo
- Drug · Other
- Lead sponsor
- Michio Hirano
- Other
- Eligibility
- 8 Years to 65 Years
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Fabry Disease
- Interventions
- Replagal
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 44 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 4
- States / cities
- Tucson, Arizona • Iowa City, Iowa • New York, New York + 1 more
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)
- Interventions
- Lenti-D Drug Product (eli-cel)
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2021
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
- Conditions
- Alpha-Mannosidosis
- Interventions
- Velmanase Alfa
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Up to 3 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 2
- States / cities
- Miami, Florida • Greenwood, South Carolina
- Conditions
- Hyperuricemia, Gout, Chronic Kidney Diseases
- Interventions
- ALLN-346, Placebo
- Drug
- Lead sponsor
- Allena Pharmaceuticals
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 17
- States / cities
- Birmingham, Alabama • Muscle Shoals, Alabama • Sheffield, Alabama + 12 more
- Conditions
- Cystinosis
- Interventions
- Cystagon®, RP103
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- Not listed
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Cytomegalovirus Infections, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 13 Years to 60 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 2
- States / cities
- San Francisco, California • Washington D.C., District of Columbia
- Conditions
- Congenital Adrenal Hyperplasia, Hyperplasia, Adrenal Hyperplasia, Congenital Disorders, Adrenocortical Hyperfunction, Disorders of Sex Development, Urogenital Abnormalities, Genetic Diseases, Inborn, Steroid Metabolic Diseases, Inborn, Adrenal Gland Disease, Hydrocortisone
- Interventions
- Subcutaneous hydrocortisone
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 4 Years to 18 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Interventions
- SBT101 Treatment
- Genetic
- Lead sponsor
- SwanBio Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 1
- States / cities
- Worcester, Massachusetts
- Conditions
- Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease
- Interventions
- BMN 190
- Biological
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 3 Years to 15 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- COPD, Tetrahydrobiopterin Deficiency, Oxidative Stress
- Interventions
- Tetrahydrobiopterin, Placebo oral tablet
- Drug
- Lead sponsor
- University of Massachusetts, Amherst
- Other
- Eligibility
- 18 Years to 85 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Amherst, Massachusetts
- Conditions
- Cardiovascular Disease With Mention of Arteriosclerosis, Elevated Cholesterol, Familial Hypercholesterolemia
- Interventions
- lerodalcibep
- Drug
- Lead sponsor
- LIB Therapeutics LLC
- Industry
- Eligibility
- 10 Years and older
- Enrollment
- 2,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 4
- States / cities
- Evanston, Illinois • Cincinnati, Ohio
- Conditions
- Digestive Health, Abdominal Discomfort, Gas and Bloating
- Interventions
- Bloat Gummies, Placebo
- Dietary Supplement
- Lead sponsor
- Arrae
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Schizophrenia, Psychotic Disorders
- Interventions
- Glycine administration
- Dietary Supplement
- Lead sponsor
- Mclean Hospital
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 21 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2013
- U.S. locations
- 1
- States / cities
- Belmont, Massachusetts
- Conditions
- Hypophosphatasia, Bone Disease
- Interventions
- Retrospective Case Only
- Biological
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Familial Hypercholesterolemia - Homozygous
- Interventions
- Inclisiran, Placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 4
- States / cities
- San Francisco, California • Washington D.C., District of Columbia • St Louis, Missouri + 1 more