- 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
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 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Fabry Disease
- Interventions
- Fabrazyme (agalsidase beta)
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 7 Years to 15 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2005
- U.S. locations
- 1
- States / cities
- Tucson, Arizona
- Conditions
- Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
- Interventions
- rivogenlecleucel, rimiducid
- Biological · Drug
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 3 Months to 21 Years
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Palo Alto, California
- Conditions
- Mucopolysaccharidosis VI
- Interventions
- N-acetylgalactosamine 4-sulfatase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2006
- U.S. locations
- 1
- States / cities
- Novato, California
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years · Female only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
- Conditions
- Aspartylglucosaminuria, Fucosidosis, Galactosialidosis, Alpha Mannosidosis, Beta Mannosidosis, Mucolipidosis II, Mucolipidosis III, Schindler Disease, Sialidosis
- Interventions
- Non-interventional.
- Other
- Lead sponsor
- Greenwood Genetic Center
- Other
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2020
- U.S. locations
- 1
- States / cities
- North Charleston, South Carolina
- Conditions
- Hunter Syndrome
- Interventions
- Elaprase for intravenous (IV) infusion
- Drug
- Lead sponsor
- Shire
- Industry
- Eligibility
- Up to 5 Years · Male only
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
- Interventions
- PBGM01
- Biological
- Lead sponsor
- Gemma Biotherapeutics
- Industry
- Eligibility
- 1 Month to 24 Months
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2029
- U.S. locations
- 4
- States / cities
- Oakland, California • Minneapolis, Minnesota • New Brunswick, New Jersey + 1 more
- Conditions
- MPS II, Mucopolysaccharidosis II, Hunter Syndrome
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- Not listed
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Mucopolysaccharidosis II
- Interventions
- JR-141
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 2
- States / cities
- Oakland, California • Philadelphia, Pennsylvania
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride, Placebo
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 16 Years to 74 Years
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2014
- U.S. locations
- 13
- States / cities
- Los Angeles, California • San Francisco, California • Decatur, Georgia + 10 more
- Conditions
- Lysosomal Acid Lipase Deficiency
- Interventions
- There is no intervention in the study
- Other
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 2 Years to 16 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 2
- States / cities
- Atlanta, Georgia • Houston, Texas
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Not listed
- Lead sponsor
- REGENXBIO Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Gaucher Disease, Type 2
- Interventions
- LY3884961, Methylprednisolone, Sirolimus, Prednisone
- Genetic · Drug
- Lead sponsor
- Prevail Therapeutics
- Industry
- Eligibility
- 0 Months to 24 Months
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2028
- U.S. locations
- 4
- States / cities
- Oakland, California • Minneapolis, Minnesota • Pittsburgh, Pennsylvania + 1 more
- Conditions
- Parkinson Disease, Gaucher Disease, Healthy, GBA Gene Mutation
- Interventions
- Biological Sample Collection
- Other
- Lead sponsor
- New York Stem Cell Foundation Research Institute
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2025
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Mucopolysaccharidosis I, Lysosomal Storage Diseases, Spinal Cord Compression
- Interventions
- laronidase
- Drug
- Lead sponsor
- Patricia I. Dickson, M.D.
- Individual
- Eligibility
- 8 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2011
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Lysosomal Storage Diseases, Inborn Errors of Metabolism
- Interventions
- ALD-601
- Biological
- Lead sponsor
- Joanne Kurtzberg, MD
- Other
- Eligibility
- 18 Years and older · Female only
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2017
- U.S. locations
- 2
- States / cities
- Durham, North Carolina
- Conditions
- Fabry's Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 325 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 1995 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- N-Acetyl-L-Leucine, Placebo
- Drug · Other
- Lead sponsor
- IntraBio Inc
- Industry
- Eligibility
- 4 Years and older
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2030
- U.S. locations
- 1
- States / cities
- Rochester, Minnesota
- Conditions
- GM2 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
- Interventions
- AXO-AAV-GM2
- Drug
- Lead sponsor
- Terence Flotte
- Other
- Eligibility
- Not listed
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- Worcester, Massachusetts
- Conditions
- Fabry Disease
- Interventions
- GZ/SAR402671
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years to 49 Years · Male only
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 3
- States / cities
- Atlanta, Georgia • Cincinnati, Ohio • Fairfax, Virginia
- Conditions
- Morquio Disease, MPS - Mucopolysaccharidosis
- Interventions
- Interview
- Other
- Lead sponsor
- Emory University
- Other
- Eligibility
- 15 Years to 100 Years · Female only
- Enrollment
- 18 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2018
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Sanfilippo Syndrome, MPS3
- Interventions
- Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day, Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day
- Drug
- Lead sponsor
- Ozlem Goker-Alpan
- Other
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia