- Conditions
- GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
- Interventions
- Zavesca (Miglustat)
- Drug
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 6 Months to 5 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2007
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
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 are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–24
of 317
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Fabry Disease
- Interventions
- Replagal
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2005
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Lysosomal Storage Disease, Peroxisomal Disorder
- Interventions
- Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil
- Drug · Radiation · Biological
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2013
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Sjögren-Larsson Syndrome
- Interventions
- Active topical NS2 1% dermatologic cream, Vehicle placebo 0.0% NS2 dermatologic cream
- Drug
- Lead sponsor
- Aldeyra Therapeutics, Inc.
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 3
- States / cities
- Omaha, Nebraska • Hershey, Pennsylvania • Fairfax, Virginia
- Conditions
- Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
- Interventions
- AAV2CUhCLN2 (3x10^12 particle units)
- Biological
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2019
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease
- Interventions
- enzyme analysis
- Other
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 60 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2012
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Fabry Disease, Healthy
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Male only
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2000 – 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease, Alpha Galactosidase A Deficiency
- Interventions
- agalsidase beta
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2024
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
- Conditions
- Fabry Disease
- Interventions
- AMT-191
- Drug
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Eligibility
- 18 Years to 50 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 8
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 5 more
- Conditions
- Fabry Disease
- Interventions
- agalsidase alfa
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- Not listed
- U.S. locations
- 25
- States / cities
- Tucson, Arizona • La Jolla, California • Los Angeles, California + 21 more
- Conditions
- Niemann-Pick Diseases
- Interventions
- Not listed
- Lead sponsor
- National Niemann-Pick Disease Foundation
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 76 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Gaucher Disease
- Interventions
- Standard of Care
- Other
- Lead sponsor
- Takeda
- Industry
- Eligibility
- Up to 5 Years
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Gaucher Disease, Type 1
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 16 Years to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1, Neuronal Ceroid Lipofuscinosis CLN2, Neuronal Ceroid Lipofuscinosis CLN3, Neuronal Ceroid Lipofuscinosis CLN5, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN7, Neuronal Ceroid Lipofuscinosis CLN8, Neuronal Ceroid Lipofuscinosis CLN10, Batten Disease
- Interventions
- Not listed
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- Not listed
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2035
- U.S. locations
- 1
- States / cities
- Rochester, New York
- 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
- 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
- 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
- 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
- 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
- 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