- 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 are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 1–24
of 268
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Adrenoleukodystrophy
- Interventions
- glyceryl trierucate/glyceryl trioleate
- Drug
- Lead sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
- Other
- Eligibility
- 18 Months to 8 Years · Male only
- Enrollment
- 126 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1998 – 2014
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2,724 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2017
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- 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
- Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
- Interventions
- afegostat tartrate
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 3
- States / cities
- Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 3 Months and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Metachromatic Leukodystrophy
- Interventions
- Enriched Hematopoetic Stem Cell Infusion
- Biological
- Lead sponsor
- Talaris Therapeutics Inc.
- Industry
- Eligibility
- 3 Years and older · Male only
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- 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
- 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
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- University of Washington
- Other
- Eligibility
- 1 Day to 90 Years
- Enrollment
- 58 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Minneapolis, Minnesota • Seattle, Washington
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- Type I Gaucher Disease
- Interventions
- Ambroxol
- Drug
- Lead sponsor
- Exsar Corporation
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Monmouth Junction, New Jersey
- Conditions
- Sphingolipidoses, Enzyme Deficiency
- Interventions
- no intervention
- Other
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- Not listed
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2028
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Fabry Disease
- Interventions
- Replagal (agalsidase alfa)
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 5
- States / cities
- Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
- Interventions
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 135 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1995 – 2010
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Sphingomyelin Lipidosis
- Interventions
- placebo (saline), Olipudase alfa
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 36 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2023
- U.S. locations
- 3
- States / cities
- San Francisco, California • Decatur, Georgia • The Bronx, New York
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- N-Acetyl Cysteine
- Drug
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 12 Months and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
- Interventions
- Sudoscan, Skin biopsy, QSART
- Device · Procedure
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 8 Years to 70 Years
- Enrollment
- 102 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2020
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Fabry Disease
- Interventions
- Interview
- Other
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Not listed
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
- Conditions
- Niemann-Pick Disease, Type C1
- Interventions
- Hydroxypropyl-beta-cyclodextrin, Placebo
- Drug
- Lead sponsor
- Cyclo Therapeutics, Inc.
- Industry
- Eligibility
- 3 Years and older
- Enrollment
- 94 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 7
- States / cities
- Oakland, California • Jacksonville, Florida • Atlanta, Georgia + 4 more
- Conditions
- Fabry Disease
- Interventions
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- Biological
- Lead sponsor
- Protalix
- Industry
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 78 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2022
- U.S. locations
- 15
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
- Conditions
- Inherited Metabolic Disorders (IMD)
- Interventions
- Safety and efficacy assessments
- Other
- Lead sponsor
- Magenta Therapeutics, Inc.
- Industry
- Eligibility
- Up to 16 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease
- Interventions
- Afegostat tartrate
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 74 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 10
- States / cities
- San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 7 more