- Conditions
- Gaucher Disease
- Interventions
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- U.S. locations
- 14
- States / cities
- La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
Search Results
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Showing 1–24
of 94
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- 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
- I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy, Adrenoleukodystrophy, Mannosidosis, Niemann-Pick Disease, Pulmonary Complications, Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Metachromatic Leukodystrophy, Gaucher's Disease, Wolman Disease
- Interventions
- Not listed
- Lead sponsor
- Fairview University Medical Center
- Other
- Eligibility
- 0 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Gaucher
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 5 Years to 20 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Gaucher Disease Type 1
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 16 Years to 90 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
- Interventions
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Biological · Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Type 1 Gaucher Disease
- Interventions
- AT2101
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009
- U.S. locations
- 1
- States / cities
- Miramar, Florida
- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Gaucher Disease Type 1
- Interventions
- N-acetylcysteine
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2025
- U.S. locations
- 2
- States / cities
- Minneapolis, Minnesota • New York, New York
- Conditions
- Gaucher Disease, Type 1
- Interventions
- VPRIV®
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 95 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Oakland, California • Atlanta, Georgia + 7 more
- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2016
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Gaucher Disease, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Interventions
- Not listed
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 12,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1991 – 2034
- U.S. locations
- 86
- States / cities
- Phoenix, Arizona • Tucson, Arizona • Little Rock, Arkansas + 65 more
- Conditions
- Gaucher Disease, Type 1
- Interventions
- Eliglustat tartrate, Imiglucerase
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 160 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2015
- U.S. locations
- 17
- States / cities
- Beverly Hills, California • San Francisco, California • Aurora, Colorado + 13 more
- Conditions
- Type 1 Gaucher Disease
- Interventions
- Safety and efficacy assessments
- Other
- Lead sponsor
- AVROBIO
- Industry
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 2
- States / cities
- Iowa City, Iowa • Hackensack, New Jersey
- Conditions
- Gaucher Disease Type 1
- Interventions
- FLT201
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 16
- States / cities
- Los Angeles, California • San Francisco, California • Aurora, Colorado + 11 more
- 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 – 2027
- U.S. locations
- 5
- States / cities
- Oakland, California • Minneapolis, Minnesota • New York, New York + 2 more
- Conditions
- Gaucher Disease
- Interventions
- no intervention
- Other
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 30 participants
- Timeline
- 2023 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Lysosomal Storage Disorders
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 80 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2022
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Gaucher Disease
- Interventions
- Blood sample
- Procedure
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2007
- U.S. locations
- 7
- States / cities
- San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 4 more
- Conditions
- Gaucher Disease
- Interventions
- Taliglucerase alfa
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 2
- States / cities
- Decatur, Georgia • New York, New York
- Conditions
- Gaucher Disease
- Interventions
- AVR-RD-02
- Drug
- Lead sponsor
- AVROBIO
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 4
- States / cities
- San Diego, California • Iowa City, Iowa • Hackensack, New Jersey + 1 more
- Conditions
- Lysosomal Storage Diseases
- Interventions
- Enzyme assay and molecular sequencing
- Diagnostic Test
- Lead sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
- Other
- Eligibility
- Not listed
- Enrollment
- 100,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Gaucher Disease, Type 1
- Interventions
- FLT201
- Genetic
- Lead sponsor
- Spur Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Fairfax, Virginia