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ClinicalTrials.gov public records Last synced May 22, 2026, 1:09 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Gaucher Disease Type I Clear all
Enrolling by invitation No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced May 22, 2026, 1:09 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions
There is no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 64 Years
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2050
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 7, 2026 · Synced May 22, 2026, 1:09 AM EDT
No Longer Available No phase listed Expanded access
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Sep 6, 2018 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
Interventions
afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 74 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
7
States / cities
Beverly Hills, California • Coral Springs, Florida • Decatur, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2018 · Synced May 22, 2026, 1:09 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Apr 3, 2017 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 3, 2018 · Synced May 22, 2026, 1:09 AM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Neuroblastoma, Osteosarcoma
Interventions
iC9.GD2.CAR.IL-15 T-cells, Cyclophosphamide, Fludarabine
Biological · Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Months and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2044
U.S. locations
2
States / cities
Atlanta, Georgia • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 26, 2026 · Synced May 22, 2026, 1:09 AM EDT
Not listed Phase 2 Interventional
View directory record Official record
Conditions
Gaucher's Disease
Interventions
glucocerebrosidase
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 22, 2026, 1:09 AM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Gaucher Disease
Interventions
AVR-RD-02
Biological
Lead sponsor
John Bernat
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2037
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Jul 21, 2025 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Gaucher Disease
Interventions
Taliglucerase alfa
Drug
Lead sponsor
Pfizer
Industry
Eligibility
2 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 3, 2018 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 79 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
3
States / cities
Miami, Florida • Saint Paul, Minnesota • Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 7, 2017 · Synced May 22, 2026, 1:09 AM EDT
Suspended Phase 1Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 14, 2013 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Parkinson's Disease, Gaucher's Disease
Interventions
N-acetylcysteine
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 31, 2019 · Synced May 22, 2026, 1:09 AM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Gaucher Disease, Gaucher Disease, Type 1
Interventions
LY3884961
Genetic
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Durham, North Carolina • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Los Angeles, California • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Gaucher's Disease
Interventions
CEREDASE™
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 21, 2016 · Synced May 22, 2026, 1:09 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 31, 2026 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Gaucher Disease
Interventions
Digital Engagement Application (GD App), No Intervention
Other
Lead sponsor
Takeda
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Dec 20, 2023 · Synced May 22, 2026, 1:09 AM EDT
Completed No phase listed Observational
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 18, 2010 · Synced May 22, 2026, 1:09 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Parkinson Disease, GBA Gene Mutation, Gaucher Disease
Interventions
PET scan, neuroQWERTY
Diagnostic Test
Lead sponsor
Pacific Parkinson's Research Centre
Other
Eligibility
18 Years to 80 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
2
States / cities
Portland, Oregon • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 30, 2025 · Synced May 22, 2026, 1:09 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 22, 2021 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Gaucher's Disease, Osteopenia
Interventions
alendronate sodium, calcium carbonate, cholecalciferol
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 50 Years
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
2
States / cities
Cincinnati, Ohio • Dayton, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 1 Interventional Results available
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 12, 2014 · Synced May 22, 2026, 1:09 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
velaglucerase alfa, imiglucerase
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 7, 2021 · Synced May 22, 2026, 1:09 AM EDT