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ClinicalTrials.gov public records Last synced Oct 30, 2026, 7:07 AM EDT

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Showing 1–24 of 95 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Gaucher Disease Clear all
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

NCT00001234
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 30, 2026, 7:07 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599
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
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
2
States / cities
Durham, North Carolina • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 30, 2026, 7:07 AM EDT
Not listed No phase listed Observational

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

NCT00005900
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 2 Interventional

OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

NCT00041535
View directory record Official record
Conditions
Gaucher Disease
Interventions
OGT 918
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
4 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 30, 2026, 7:07 AM EDT
Completed No phase listed Observational

Effects of Enzyme Replacement in Gaucher's Disease

NCT00001289
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1991 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 2 Interventional

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

NCT00004488
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 25, 2015 · Synced Oct 30, 2026, 7:07 AM EDT
Completed No phase listed Observational

A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease

NCT00465062
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 30, 2026, 7:07 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Biomarkers Related to Bone in Pediatric Gaucher Disease

NCT06116071
View directory record Official record
Conditions
Gaucher Disease, Bone Diseases
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
5 Years to 21 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 13, 2024 · Synced Oct 30, 2026, 7:07 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Parkinson's Disease in Type 1 Gaucher Disease

NCT07886879
View directory record Official record
Conditions
Parkinson Disease (PD), Type 1 Gaucher Disease
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
40 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 27, 2026 · Synced Oct 30, 2026, 7:07 AM EDT
Not listed No phase listed Observational

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT06539169
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 3 Interventional Results available

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

NCT00553631
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 8, 2021 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

NCT00478647
View directory record Official record
Conditions
Gaucher Disease
Interventions
GA-GCB (velaglucerase alfa)
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
11
States / cities
Los Angeles, California • Oakland, California • Decatur, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 30, 2026, 7:07 AM EDT
Completed No phase listed Observational

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

NCT01136304
View directory record Official record
Conditions
Gaucher Disease
Interventions
Imiglucerase
Drug
Lead sponsor
University Research Foundation for Lysosomal Storage Diseases, Inc.
Other
Eligibility
18 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Beverly Hills, California • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 30, 2026, 7:07 AM EDT
Recruiting No phase listed Observational

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

NCT05586243
View directory record Official record
Conditions
Gaucher Disease, Type 3
Interventions
No intervention
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Timeline
2023 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 4 Interventional Results available

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

NCT00364858
View directory record Official record
Conditions
Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form
Interventions
Cerezyme
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2007
U.S. locations
18
States / cities
Washington D.C., District of Columbia • Coral Springs, Florida • Atlanta, Georgia + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2015 · Synced Oct 30, 2026, 7:07 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 30, 2026, 7:07 AM EDT
Not listed No phase listed Observational

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

NCT02605603
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 13, 2019 · Synced Oct 30, 2026, 7:07 AM EDT
Recruiting No phase listed Observational

Gaucher Disease Outcome Survey (GOS)

NCT03291223
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
1,257 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2028
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 1 Interventional

PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

NCT00001410
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Lysodase
Drug
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1993 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 4 Interventional Results available

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

NCT02574286
View directory record Official record
Conditions
Gaucher Disease
Interventions
Velaglucerase alfa, Vitamin D
Drug · Dietary Supplement
Lead sponsor
Shire
Industry
Eligibility
18 Years to 70 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
8
States / cities
Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2022 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Taste Evaluation of Different Liquid Formulations With Eliglustat

NCT02422654
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 55 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated May 27, 2015 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease

NCT01427517
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 Nov 1, 2019 · Synced Oct 30, 2026, 7:07 AM EDT
Completed Phase 3 Interventional Results available

A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease

NCT00376168
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
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 30, 2026, 7:07 AM EDT