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ClinicalTrials.gov public records Last synced Sep 30, 2026, 3:03 PM EDT

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Showing 1–24 of 92 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Glucocerebrosidase Deficiency Disease Clear all
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038
View directory record Official record
Conditions
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions
Confirmatory Testing
Diagnostic Test
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
Up to 4 Weeks
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2029
U.S. locations
9
States / cities
Brooklyn, New York • Manhasset, New York • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Sep 30, 2026, 3:03 PM 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 Sep 30, 2026, 3:03 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-Up for Safety of AVR-RD-02

NCT06488261
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 22, 2025 · Synced Sep 30, 2026, 3:03 PM EDT
Recruiting No phase listed Observational

A Long-term Follow-up Study of Gaucher Disease

NCT03190837
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
200 participants
Timeline
2017 – 2028
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Sep 30, 2026, 3:03 PM 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 Sep 30, 2026, 3:03 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

NCT06188325
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Sep 30, 2026, 3:03 PM 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 Sep 30, 2026, 3:03 PM 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 Sep 30, 2026, 3:03 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Investigating Lysosomal Storage Diseases in Minority Groups

NCT02120235
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day to 100 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 4, 2017 · Synced Sep 30, 2026, 3:03 PM EDT
Completed No phase listed Observational

Genzyme Osteopenia/Osteoporosis Study

NCT02785744
View directory record Official record
Conditions
Gaucher Disease
Interventions
Gaucher disease DNA mutation analysis
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 23, 2021 · Synced Sep 30, 2026, 3:03 PM 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 Sep 30, 2026, 3:03 PM EDT
Completed Phase 2 Interventional Results available

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

NCT00358150
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
Eliglustat tartrate
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 15, 2017 · Synced Sep 30, 2026, 3:03 PM EDT
Completed Phase 4 Interventional Results available

A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

NCT04718779
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 21, 2023 · Synced Sep 30, 2026, 3:03 PM 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 Sep 30, 2026, 3:03 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

NCT04145037
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jan 18, 2024 · Synced Sep 30, 2026, 3:03 PM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

NCT01074944
View directory record Official record
Conditions
Gaucher Disease
Interventions
Eliglustat tartrate
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
10
States / cities
San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2017 · Synced Sep 30, 2026, 3:03 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

NCT04127578
View directory record Official record
Conditions
Parkinson Disease
Interventions
LY3884961, Methylprednisolone, Sirolimus
Biological · Drug
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
35 Years to 80 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2031
U.S. locations
11
States / cities
Scottsdale, Arizona • Murrieta, California • Englewood, Colorado + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Sep 30, 2026, 3:03 PM EDT
Completed Phase 3 Interventional Results available

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

NCT00635427
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Sep 30, 2026, 3:03 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Biomarker Analysis for GBA Associated Parkinson's Disease

NCT03811496
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
50 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 23, 2019 · Synced Sep 30, 2026, 3:03 PM 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 Sep 30, 2026, 3:03 PM EDT
Not listed Not applicable Interventional

Identification of Undiagnosed Gaucher Disease

NCT01716741
View directory record Official record
Conditions
Gaucher Disease
Interventions
Enzyme analysis
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 30, 2012 · Synced Sep 30, 2026, 3:03 PM EDT
Recruiting No phase listed Observational

Registry of Patients Diagnosed With Lysosomal Storage Diseases

NCT05619900
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 8, 2026 · Synced Sep 30, 2026, 3:03 PM 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 Sep 30, 2026, 3:03 PM EDT
Completed No phase listed Observational

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00351156
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 19, 2010 · Synced Sep 30, 2026, 3:03 PM EDT