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ClinicalTrials.gov public records Last synced Jul 22, 2026, 2:00 PM EDT

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Showing 1–24 of 91 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Gaucher's Disease Clear all
Completed Phase 2 Interventional Results available

A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

NCT00446550
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 Jul 22, 2026, 2:00 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 21, 2025 · Synced Jul 22, 2026, 2:00 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Exploration of Immunity in Gaucher Disease

NCT01358188
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 Jul 22, 2026, 2:00 PM EDT
Withdrawn No phase listed Observational

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 7, 2015 · Synced Jul 22, 2026, 2:00 PM EDT
Completed No phase listed Observational

Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations

NCT00302146
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease
Interventions
15-0 H20
Drug
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
18 Years to 110 Years
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Jul 22, 2026, 2:00 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

MR Spectroscopy and Disease Severity Score for Gaucher in Pediatric Population

NCT01397435
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 3, 2015 · Synced Jul 22, 2026, 2:00 PM EDT
Withdrawn Phase 4 Interventional

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

NCT02528617
View directory record Official record
Conditions
Gaucher Disease Type 1, Gaucher Disease Type 3
Interventions
Velaglucerase alfa
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
4 Years to 14 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 1, 2018 · Synced Jul 22, 2026, 2:00 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

NCT02000310
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 Jul 22, 2026, 2:00 PM EDT
Recruiting No phase listed Observational

The GBA Multimodal Study in Parkinson's Disease

NCT04101968
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 Jul 22, 2026, 2:00 PM 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 3, 2008 · Synced Jul 22, 2026, 2:00 PM EDT
Completed No phase listed Observational

Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease

NCT00244582
View directory record Official record
Conditions
Type 2 Gaucher Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Up to 5 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 22, 2026, 2:00 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 7, 2026 · Synced Jul 22, 2026, 2:00 PM EDT
Recruiting Phase 1 Interventional

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

NCT04532047
View directory record Official record
Conditions
MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions
Aldurazyme (laronidase)
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Jul 22, 2026, 2:00 PM EDT
Withdrawn Phase 4 Interventional

Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease

NCT03021941
View directory record Official record
Conditions
Type 1 Gaucher Disease
Interventions
Elelyso 60 units/kg
Drug
Lead sponsor
Pfizer
Industry
Eligibility
Up to 12 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Sep 23, 2019 · Synced Jul 22, 2026, 2:00 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 – 2027
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 21, 2025 · Synced Jul 22, 2026, 2:00 PM EDT
Completed Phase 2 Interventional

Bone Response to Enzyme Replacement in Gaucher's Disease

NCT00001416
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 Jul 22, 2026, 2:00 PM EDT
Completed Phase 3 Interventional Results available

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

NCT00712348
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 Jul 22, 2026, 2:00 PM EDT
Suspended Phase 1Phase 2 Interventional

Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease

NCT01463215
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 Jul 22, 2026, 2:00 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 Jul 15, 2026 · Synced Jul 22, 2026, 2:00 PM EDT
Completed No phase listed Observational

A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

NCT04721366
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Mar 28, 2024 · Synced Jul 22, 2026, 2:00 PM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

NCT00891202
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Eliglustat tartrate, Placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
6
States / cities
San Francisco, California • New Haven, Connecticut • Decatur, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2017 · Synced Jul 22, 2026, 2:00 PM EDT
Terminated No phase listed Observational

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

NCT04836377
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 28, 2023 · Synced Jul 22, 2026, 2:00 PM EDT
Completed No phase listed Observational

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

NCT04470713
View directory record Official record
Conditions
GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
Interventions
Not listed
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
0 Months and older
Enrollment
226 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
3
States / cities
Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Nov 7, 2021 · Synced Jul 22, 2026, 2:00 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 9, 2021 · Synced Jul 22, 2026, 2:00 PM EDT