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Showing 25–41 of 17 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Type I Gaucher Disease Clear all
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 9, 2021 · Synced Jul 23, 2026, 4:17 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 14, 2025 · Synced Jul 23, 2026, 4:17 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 23, 2026, 4:17 PM EDT
Terminated Phase 1 Interventional

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

NCT00875160
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 18, 2010 · Synced Jul 23, 2026, 4:17 PM EDT
Completed Phase 1 Interventional

Human Placental-Derived Stem Cell Transplantation

NCT01586455
View directory record Official record
Conditions
Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease, Metachromatic Leukodystrophy, Wolman Disease, Krabbe's Disease, Gaucher's Disease, Fucosidosis, Batten Disease, Severe Aplastic Anemia, Diamond-Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Interventions
Human Placental Derived Stem Cell
Drug
Lead sponsor
New York Medical College
Other
Eligibility
0 Years to 55 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
3
States / cities
Denver, Colorado • Valhalla, New York • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced Jul 23, 2026, 4:17 PM EDT
Completed Phase 2 Interventional Results available

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865
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 and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 14, 2018 · Synced Jul 23, 2026, 4:17 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 17, 2024 · Synced Jul 23, 2026, 4:17 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 23, 2026, 4:17 PM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Jul 23, 2026, 4:17 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

NCT04055831
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Aug 14, 2019 · Synced Jul 23, 2026, 4:17 PM 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 12, 2019 · Synced Jul 23, 2026, 4:17 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 23, 2026, 4:17 PM EDT
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
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
4
States / cities
Los Angeles, California • Chicago, Illinois • Durham, North Carolina + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Jul 23, 2026, 4:17 PM EDT
Recruiting Phase 3 Interventional

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Jul 23, 2026, 4:17 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 5, 2017 · Synced Jul 23, 2026, 4:17 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 23, 2026, 4:17 PM 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 3, 2008 · Synced Jul 23, 2026, 4:17 PM EDT