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Gaucher Disease Clinical Trial Landscape

Data updated: May 20, 2026

50 clinical trials tracked since 2008

Gaucher Disease is being studied across 50 clinical trials registered since 2008, with 8 programs currently active. The competitive pipeline includes 1 active Phase 3 trials, 6 active Phase 2 trials, and 5 active Phase 1 trials. Top industry sponsors include Spur Therapeutics, Prevail Therapeutics, Genzyme, a Sanofi Company.

8 active
/
50 total (since 2008)
5
Phase 1 Active
16 total
6
Phase 2 Active
17 total
1
Phase 3 Active
18 total
0
Phase 4 Active
7 total

Top Industry Sponsors

Sorted by active Active Done Failed
Spur Therapeutics 2 1 0
Prevail Therapeutics 2 0 0
Genzyme, a Sanofi Company 1 6 0
Lingyi Biotech Co., Ltd. 1 0 0
Takeda 0 8 0

+ 3 more sponsors — see full table below

Featured Phase 3 Trials

NCT07223944 RECRUITING
A Gaucher Disease Gene Therapy Trial With FLT201
Spur Therapeutics n=45
NCT05529992 COMPLETED
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
Takeda n=20
NCT06211478 COMPLETED
Role of Vitamin E in Gaucher Disease Patients
Ain Shams University n=40
NCT01422187 COMPLETED
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
Pfizer n=19
NCT02536755 COMPLETED
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
Genzyme, a Sanofi Company n=31

+ 13 more Phase 3 trials — see full database below

Gaucher Disease — Trials Started by Year (last 10 years)

2016
2
2017
1
2018
1
2019
3
2020
2
2021
3
2022
3
2023
3
2024
3
2025
0
2026
1

TheraRadar.com

Full Trial Database

Complete sponsor table, every trial across all phases, plus competitive analytics. Sortable, filterable, exportable for Pro users.

Top Sponsors (all-time)

Spur Therapeutics
3
Prevail Therapeutics
2
Genzyme, a Sanofi Company
7
Lingyi Biotech Co., Ltd.
1
Takeda
8
Pfizer
6
ISU Abxis Co., Ltd.
4
Amicus Therapeutics
3
AVROBIO
2
Protalix
2

Active Trials by Phase

Phase 1
5
Phase 2
6
Phase 3
1

Enrollment Size Distribution

1-50
42
51-200
4
201-500
0
501-1k
0
1k+
0

All Industry Sponsors

Sorted by active Active Done Failed
Spur Therapeutics 2 1 0
Prevail Therapeutics 2 0 0
Genzyme, a Sanofi Company 1 6 0
Lingyi Biotech Co., Ltd. 1 0 0
Takeda 0 8 0
Pfizer 0 5 1
ISU Abxis Co., Ltd. 0 3 1
Amicus Therapeutics 0 2 1
NCT07223944 RECRUITING
A Gaucher Disease Gene Therapy Trial With FLT201
Spur Therapeutics n=45
NCT05529992 COMPLETED
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
Takeda n=20
NCT06211478 COMPLETED
Role of Vitamin E in Gaucher Disease Patients
Ain Shams University n=40
NCT05815004 WITHDRAWN
An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3
AVROBIO
NCT01422187 COMPLETED
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
Pfizer n=19
NCT02536755 COMPLETED
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
Genzyme, a Sanofi Company n=31
NCT01614574 COMPLETED
Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Shire n=6
NCT01842841 COMPLETED
Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
Shire n=5
NCT00635427 COMPLETED
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
Shire n=95
NCT00553631 COMPLETED
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
Shire n=34
NCT00712348 COMPLETED
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
Pfizer n=31
NCT00705939 COMPLETED
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Pfizer n=45
NCT01411228 COMPLETED
A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Pfizer n=15
NCT02770625 COMPLETED
Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
ISU Abxis Co., Ltd. n=8
NCT00891202 COMPLETED
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
Genzyme, a Sanofi Company n=40
NCT01074944 COMPLETED
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)
Genzyme, a Sanofi Company n=170
NCT00943111 COMPLETED
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
Genzyme, a Sanofi Company n=160
NCT01161914 WITHDRAWN
The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease
ISU Abxis Co., Ltd.

Full Gaucher Disease Pipeline

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Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only