TheraRadar
Landscape / Rare Disease
Data updated: Jun 28, 2026

Gaucher Disease Clinical Trial Landscape

Gaucher Disease is being studied across 51 clinical trials registered since 2008, with 9 programs currently active. The competitive pipeline includes 1 active Phase 3 trials, 7 active Phase 2 trials, and 1 active Phase 1 trials.

Top industry sponsors include Spur Therapeutics, Prevail Therapeutics, Genzyme, a Sanofi Company.

Trial activity

9 active / 51 total since 2008
Active by phase 1 Ph3 / 18 7 Ph2 / 17 1 Ph1 / 9 0 Ph4 / 7

Sponsor activity

Who is running trials now — green active, blue completed, red failed/terminated.

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
Shanghai Vitalgen BioPharma 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

All 9 active Gaucher Disease sponsors

Unlock the remaining 1 sponsors with active / completed / failed counts — sortable and exportable.

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How the field has grown

New-trial starts peaked in 2019 (3 registered); 2025 saw 0. The right-hand chart shows median Phase 3 enrollment by start year — the number in parentheses is that year's Phase 3 trial count (3 in total), so single-trial years (and years with no Phase 3 starts) are obvious. Both are by trial start date; the current year is partial.

New trials started by year

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

TheraRadar.com

Median Phase 3 enrollment by start year

2016 (0)
0
2017 (0)
0
2018 (0)
0
2019 (0)
0
2020 (0)
0
2021 (0)
0
2022 (0)
0
2023 (2)
30
2024 (0)
0
2025 (0)
0
2026 (1)
45

TheraRadar.com

Full trial pipeline

Every active and completed trial across Phase 1–4, with enrollment analytics. Sortable, filterable, exportable with Pro.

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NCT05815004 WITHDRAWN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

Every trial across Phase 1–4, plus enrollment analytics. Sortable, filterable, exportable.

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