Diseases / Gaucher Disease
Gaucher Disease Clinical Trials
DiseaseGaucher disease is an inherited lysosomal storage disorder caused by deficient glucocerebrosidase activity, leading to accumulation of fatty substances in macrophages and multisystem manifestations.
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Recruiting Phase 3
| Trial | Phase | Status | Sponsor | Started |
|---|---|---|---|---|
| A Gaucher Disease Gene Therapy Trial With FLT201 NCT07223944 | Phase 3 | Recruiting | Spur Therapeutics | Started 2026-04 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT NCT01962415 | Phase 2 | Recruiting | Paul Szabolcs | Started 2014-02 |
Recently started
| Trial | Phase | Status | Sponsor | Started |
|---|---|---|---|---|
| Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3) NCT07715084 | Phase 2 | Not yet recruiting | Azafaros B.V. | Started 2026-09 |
| Nutritional Status of Gaucher Disease Patients NCT07845188 | Phase unknown | Not yet recruiting | Assiut University | Started 2026-09 |
| Study of Velaglucerase Beta ERT in Children With Gaucher Disease NCT07847411 | Phase 3 | Not yet recruiting | CANbridge Life Sciences Ltd. | Started 2026-09 |
| A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease NCT07603050 | Phase 1/2 | Not yet recruiting | Shanghai Vitalgen BioPharma Co., Ltd. | Started 2026-05 |
| Advancing Integrated Therapies for Gaucher Disease NCT07758816 | Phase unknown | Recruiting | Yale University | Started 2026-05 |
Results recently posted
| Trial | Phase | Status | Sponsor | Results posted |
|---|---|---|---|---|
| A Gene Therapy Study in Patients With Gaucher Disease Type 1 NCT05324943 | Phase 1 | Completed | Spur Therapeutics | Results posted 2026-06-02 |
| A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease NCT05529992 | Phase 3 | Completed | Takeda | Results posted 2025-02-26 |
| Survey Study for Velaglucerase Alfa (VPRIV) in Japan NCT03625882 | Phase unknown | Completed | Takeda | Results posted 2025-01-10 |
| A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease NCT04002830 | Phase 4 | Completed | Ari Zimran | Results posted 2024-12-17 |
| Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease NCT04145037 | Phase 1/2 | Terminated | AVROBIO | Results posted 2024-01-18 |
Pipeline
Phase by status
| Phase | Recruiting | Not yet | By invitation | Active | Completed | Terminated | Withdrawn | Suspended | Unknown | Approved for marketing | Available | No longer available | Total |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Early Phase 1 | 1 | — | — | 1 | — | — | — | — | — | — | — | — | 2 |
| Phase 1 | 2 | — | — | 1 | 16 | 1 | — | — | — | — | — | — | 20 |
| Phase 1/2 | 1 | 2 | — | 4 | 2 | 1 | — | 1 | 1 | — | — | — | 12 |
| Phase 2 | 1 | 1 | — | 1 | 11 | 1 | — | — | 3 | — | — | — | 18 |
| Phase 2/3 | — | — | — | — | 2 | — | 1 | — | — | — | — | — | 3 |
| Phase 3 | 1 | 1 | — | 1 | 19 | — | 1 | — | — | — | — | — | 23 |
| Phase 4 | — | — | — | — | 8 | — | 2 | — | 1 | — | — | — | 11 |
| Not applicable | 1 | — | — | — | 7 | — | — | — | 4 | — | — | — | 12 |
| Unknown | 15 | 3 | 3 | 1 | 26 | 3 | 3 | — | 15 | 1 | 1 | 1 | 72 |
Trials started per year
Conditions studied, by phase
| Condition | Early Phase 1 | Phase 1 | Phase 1/2 | Phase 2 | Phase 2/3 | Phase 3 | Phase 4 | Not applicable | Unknown | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Gaucher Disease | 2 | 20 | 12 | 18 | 3 | 23 | 11 | 12 | 72 | 173 |
| Lysosomal Storage Diseases | — | — | — | — | — | 1 | — | 2 | 10 | 13 |
| Niemann-Pick Diseases | — | 1 | — | — | 1 | — | — | — | 8 | 10 |
| Metabolism, Inborn Errors | — | 3 | — | 2 | — | — | — | — | 4 | 9 |
| Parkinson Disease | — | 1 | 1 | — | — | — | — | — | 7 | 9 |
| Fabry Disease | — | — | — | — | — | — | — | 2 | 5 | 7 |
| Leukodystrophy, Metachromatic | — | 1 | — | 1 | 1 | — | — | — | 4 | 7 |
| Rare Diseases | — | 1 | — | 2 | — | — | — | — | 4 | 7 |
| Wolman Disease | — | 2 | — | — | 1 | — | — | — | 4 | 7 |
| Adrenoleukodystrophy | — | 1 | — | 1 | 1 | — | — | — | 3 | 6 |
Most active sponsors
| # | Sponsor | Trials | Phase 3 |
|---|---|---|---|
| 1 | Shire | 14 | 6 |
| 2 | Genzyme, a Sanofi Company | 12 | 4 |
| 3 | Sanofi | 11 | 2 |
| 4 | Pfizer | 8 | 5 |
| 5 | Shaare Zedek Medical Center | 7 | 0 |
| 6 | National Institute of Neurological Disorders and Stroke (NINDS) | 6 | 0 |
| 7 | Takeda | 6 | 1 |
| 8 | University of Minnesota | 6 | 0 |
| 9 | Amicus Therapeutics | 5 | 0 |
| 10 | ISU Abxis Co., Ltd. | 5 | 2 |
Most studied interventions
| # | Intervention | Trials |
|---|---|---|
| 1 | eliglustat | 14 |
| 2 | Glucosylceramidase | 14 |
| 3 | imiglucerase | 13 |
| 4 | taliglucerase alfa | 11 |
| 5 | AT2101 | 4 |
| 6 | Ambroxol | 3 |
| 7 | miglustat | 3 |
| 8 | Acetylcysteine | 2 |
| 9 | Blood Specimen Collection | 2 |
| 10 | Cyclophosphamide | 2 |
Terminations
Stated reasons
Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.
Recently stopped
| Trial | Status | Stated reason | Stopped |
|---|---|---|---|
| NCT04145037 Terminated | Terminated | This study was voluntarily terminated by the sponsor to halt development and was not based on any safety or medical reasons. | 2023-08-21 |
| NCT04836377 Terminated | Terminated | This study was voluntarily terminated due to a business decision not to proceed, and not due to any safety or efficacy issue | 2023-08-21 |
| NCT03896607 Terminated | Terminated | study has ended due to internal changes | 2022-10-31 |
| NCT04094181 Terminated | Terminated | The decision was taken after careful consideration relating to recruitment challenges that could impact the scientific value of the study. | 2021-11-17 |
| NCT03746587 Terminated | Terminated | COVID-19 pandemic made it impossible to assess the trial objective | 2021-10-15 |
| NCT01331642 Withdrawn | Withdrawn | Transition to BioMetabol | 2021-02-28 |
| NCT02528617 Withdrawn | Withdrawn | No subjects accrued and no potential subjects were identified.. | 2017-10-31 |
| NCT00795197 Withdrawn | Withdrawn | No recruitment of patients | 2009-12-31 |
| NCT00875160 Terminated | Terminated | Subject Recruitment | 2009-07-31 |
| NCT01161914 Withdrawn | Withdrawn | — |
Evidence
- Results reported
- 38.5%35 of 91 completed
- Published
- 15.4%14 of 91 completed
- Primary endpoint met
- 100%4 of 4 analyses
- Median months to report
- 14
Documents disclosed
Trial profile
- Median enrollment
- 31
- Median duration
- 40.3months
- Countries
- 60
- Study sites
- 965
Who is enrolled
Median enrollment by phase
- Early Phase 1
- 5
- Phase 1
- 17
- Phase 1/2
- 12
- Phase 2
- 28
- Phase 2/3
- 40
- Phase 3
- 32
- Phase 4
- 12
- Not applicable
- 25
- Unknown
- 60
Where trials run
Busiest cities
| # | City | Country | Trials |
|---|---|---|---|
| 1 | Jerusalem | Israel | 29 |
| 2 | New York | United States | 25 |
| 3 | Fairfax | United States | 21 |
| 4 | London | United Kingdom | 19 |
| 5 | Zaragoza | Spain | 17 |
| 6 | Minneapolis | United States | 15 |
| 7 | Decatur | United States | 14 |
| 8 | Pittsburgh | United States | 14 |
| 9 | Coral Springs | United States | 13 |
| 10 | Bethesda | United States | 11 |