RecruitingPhase 3NCT07223944
Metabolic
Gaucher disease type 1
8
studies recruiting now
as of 7 Sept 2026
70
studies registered in total
as of 7 Sept 2026
11
countries with a recruiting site
as of 7 Sept 2026
3 Nov 2025
most recent study posted
among recruiting studies
Recruiting trials
RecruitingNCT02437396
Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
RecruitingNCT05253560
Prodromal Parkinsonian Features in GBA1 Mutation Carriers
RecruitingPhase 1 / Phase 2NCT06818838
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
RecruitingNCT04528355
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 8 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Patient organisations
We do not yet list a dedicated organisation for this condition. The directories below are the best route.
About Gaucher disease type 1
RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations, and the genes involved (GBA1, SCARB2).
Treatments being studied
2 approved treatments and 4 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Miglustat (Miglustat dipharma)Approved: Velaglucerase Alfa (Vpriv)
Phase 3Eliglustat Tartrate (Cerdelga)
Phase 3Imiglucerase (Cerezyme)
Phase 2Venglustat
Phase 2Acetylcysteine (A-cys)
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).