Metabolic

Gaucher disease type 2

ORPHA:77260 ↗Gene GBA1Clinical subtype

22

studies recruiting now

as of 7 Sept 2026

112

studies registered in total

as of 7 Sept 2026

7

countries with a recruiting site

as of 7 Sept 2026

4 Aug 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 22 recruiting studies

Where 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 2

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 gene involved (GBA1).

Treatments being studied

3 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Phase 1/2Sirolimus (Fyarro)
Phase 1/2Methylprednisolone (Medrol)
Phase 1/2Prednisone (Cortan)

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).