Full results & analysis
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Velaglucerase Beta in Children With Gaucher Disease
NCT07847411Phase 3
Key facts
- Status
- Not yet recruiting
- Start
- Sep 2026
- Primary completion
- Feb 2028
- Countries
- 1
- Drugs
- velaglucerase beta
- Conditions
- Gaucher disease (GD)
01
Overview
Phase 3, open-label, single-arm trial of velaglucerase beta in Gaucher disease (GD), sponsored by CANbridge Pharmaceuticals Inc. Primary endpoint: Mean percent change in MN spleen volume at 39 weeks. Planned start Sep 2026; status: not yet recruiting.
02
Design
1 arm- Study design
- Non-randomizedOpen-labelSingle group
velaglucerase beta 60 U/kg
All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks.
03
Who it enrols
Children under 12 years of age with a documented diagnosis of Gaucher Disease Type 1 or Type 3, presenting with GD-related splenomegaly and at least one of anemia, hepatomegaly, or thrombocytopenia. Must not have received ERT or SRT for at least 3 months prior to screening.
04
Endpoints
Primary
Mean percent change in MN spleen volume
Secondary
Mean change in hemoglobin concentration
Mean percent change in MN liver volume
Mean percent change in platelet count
Mean change in height Z-score
Mean change in weight Z-score
Beyond the preview
Go deeper on NCT07847411.
Full results, competitive context and analysis are in the Gosset platform.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NCT07847411.