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ONCE: Once-daily eliglustat in stable CYP2D6 intermediate and extensive metabolizers with Gaucher disease type 1
2026-527117-33-00Phase 4Authorised
- Sponsor
- Amsterdam University Medical Center
- Start
- Oct 2026
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 4, open-label, single-arm trial of eliglustat in Gaucher disease type 1 (GD1), sponsored by Amsterdam University Medical Center. Primary endpoint: Clinical stability at Month 12. Planned start Oct 2026; status: authorised.
02
Drugs
eliglustat
03
Conditions
Gaucher disease type 1 (GD1)
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