ONCE: Once daily eliglustat in stable CYP2D6 intermediate and extensive metabolizers with Gaucher disease type 1
Key facts
- Where
- Amsterdam, HollandiaSee the site
- Phase
- 4. fázis
Where in Hollandia
- Amsterdam UMC Research B.V.Amsterdam, HollandiaToborzás még nem indult
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Amsterdam UMC Stichting
- Trial code
- EU 2026-527117-33-00
- Record updated
- 2026. október 22.
- Original description
To evaluate whether switching from 84 mg BID to 84 mg QD eliglustat maintains clinical stability over a 12-month period in adult GD1 patients who are CYP2D6 IM/EM and clinically stable on long-term BID therapy.Read full original descriptionShow less
To evaluate whether switching from 84 mg BID to 84 mg QD eliglustat maintains clinical stability over a 12-month period in adult GD1 patients who are CYP2D6 IM/EM and clinically stable on long-term BID therapy.
Inclusion: Adults (≥ 18 years) with confirmed GD1 and indication for therapy, as determined by the expert center multidisciplinary team; CYP2D6 intermediate or extensive metabolizer status; Treated with eliglustat 84 mg BID for ≥ 6 months; Stable disease, defined as: o Hemoglobin ≥ 9.7 g/dL (women & men) o Platelets ≥100x109/L o Spleen volume < 10 MN (if present) o Liver volume < 1.5 MN o No bone crisis or symptomatic bone disease in the past 12 months.
Exclusion: Use of strong CYP2D6 or CYP3A inhibitors or inducers, as defined in the eliglustat SmPC; Non-adherence to current therapy or inability to comply with study procedures; Participation in another interventional trial within 3 months before enrolment; Active malignancy or uncontrolled medical condition that may confound study outcomes or pose additional risk; CYP2D6 poor metabolizer (PM) status; Pregnancy or lactation.