2026-527117-33-00Not yet recruitingPhase 4
ONCE: Once daily eliglustat in stable CYP2D6 intermediate and extensive metabolizers with Gaucher disease type 1
Source: EU CTIS (euclinicaltrials.eu) · Last updated 22 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 7 (estimated)
- First submitted
- 11 Aug 2026
- Start date
- 1 Oct 2026
- Lead sponsor
- Amsterdam UMC Stichting
- Diseases
- Gaucher disease
- EU CT number
- 2026-527117-33-00
What you commit to
- Study type: Interventional · Phase 4
- Planned participants: 7
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study design.
AI-kivonat: who the study is looking for
Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with Gaucher disease type 1 who are stable on eliglustat 84 mg twice a day and are CYP2D6 intermediate or extensive metabolizers. It tests a switch to once a day.
- Age
- 18 years or older
- Sex
- All sexes can join.
- Disease stage
- Confirmed Gaucher disease type 1 with an indication for therapy, and stable disease: hemoglobin ≥ 9.7 g/dL, platelets ≥100x109/L, spleen volume < 10 MN (if present), liver volume < 1.5 MN, and no bone crisis or symptomatic bone disease in the past 12 months.
- Earlier treatment
- Treated with eliglustat 84 mg twice a day (BID) for ≥ 6 months.
- Biomarker or test result
- CYP2D6 intermediate or extensive metabolizer status.
Main reasons for not taking part
- CYP2D6 poor metabolizer status
- Active malignancy or an uncontrolled medical condition that may affect the results or add risk
- Pregnancy or breastfeeding
- Use of strong CYP2D6 or CYP3A inhibitors or inducers
- Not taking current treatment as directed, or unable to follow study procedures
Read by machine on 9 Nov 2026.
Who cannot take part
Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- Active malignancy or uncontrolled medical condition that may confound study outcomes or pose additional risk.
Other
- CYP2D6 poor metabolizer (PM) status.
- Pregnancy or lactation.
Who the study is looking for
- 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
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Amsterdam UMC Research B.V. | Amsterdam | Netherlands | Not yet recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.