Conditions: Gaucher Disease Type 1
Keywords: enzyme replacement therapy Type 1 Gaucher Disease miglustat
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 42
Healthy volunteers: no

Brief summary:
Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are required to establish the long term efficacy, safety and tolerability of miglustat in maintaining diseases stability after a switch from ERT.

Detailed description:

Arms:
arm 1: Oral administration of miglustat 100 mg t.i.d. for a period of 2 years

Interventions: intervention 1: Miglustat
Intervention descriptions: intervention 1: Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.)

Protocol document: