Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patien… (NCT00319046) | Clinical Trial Compass
CompletedPhase 3
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
42 participantsStarted 2006-02-01
Plain-language 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.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Males or females aged 18 years or older
✓. Type 1 Gaucher disease, diagnosed by glucocerebrosidase assay or molecular analysis of the glucocerebrosidase gene.
✓. Treatment with ERT for at least 3 years, with a stable dose regimen for at least the last 6 months.
✓. Clinically and biologically stable disease for the previous 2 years, with at least 2 time points assessments (including baseline as one potential time point), defined as:
✓. Written informed consent.
Exclusion criteria
✕. History or evidence of oculomotor gaze palsy, ataxia or other clinical manifestations typically associated with neuronopathic type 3 Gaucher disease.
✕. Not ambulant patients, or with progressive symptomatic documented bone disease.
✕. Splenectomy before 18 years of age for splenomegaly and/or thrombocytopenia.
✕. Peripheral polyneuropathy (not mononeuropathy) documented with both clinical signs and symptoms, and electrodiagnostic (EDX).
✕. Patients (males and females) who do not agree to use reliable contraception throughout the study and for 3 months after cessation of miglustat treatment.
✕. Female patients who are pregnant or breast feeding, or without pregnancy test prior to Day 1.
✕
What they're measuring
1
Liver Volume at Baseline and at End of Treatment
Timeframe: Baseline and end of treatment (Month 24)
2
Mean Within-patient Percent Change From Baseline in Liver Volume
✕. Clinically significant diarrhea (\>3 liquid stools per day for \>7 days) without definable cause within 6 months prior to Day 1, or a history of clinically relevant gastrointestinal disorders.