Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00319046
• Date of registration: 26/04/2006
• Prospective Registration: No
• Primary sponsor: Actelion
• Public title: Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
• Scientific title: Open-label, Non Comparative, Multi-center Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Oral Miglustat as a Maintenance Therapy After a Switch From Enzyme Replacement Therapy in Adult Patients With Stable Type 1 Gaucher Disease
• Date of first enrolment: February 1, 2006
• Target sample size: 42
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00319046
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Brazil, Canada, Czech Republic, Czechia, France, Germany, Hungary, Italy, Netherlands, Spain, Taiwan, United Kingdom, United States

Contacts

• Name: Timothy Cox, Prof
• Affiliation: University of Cambridge

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Males or females aged 18 years or older

2. Type 1 Gaucher disease, diagnosed by glucocerebrosidase assay or molecular analysis of
the glucocerebrosidase gene.

3. Treatment with ERT for at least 3 years, with a stable dose regimen for at least the
last 6 months.

4. 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:

- Stable organomegaly (assessed by magnetic resonance imaging (MRI) or computed
tomography (CT)):

- Liver volume within 10% of the mean.

- Spleen volume within 10% of the mean.

- Free of progressive symptomatic documented bone disease.

- Hemoglobin levels > 11g/dl

- Mean platelet count > 100x10^9 /l.

- Chitotriosidase activity within 20% of the mean.

- If chitotriosidase is not available (in the case of chitotriosidase
deficiency, or if it was not determined), other relevant biomarkers (e.g.,
angiotensin converting enzyme (ACE), tartrate resistant acid phosphatase
(TRAP) and ferritin) could be considered.

5. Written informed consent.

Exclusion Criteria:

1. History or evidence of oculomotor gaze palsy, ataxia or other clinical manifestations
typically associated with neuronopathic type 3 Gaucher disease.

2. Not ambulant patients, or with progressive symptomatic documented bone disease.

3. Splenectomy before 18 years of age for splenomegaly and/or thrombocytopenia.

4. Peripheral polyneuropathy (not mononeuropathy) documented with both clinical signs and
symptoms, and electrodiagnostic (EDX).

5. Patients (males and females) who do not agree to use reliable contraception throughout
the study and for 3 months after cessation of miglustat treatment.

6. Female patients who are pregnant or breast feeding, or without pregnancy test prior to
Day 1.

7. History of significant lactose intolerance.

8. 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.

9. History of cataracts or known increased risk of cataract formation.

10. Severe renal impairment i.e., with a creatinine clearance <30 ml/min/1.73m^2

11. Concomitant active medical condition such as human immunodeficiency virus (HIV) or
hepatitis B/C that would render patients unsuitable for study.

12. Previous treatment with miglustat.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Gaucher Disease Type 1

Intervention(s)

Drug: Miglustat

Primary Outcome(s)

Liver Volume at Baseline and at End of Treatment [Time Frame: Baseline and end of treatment (Month 24)]

Mean Within-patient Percent Change From Baseline in Liver Volume [Time Frame: End of treatment (Month 24)]

Secondary Outcome(s)

Mean Percent Change From Baseline in Spleen Volume [Time Frame: End of treatment (Month 24)]

Spleen Volume at Baseline and End of Treatment [Time Frame: Baseline and end of treatment (Month 24)]

Secondary ID(s)

OGT 918-011

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 23/05/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00319046