Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
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ClinicalTrials.gov Identifier: NCT00319046 |
Recruitment Status :
Completed
First Posted : April 27, 2006
Results First Posted : May 23, 2012
Last Update Posted : November 21, 2018
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Condition or disease | Intervention/treatment | Phase |
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Gaucher Disease Type 1 | Drug: Miglustat | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 42 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official 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 |
Study Start Date : | February 1, 2006 |
Actual Primary Completion Date : | June 1, 2010 |
Actual Study Completion Date : | July 1, 2010 |

Arm | Intervention/treatment |
---|---|
Experimental: Open-label miglustat
Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
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Drug: Miglustat
Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.)
Other Name: Zavesca |
- Liver Volume at Baseline and at End of Treatment [ Time Frame: Baseline and end of treatment (Month 24) ]Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging. Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the 'worst' within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
- Mean Within-patient Percent Change From Baseline in Liver Volume [ Time Frame: End of treatment (Month 24) ]Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging. Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the 'worst' within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
- Spleen Volume at Baseline and End of Treatment [ Time Frame: Baseline and end of treatment (Month 24) ]
Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging.
Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the 'worst' within-patient value not more than 2 days after the end of treatment was used to impute the missing value.
- Mean Percent Change From Baseline in Spleen Volume [ Time Frame: End of treatment (Month 24) ]
Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging.
Imputation methods for patients with missing values at Month 24 were applied as follows: if a patient had at least 640 days of treatment with the study drug, the last observation that was not more than 2 days after the end of treatment was carried forward. If a patient had discontinued study drug before Day 640, the 'worst' within-patient value not more than 2 days after the end of treatment was used to impute the missing value.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.
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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:
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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.
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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.
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- 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.
- History of significant lactose intolerance.
- 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.
- History of cataracts or known increased risk of cataract formation.
- Severe renal impairment i.e., with a creatinine clearance <30 ml/min/1.73m^2
- Concomitant active medical condition such as human immunodeficiency virus (HIV) or hepatitis B/C that would render patients unsuitable for study.
- Previous treatment with miglustat.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00319046

Principal Investigator: | Timothy Cox, Prof | University of Cambridge |
Responsible Party: | Actelion |
ClinicalTrials.gov Identifier: | NCT00319046 |
Other Study ID Numbers: |
OGT 918-011 |
First Posted: | April 27, 2006 Key Record Dates |
Results First Posted: | May 23, 2012 |
Last Update Posted: | November 21, 2018 |
Last Verified: | October 2018 |
enzyme replacement therapy Type 1 Gaucher Disease miglustat |
Gaucher Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases |
Metabolic Diseases Lipid Metabolism Disorders Miglustat Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Anti-HIV Agents Anti-Retroviral Agents Antiviral Agents Anti-Infective Agents Glycoside Hydrolase Inhibitors Hypoglycemic Agents Physiological Effects of Drugs |