Trial record 2 of 18 for:
"Gaucher disease type 1"
Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease
This study has been completed.
Sponsor:
Actelion
Information provided by (Responsible Party):
Actelion
ClinicalTrials.gov Identifier:
NCT00319046
First received: April 26, 2006
Last updated: May 24, 2012
Last verified: May 2012
- Full Text View
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Type 1 Gaucher Disease |
Drug: miglustat |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
cholesteryl ester storage disease
Farber lipogranulomatosis
Gaucher disease
Schindler disease
succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics:
Gaucher's Disease
Drug Information available for:
Miglustat
U.S. FDA Resources
Further study details as provided by Actelion:
Primary Outcome Measures:
- Liver Volume [ Time Frame: baseline to end of treatment (month 24 or imputed value) ] [ Designated as safety issue: No ]Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging
- Percent Change in Liver Volume [ Time Frame: baseline to end of treatment (month 24 or imputed value) ] [ Designated as safety issue: No ]Liver volume was assessed at baseline and end of treatment by magnetic resonance imaging
Secondary Outcome Measures:
- Spleen Volume [ Time Frame: baseline to end of treatment (month 24 or imputed value) ] [ Designated as safety issue: No ]Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging
- Percent Change in Spleen Volume [ Time Frame: baseline to end of treatment (month 24 or imputed value) ] [ Designated as safety issue: No ]Spleen volume was assessed at baseline and end of treatment by magnetic resonance imaging
| Enrollment: | 42 |
| Study Start Date: | February 2006 |
| Study Completion Date: | July 2010 |
| Primary Completion Date: | June 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: 1 |
Drug: miglustat
miglustat oral capsules 100mg three times daily (TID)
Other Name: Zavesca
|
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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:
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 > 100x109 /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.
- 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00319046
Show 20 Study Locations
Show 20 Study LocationsSponsors and Collaborators
Actelion
Investigators
| Principal Investigator: | Timothy Cox, Prof | University of Cambridge |
More Information
No publications provided by Actelion
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Actelion |
| ClinicalTrials.gov Identifier: | NCT00319046 History of Changes |
| Other Study ID Numbers: | OGT 918-011 |
| Study First Received: | April 26, 2006 |
| Results First Received: | April 24, 2012 |
| Last Updated: | May 24, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Actelion:
|
Type 1 Gaucher Disease miglustat enzyme replacement therapy |
Additional relevant MeSH terms:
|
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 Pharmacologic Actions Anti-HIV Agents Anti-Retroviral Agents Antiviral Agents Anti-Infective Agents Therapeutic Uses |
ClinicalTrials.gov processed this record on June 17, 2013