Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Actelion
ClinicalTrials.gov Identifier:
NCT00319046
First received: April 26, 2006
Last updated: May 24, 2012
Last verified: May 2012

April 26, 2006
May 24, 2012
February 2006
June 2010   (final data collection date for primary outcome measure)
  • 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
Percent change from baseline to Month 24 in liver volume
Complete list of historical versions of study NCT00319046 on ClinicalTrials.gov Archive Site
  • 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
Percent change from baseline to Month 24 in spleen volume
Not Provided
Not Provided
 
Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease
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

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.

Not Provided
Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Type 1 Gaucher Disease
Drug: miglustat
miglustat oral capsules 100mg three times daily (TID)
Other Name: Zavesca
Experimental: 1
Intervention: Drug: miglustat
Cox TM, Amato D, Hollak CE, Luzy C, Silkey M, Giorgino R, Steiner RD; Miglustat Maintenance Study Group. Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study. Orphanet J Rare Dis. 2012 Dec 27;7:102. doi: 10.1186/1750-1172-7-102.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
42
July 2010
June 2010   (final data collection date for primary outcome measure)

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 > 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.
  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.
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States,   Australia,   Brazil,   Canada,   Czech Republic,   France,   Germany,   Hungary,   Italy,   Netherlands,   Spain,   Taiwan,   United Kingdom
 
NCT00319046
OGT 918-011
Not Provided
Actelion
Actelion
Not Provided
Principal Investigator: Timothy Cox, Prof University of Cambridge
Actelion
May 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP