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Oral Miglustat in Adult Patients With Stable Type 1 Gaucher Disease
This study is ongoing, but not recruiting participants.
First Received: April 26, 2006   Last Updated: July 1, 2008   History of Changes
Sponsored by: Actelion
Information provided by: Actelion
ClinicalTrials.gov Identifier: NCT00319046
  Purpose

Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for 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 III

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency mitochondrial trifunctional protein deficiency primary carnitine deficiency succinic semialdehyde dehydrogenase deficiency
MedlinePlus related topics: Gaucher's Disease
Drug Information available for: SC 48334
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
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

Further study details as provided by Actelion:

Primary Outcome Measures:
  • Percent change from baseline to Month 24 in liver volume [ Time Frame: From baseline to Month 24 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Percent change from baseline to Month 24 in spleen volume [ Time Frame: From Baseline to Month 24 ] [ Designated as safety issue: No ]

Enrollment: 42
Study Start Date: February 2006
Estimated Study Completion Date: July 2010
Estimated Primary Completion Date: June 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
1: Experimental Drug: miglustat
miglustat oral capsules 100mg TID

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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 MRI or CT):

      • Liver volume within 10% of the mean.
      • Spleen volume within 10% of the mean.
    • Free of progressive symptomatic documented bone disease (see Appendix 1).
    • 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., ACE, 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 (see Appendix 1).
  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/1.73mP2P
  11. Concomitant active medical condition such as HIV or hepatitis B/C that would render patients unsuitable for study.
  12. Previous treatment with miglustat.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00319046

  Show 20 Study Locations
Sponsors and Collaborators
Actelion
Investigators
Principal Investigator: Timothy Cox, Prof University of Cambridge
  More Information

Additional Information:
No publications provided

Responsible Party: Actelion ( Ruben Giorgino, MD )
Study ID Numbers: OGT 918-011
Study First Received: April 26, 2006
Last Updated: July 1, 2008
ClinicalTrials.gov Identifier: NCT00319046     History of Changes
Health Authority: United States: Food and Drug Administration

Keywords provided by Actelion:
Type 1 Gaucher Disease
miglustat
enzyme replacement therapy

Study placed in the following topic categories:
Lipid Metabolism, Inborn Errors
Sphingolipidoses
Anti-HIV Agents
Metabolic Diseases
Lysosomal Storage Diseases
Sphingolipidosis
Central Nervous System Diseases
Brain Diseases
Antiviral Agents
Lymphatic Diseases
Metabolism, Inborn Errors
Miglustat
Anti-Retroviral Agents
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Lipidoses
Gaucher Disease
Metabolic Disorder
Lipid Metabolism Disorders
Brain Diseases, Metabolic

Additional relevant MeSH terms:
Lipid Metabolism, Inborn Errors
Anti-Infective Agents
Sphingolipidoses
Molecular Mechanisms of Pharmacological Action
Brain Diseases
Metabolism, Inborn Errors
Anti-Retroviral Agents
Therapeutic Uses
Brain Diseases, Metabolic, Inborn
Reticuloendotheliosis
Metabolic Diseases
Anti-HIV Agents
Lysosomal Storage Diseases, Nervous System
Lysosomal Storage Diseases
Nervous System Diseases
Central Nervous System Diseases
Enzyme Inhibitors
Antiviral Agents
Pharmacologic Actions
Lymphatic Diseases
Miglustat
Genetic Diseases, Inborn
Lipidoses
Gaucher Disease
Brain Diseases, Metabolic
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on May 06, 2009