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Trial record 35 of 99 for:    "Gaucher disease"

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
Sponsor:
Information provided by (Responsible Party):
Actelion

Brief Summary:
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 or disease Intervention/treatment Phase
Gaucher Disease Type 1 Drug: Miglustat Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 42 participants
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
Drug: Miglustat
Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.)
Other Name: Zavesca




Primary Outcome Measures :
  1. 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.

  2. 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.


Secondary Outcome Measures :
  1. 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.


  2. 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
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.

Information from the National Library of Medicine

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


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

Publications of Results:
Responsible Party: Actelion
ClinicalTrials.gov Identifier: NCT00319046     History of Changes
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

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

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
1-Deoxynojirimycin
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