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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT00319046
Registration number
NCT00319046
Ethics application status
Date submitted
26/04/2006
Date registered
27/04/2006
Date last updated
21/11/2018
Titles & IDs
Public title
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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Scientific 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
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Secondary ID [1]
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OGT 918-011
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease Type 1
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Miglustat
Experimental: Open-label miglustat - Oral administration of miglustat 100 mg t.i.d. for a period of 2 years
Treatment: Drugs: Miglustat
Oral capsules containing miglustat 100 mg, administered three times daily (t.i.d.)
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Liver Volume at Baseline and at End of Treatment
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline and end of treatment (Month 24)
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Primary outcome [2]
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Mean Within-patient Percent Change From Baseline in Liver Volume
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Assessment method [2]
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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.
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Timepoint [2]
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End of treatment (Month 24)
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Secondary outcome [1]
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Spleen Volume at Baseline and End of Treatment
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Assessment method [1]
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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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Timepoint [1]
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Baseline and end of treatment (Month 24)
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Secondary outcome [2]
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Mean Percent Change From Baseline in Spleen Volume
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Assessment method [2]
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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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Timepoint [2]
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End of treatment (Month 24)
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Eligibility
Key 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.
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/02/2006
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/07/2010
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Sample size
Target
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Accrual to date
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Final
42
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Royal Perth Hospital - Perth
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Recruitment hospital [2]
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Royal Brisbane and Women's Hospital - Queensland
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Recruitment hospital [3]
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Royal Melbourne Hospital - Victoria
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Recruitment postcode(s) [1]
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- Perth
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Recruitment postcode(s) [2]
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- Queensland
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Recruitment postcode(s) [3]
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- Victoria
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Recruitment outside Australia
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United States of America
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California
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United States of America
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District of Columbia
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Georgia
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New York
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Oregon
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Wisconsin
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Brazil
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Porto Alegre
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Canada
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Ontario
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Czechia
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Prague
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France
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Clichy
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Germany
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Mainz
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Hungary
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Debrecen
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Italy
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Trieste
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Netherlands
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Amsterdam
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Spain
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Zaragoza
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Taiwan
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Taipei
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United Kingdom
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State/province [17]
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Cambridge
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Actelion
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Address
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT00319046
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Timothy Cox, Prof
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Address
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University of Cambridge
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT00319046
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