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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
Secondary ID [1] 0 0
OGT 918-011
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Gaucher Disease Type 1 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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.)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Liver Volume at Baseline and at End of Treatment
Timepoint [1] 0 0
Baseline and end of treatment (Month 24)
Primary outcome [2] 0 0
Mean Within-patient Percent Change From Baseline in Liver Volume
Timepoint [2] 0 0
End of treatment (Month 24)
Secondary outcome [1] 0 0
Spleen Volume at Baseline and End of Treatment
Timepoint [1] 0 0
Baseline and end of treatment (Month 24)
Secondary outcome [2] 0 0
Mean Percent Change From Baseline in Spleen Volume
Timepoint [2] 0 0
End of treatment (Month 24)

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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/02/2006
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/07/2010
Sample size
Target
Accrual to date
Final
42
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Perth Hospital - Perth
Recruitment hospital [2] 0 0
Royal Brisbane and Women's Hospital - Queensland
Recruitment hospital [3] 0 0
Royal Melbourne Hospital - Victoria
Recruitment postcode(s) [1] 0 0
- Perth
Recruitment postcode(s) [2] 0 0
- Queensland
Recruitment postcode(s) [3] 0 0
- Victoria
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Oregon
Country [6] 0 0
United States of America
State/province [6] 0 0
Wisconsin
Country [7] 0 0
Brazil
State/province [7] 0 0
Porto Alegre
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Czechia
State/province [9] 0 0
Prague
Country [10] 0 0
France
State/province [10] 0 0
Clichy
Country [11] 0 0
Germany
State/province [11] 0 0
Mainz
Country [12] 0 0
Hungary
State/province [12] 0 0
Debrecen
Country [13] 0 0
Italy
State/province [13] 0 0
Trieste
Country [14] 0 0
Netherlands
State/province [14] 0 0
Amsterdam
Country [15] 0 0
Spain
State/province [15] 0 0
Zaragoza
Country [16] 0 0
Taiwan
State/province [16] 0 0
Taipei
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Cambridge

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Actelion
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/study/NCT00319046
Trial related presentations / publications
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.
Public notes

Contacts
Principal investigator
Name 0 0
Timothy Cox, Prof
Address 0 0
University of Cambridge
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00319046