Multicenter randomized study to assess the efficacy and the safety of two therapeutic regimens(high dose of imiglucerase versus co-administration of imiglucerase and miglustat) in type I Gaucher disease patients who have not responded to previous treatment with low dose imiglucerase - ND

• Conditions: GAUCHER disease; MedDRA version: 9.1Level: LLTClassification code 10018048Term: Gaucher's disease

• Registration Number: EUCTR2007-005516-61-IT
• Lead Sponsor: ISTITUTO GIANNINA GASLINI

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-03-23
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

confirmed diagnosis of gaucher disease with a documented deficiency of glucocerebrosidase by enzyme assay genotypic characterization minimum age 18 years
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

pregnancy evidence of medical emotional behavioural or psychological conditions which would interfere with the patient`s compliance

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: VERIFY THE EFFICACY AND THE SAFETY OF TWO DIFFERENT THERAPEUTIC REGIMENS (HIGH DOSAGE OF IMIGLUCERASE VERSUS CO-ADMINISTRATION OF IMIGLUCERASE AND MIGLUSTAT) IN TYPE i GAUCHER PATIENTS WITH ACTIVE BONE DISEASE WHICH IS NOT RESPONDING TO PREVIOUS TREATMENT WITH IMIGLUCERASE;Secondary Objective: verificare l?efficacia dell?associazione della terapia enzimatica sostitutiva con la terapia d?inibizione del substrato nella malattia di Gaucher, quale modello eventuale di terapia delle malattie lisosomiali;Primary end point(s): VERIFY THE EFFICACY AND THE SAFETY OF TWO DIFFERENT THERAPEUTIC REGIMENS (HIGH DOSAGE OF IMIGLUCERASE VERSUS CO-ADMINISTRATION OF IMIGLUCERASE AND MIGLUSTAT) IN TYPE i GAUCHER PATIENTS WITH ACTIVE BONE DISEASE WHICH IS NOT RESPONDING TO PREVIOUS TREATMENT WITH IMIGLUCERASE