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

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2007-005516-61-IT
• Date of registration: 23/03/2009
• Prospective Registration: No
• Primary sponsor: ISTITUTO GIANNINA GASLINI
• Public title: 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
• Scientific title: 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
• Date of first enrolment: 06/02/2009
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2007-005516-61
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: yes Placebo: no Other: no

Countries of recruitment

Italy

Key inclusion & exclusion criteria

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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

GAUCHER disease
MedDRA version: 9.1 Level: LLT Classification code 10018048 Term: Gaucher's disease

Intervention(s)

Trade Name: CEREZYME
Pharmaceutical Form: Powder and solvent for solution for infusion
INN or Proposed INN: Imiglucerase
Concentration unit: U unit(s)
Concentration type: equal
Concentration number: 200-

Trade Name: ZAVESCA
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Miglustat
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-

Primary Outcome(s)

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

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

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

Secondary ID(s)

FARM6JBLZ2