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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

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

Status
Active, not recruiting
Phases
Unknown
Study type
Interventional
Source
EU CTR
Registry ID
EUCTR2007-005516-61-IT
Enrollment
Unknown
Registered
2009-03-23
Start date
2009-02-06
Completion date
Unknown
Last updated
2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

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: 2

Sponsors

ISTITUTO GIANNINA GASLINI
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion 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

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

Design outcomes

Primary

MeasureTime frame
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—

Countries

Italy

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026