To Confirm Efficacy & Safety of Genz−112638 in Gaucher Type 1 Patients
Research type
Research Study
Full title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher Disease Type 1
IRAS ID
23439
Contact name
Timothy Cox
Eudract number
2008-005222-37
Clinicaltrials.gov Identifier
Research summary
A Phase III, Randomised, Double-Blind, Placebo controlled study of up to 2.5 years duration, looking at the safety and efficacy of Genz-112638 (an oral substrate inhibitor) in Gaucher's Disease Type 1 patients who have either never received previous treatment, or haven't received treatment for 6-12 months (dependant of type of previous treatment). After an initial 39 week placebo controlled, double blind period, the study drug (Genz-112638) will then be offered on an open label basis for all study participants up to a maximum of 2.5 years. At the end of the study, the sponsor agrees to ensure access to the study medicine (Genz112638) for study participants, as long as the clinical study outcomes indicate that the medicine is save and brings effective benefits to the clinical status of the patients, in accordance with the evaluation made by the physician of the study (investigator). This will be subjected to applicable laws and regulations. Primary Efficacy Analyses will be determined by percentage change in spleen volume. Secondary Efficacy Analyses will be determined by absolute change in haemoglobin levels, percentage change in liver volume and percentage change in platelet counts. Tertiary and Exploratory Efficacy Analyses will be determined by Biomarkers, Bone Disease assessments, Gaucher??s assessments and Quality of Life (QoL) questionnaires. Safety will be measured by adverse event collection, vital signs, laboratory and ECG assessment. PK analyses will be performed on all patients who receive at least 1 dose of Genz-112638.
REC name
East of England - Cambridge South Research Ethics Committee
REC reference
09/H0305/69
Date of REC Opinion
6 Nov 2009
REC opinion
Further Information Favourable Opinion