A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

PHASE2CompletedINTERVENTIONAL
Enrollment

26

Participants

Timeline

Start Date

June 30, 2006

Primary Completion Date

August 31, 2009

Study Completion Date

December 31, 2015

Conditions
Gaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency DiseaseGlucosylceramide Beta-Glucosidase Deficiency DiseaseGaucher Disease, Non-Neuronopathic Form
Interventions
DRUG

Eliglustat tartrate

Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg twice daily (BID) from Day 2 to Day 19, then either eliglustat 50 mg BID (if Genz-99067\[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\]5 nanogram per milliliter \[ng/mL\] on Day 10) or eliglustat 100 mg BID(if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing same treatment through study completion (Year 9). Participant receiving 100 mg BID could be considered for further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).

Trial Locations (17)

Unknown

New York University, New York

New York

Aprillus Asistencia e Investigación, Buenos Aires

Hospital de Oncologia Maria Curie, Buenos Aires

Hospital Ramos Mejia, Buenos Aires

IMAI, Buenos Aires

Instituto Argentino de Diagnostico y Tratamiento (IADT), Buenos Aires

Buenos Aires

Rambam Medical Center, Haifa

Haifa

Sha'are Zedek Medical Centre, Jerusalem

Jerusalem

Universita degli Studi di Milano, Milan

Instituto Mexicano del Seguro Social, D.f.

Mexico City

Hematology Research Center of Ministry of Healthcare of the Russian Federation, Moscow

Moscow

All Listed Sponsors
lead

Genzyme, a Sanofi Company

INDUSTRY

NCT00358150 - A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients | Biotech Hunter | Biotech Hunter