NCT00701701

Immune Tolerance Induction Study

An Exploratory Study of the Safety and Efficacy of Immune Tolerance Induction (ITI) in Patients With Pompe Disease Who Have Previously Received Myozyme

• Status: Terminated
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 1 Month
• Healthy volunteers: Not accepted

Summary

An exploratory, open-labeled study of participants with Pompe disease, who had previously received Myozyme® (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme®. Eligible participants who were then receiving Myozyme® therapy were enrolled into the study, and were followed for a minimum of 18 months on-study (a 6-month ITI treatment module and a 12-month follow-up module on Myozyme® alone). Eligible participants were followed for a minimum of 18 months on treatment or, if a participant was \<6 months of age at the time of enrollment, until the participant was 2 years of age. Both cross-reacting immunologic material (CRIM)-negative and CRIM-positive participants were eligible for Regimen A depending if they met the required criteria. Regimen B, however, was limited to CRIM-negative participants.

Conditions

• Pompe Disease
• Glycogen Storage Disease Type II (GSD-II)
• Glycogenesis 2 Acid Maltase Deficiency

Interventions

Type	Name	Description
BIOLOGICAL	Myozyme® (alglucosidase alfa)	Myozyme®: IV infusion of 20 mg/kg qow; Cyclophosphamide: 250 mg/m\^2 IV q4w after Myozyme infusion for 6 months.
BIOLOGICAL	Myozyme® (alglucosidase alfa)	Myozyme®: IV infusion of 20 mg/kg qow; Rituximab: 375 mg/m\^2 IV weekly beginning the day after Myozyme infusion for 4 weeks (an optional additional 2nd cycle could be administered at the discretion of the investigator); Methotrexate: 15 mg/m\^2 subcutaneous qow on the day after Myozyme infusion for 6 months.

Timeline

• Start date: 2008-12-14
• Primary completion: 2020-02-18
• Completion: 2020-02-18
• First posted: 2008-06-19
• Last updated: 2022-04-07
• Results posted: 2020-10-12

Locations

5 sites across 2 countries: United States, Israel