Immune Tolerance Induction Study

Status Terminated

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

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

Clinical Trial Details

NCT number	NCT00701701
Study type	Interventional
Source	Sanofi
Contact
Status	Terminated
Phase	Phase 4
Start date	December 14, 2008
Completion date	February 18, 2020

View Details

See also
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