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Immune Tolerance Induction Study

NCT00701701 · Status: TERMINATED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2022-04-07

Study results available
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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

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.

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-12-14
Primary Completion
2020-02-18
Completion
2020-02-18

Countries

  • United States
  • Israel

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00701701 on ClinicalTrials.gov