Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

February 4, 2014 updated by: Genzyme, a Sanofi Company

A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies

This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

7

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Lyon, France
        • Pediatrique Hospital Debrousse
      • Orleans, France
        • Hopital Porte Madeleine
  • South Africa
      • Johannesburg, South Africa
        • The Morningside Clinic
  • United States
    • California
      • Oakland, California, United States
        • Children's Hospital of Oakland
      • Orange, California, United States
        • Children's Hospital of Orange Country
    • Nebraska
      • Omaha, Nebraska, United States
        • University of Nebraska Medical Center
    • New Jersey
      • New Brunswick, New Jersey, United States
        • Institute for Genetic Medicine, Saint Peter's University Hospital
    • New York
      • New York, New York, United States
        • New York University [NYU] School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502
  • The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed
  • The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.

Exclusion Criteria:

  • Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: 1
Biological: Myozyme
10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow
Other Names:
  • Alglucosidase alfa

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The objective of this extension study was to monitor the long-term safety and efficacy
Time Frame: 3 years
3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Genzyme, a Sanofi Company

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2003

Primary Completion (ACTUAL)

June 1, 2006

Study Completion (ACTUAL)

July 1, 2006

Study Registration Dates

First Submitted

September 30, 2008

First Submitted That Met QC Criteria

September 30, 2008

First Posted (ESTIMATE)

October 1, 2008

Study Record Updates

Last Update Posted (ESTIMATE)

February 5, 2014

Last Update Submitted That Met QC Criteria

February 4, 2014

Last Verified

February 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AGLU02003

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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