Extension Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

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

A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602

Pompe disease (also known as glycogen storage disease type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

16

Phase

  • Phase 2
  • Phase 3

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
      • Amiens, France, 80054
        • CHU Amiens
      • Caen, France, 14033
        • CHU côte de Nacre
  • Germany
      • Mainz, Germany, 55131
        • Universitats-Kinderklinik Mainz
  • Israel
      • Haifa, Israel, 35254
        • Rambam Medical Center
  • Italy
      • Monza, Italy, 20052
        • San Gerardo Hospital
  • Netherlands
      • Rotterdam, Netherlands, 3015 GJ
        • Erasmus MC University
  • Taiwan
      • Hua-lien, Taiwan, 970
        • Tzu-Chi General Hospital
      • Tainan, Taiwan, 710
        • Chi-Mei Medical Center Dept of Pediatrics
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama
    • Florida
      • Gainesville, Florida, United States, 32610
        • Shands Hospital at the University of Florida
      • Miami, Florida, United States, 33155
        • Miami Children's Hospital
    • Georgia
      • Decatur, Georgia, United States, 30033
        • Emory University Medical Genetics
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Children's Hospital Medical Center

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • The patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed
  • The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol
  • The patient must have completed Protocol AGLU01602.

Exclusion Criteria:

  • Patient has experienced any unmanageable adverse event (AE) in Protocol AGLU01602 due to Myozyme that would preclude continuing treatment with Myozyme

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: Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 1
Biological: Myozyme
20 mg/kg qow or 40 mg/kg qow
Other Names:
  • alglucosidase alfa

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Long-term Safety and Efficacy
Time Frame: 52 weeks
52 weeks

Collaborators and Investigators

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

Sponsor

Genzyme, a Sanofi Company

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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

June 1, 2005

Primary Completion (Actual)

June 1, 2006

Study Completion (Actual)

December 1, 2006

Study Registration Dates

First Submitted

August 1, 2005

First Submitted That Met QC Criteria

August 1, 2005

First Posted (Estimate)

August 2, 2005

Study Record Updates

Last Update Posted (Estimate)

February 6, 2014

Last Update Submitted That Met QC Criteria

February 4, 2014

Last Verified

February 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AGLU02403

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

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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