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
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
1 other identifier
interventional
7
3 countries
8
Brief Summary
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Apr 2003
Typical duration for phase_2
8 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2003
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2006
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2006
CompletedFirst Submitted
Initial submission to the registry
September 30, 2008
CompletedFirst Posted
Study publicly available on registry
October 1, 2008
CompletedFebruary 5, 2014
February 1, 2014
3.2 years
September 30, 2008
February 4, 2014
Conditions
Outcome Measures
Primary Outcomes (1)
The objective of this extension study was to monitor the long-term safety and efficacy
3 years
Study Arms (1)
1
EXPERIMENTALInterventions
10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow
Eligibility Criteria
You may qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (8)
Children's Hospital of Oakland
Oakland, California, United States
Children's Hospital of Orange Country
Orange, California, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Institute for Genetic Medicine, Saint Peter's University Hospital
New Brunswick, New Jersey, United States
New York University [NYU] School of Medicine
New York, New York, United States
Pediatrique Hospital Debrousse
Lyon, France
Hôpital Porte Madeleine
Orléans, France
The Morningside Clinic
Johannesburg, South Africa
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Monitor
Genzyme, a Sanofi Company
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
September 30, 2008
First Posted
October 1, 2008
Study Start
April 1, 2003
Primary Completion
June 1, 2006
Study Completion
July 1, 2006
Last Updated
February 5, 2014
Record last verified: 2014-02