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
Completed · Phase 2
Conditions studied: Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
In brief
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
Key facts
- Study ID
- NCT00763932
- Run by
- Genzyme, a Sanofi Company
- People needed
- 7
- Starts
- 2003-04-01
- Expected to finish
- 2006-07-01
- Last updated by the study team
- 2014-02-05
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
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.
You may not qualify if…
- 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
Where it is running
- 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
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.