CompletedPhase 2

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

United States7 participantsStarted 2003-04

Plain-language 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

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

The objective of this extension study was to monitor the long-term safety and efficacy

Timeframe: 3 years

Trial details

NCT IDNCT00763932

SponsorGenzyme, a Sanofi Company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2006-06

View on ClinicalTrials.gov More Pompe Disease Infantile-Onset trials