A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602
- Conditions
- Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glycogen. Pompe disease is characterized by organelle bound (lysosomal) accumulation of glycogen in many body tissues. In general, there is an inverse correlation between the amount of residual GAA activity in patients with Pompe disease and the severity of the disease.
- Registration Number
- EUCTR2005-001629-27-DE
- Lead Sponsor
- Genzyme Europe BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 17
(1) the patient’s legal guardian(s) must provide written informed consent prior to any study-related procedures being performed; (2) the patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol; and (3) the patient must have completed Protocol AGLU01602.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
A patient will be excluded from this study if he/she has experienced any unmanageable AE in Protocol AGLU01602 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation) due to Myozyme that would preclude continuing recombinant human acid a-glucosidase (rhGAA) therapy.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The overall objective is to evaluate the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease;Secondary Objective: ;Primary end point(s): The proportion of patients treated with Myozyme (20 mg/kg dose group, 40 mg/kg dose group, and both dose groups combined) who are alive and free of invasive ventilatory support after each 52-week Maintenance Phase Module and at the end of the study will be estimated using the Kaplan-Meier methodology.
- Secondary Outcome Measures
Name Time Method