EUCTR2005-001629-27-DE
Active, not recruiting
Not Applicable
A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- 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.
- Sponsor
- Genzyme Europe BV
- Enrollment
- 17
- Status
- Active, not recruiting
- Last Updated
- 14 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •(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
Exclusion Criteria
- •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.
Outcomes
Primary Outcomes
Not specified
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