ISRCTN72578000
Recruiting
未知
Protocolised follow-up of Pompe patients receiving enzyme replacement therapy on a compassionate use basis
Erasmus Medical Centre (Netherlands)0 sites12 target enrollmentFebruary 23, 2007
Overview
- Phase
- 未知
- Intervention
- Not specified
- Conditions
- Pompe Disease
- Sponsor
- Erasmus Medical Centre (Netherlands)
- Enrollment
- 12
- Status
- Recruiting
- Last Updated
- 9 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. Confirmed diagnosis of Pompe Disease
- •2\. Infantile\-onset:
- •2\.1\. Age less than one year
- •2\.2\. Delayed motor milestones, and/or
- •2\.3\. Hypertrophic cardiomyopathy
- •3\. Late\-onset:
- •3\.1\. 24 hour/day artificial ventilation
- •3\.2\. Wheelchair bound
- •3\.3\. Previously enrolled in AGLU 1202 study
Exclusion Criteria
- •1\. Infantile\-onset:
- •1\.1\. congenital abnormalities
- •1\.2\. allergy to food and/or proteins
- •1\.3\. ventilator dependency
- •2\. Late\-onset:
- •2\.1\. developmental delays not explained by Pompe's Disease
- •2\.2\. allergies
- •2\.3\. severe co\-morbidity
Outcomes
Primary Outcomes
Not specified
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