Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Recruiting
Observational Study
Pompe Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 400 (estimated)
- Sponsor
- Duke University · Other
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About This Trial
This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme/Myozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have developed high and sustained antibodies …
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease
* Must provide a written informed consent
Contacts