Skip to main content
TrialFinder
TrialFinder is for informational purposes only and does not provide medical advice. Always talk to your doctor about whether a trial is right for you.
RECRUITINGOBSERVATIONAL

Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease

Determination of Cross-Reactive Immunological Material (CRIM) Status and Longitudinal Follow-up of Individuals With Pompe Disease

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

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 to ERT. To follow the long-term outcomes, we will collect medical records including but not limited to the diagnosis, clinical parameters, assessments for clinical monitoring, and laboratory values including antibody testing results.

Who May Be Eligible (Plain English)

Who May Qualify: - Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease - Must provide a written willing to sign a consent form Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease * Must provide a written informed consent

Treatments Being Tested

OTHER

Observational

This is a longitudinal study focused on the emerging natural history of Infantile Pompe disease, response to ERT using alglucosidase alfa (Myozyme) and response to Immune Tolerance Induction (ITI).

Locations (1)

Duke University Medical Center
Durham, North Carolina, United States