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

Status: Recruiting
Location: See location...
Intervention Type: Other
Study Type: Observational
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 18
Healthy Volunteers: No
View:

• Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease

• Must have a parent or guardian provide written informed consent

Locations
United States
North Carolina
Duke University Medical Center
Recruiting
Durham
Contact Information
Primary
Ankit K Desai, MBBS
ankit.desai@duke.edu
919-613-6310
Backup
Eleanor Rodriguez-Rassi, MPH
eleanor.rodriguezrassi@duke.edu
919-613-1219
Time Frame
Start Date: September 2009
Estimated Completion Date: September 2024
Participants
Target number of participants: 300
Treatments
Infantile Pompe disease
Individuals with a confirmed diagnosis of Infantile Pompe disease
Related Therapeutic Areas
Sponsors
Collaborators: Genzyme, a Sanofi Company
Leads: Duke University

This content was sourced from clinicaltrials.gov

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