NCT01665326View on ClinicalTrials.gov

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

RecruitingOBSERVATIONAL
Enrollment

400

Participants

Timeline

Start Date

September 30, 2009

Primary Completion Date

March 31, 2028

Study Completion Date

March 31, 2029

Conditions
Pompe Disease
Interventions
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).

Trial Locations (1)

27710

RECRUITING

Duke University Medical Center, Durham

Sponsors

Lead Sponsor

Duke University
All Listed Sponsors
collaborator

Genzyme, a Sanofi Company

INDUSTRY

lead

Duke University

OTHER