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

NCT01665326 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 400

Last updated 2026-04-08

No results posted yet for this study

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.

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).

Sponsors & Collaborators

  • Genzyme, a Sanofi Company

    collaborator INDUSTRY

  • Duke University

    lead OTHER

Principal Investigators

  • Priya S Kishnani, MD · Duke University

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-09-30
Primary Completion
2028-03-31
Completion
2029-03-31

Countries

  • United States

Study Locations

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Entities

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01665326 on ClinicalTrials.gov