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Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

NCT01451879 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 11

Last updated 2017-12-08

No results posted yet for this study

Summary

Hypothesis: the effectiveness of treatment of Pompe Disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzyme. Treatment with immunomodulatory drugs may dampen or eliminate the anti-rhGAA immune response in patients receiving ERT, thereby allowing for greater ERT efficacy. Studying the immune response to rhGAA may provide valuable insight into the role of the immune system in the effectiveness of ERT for Pompe Disease.

Conditions

  • Pompe Disease

Interventions

DRUG

Rituximab

Clinically prescribed immune modulation regimen dosage determined by local medical provider.

DRUG

Miglustat

Clinically prescribed immune modulation regimen dosage determined by local medical provider.

Sponsors & Collaborators

  • University of Florida

    lead OTHER

Principal Investigators

  • Barry J Byrne, MD, PhD · University of Florida

Eligibility

Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-10-31
Primary Completion
2017-10-31
Completion
2017-10-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

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 NCT01451879 on ClinicalTrials.gov