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Cardiopulmonary Exercise Test to Quantify Enzyme Replacement Response in Pediatric Pompe Disease

NCT03147664 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 5

Last updated 2017-05-16

No results posted yet for this study

Summary

Introduction: Enzyme replacement therapy (ERT) with Myozyme improved the prospect of Pompe disease patients. Our aim was to evaluate ERT acute effect on exercise capacity in pediatric Pompe patients.

Methods: Pompe patients (10-19 years) were evaluated before and two days after ERT using cardiopulmonary exercise testing (CPET), six minutes walking test (6MWT) and motor function test (GMFM-88).

Conditions

  • Enzyme Replacement Therapy in Pompe Disease

Sponsors & Collaborators

  • Rambam Health Care Campus

    lead OTHER

Principal Investigators

  • Lea Bentur, Prof. · Rambam Health Care Campus

Eligibility

Min Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-01-01
Primary Completion
2014-04-30
Completion
2017-03-30

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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 NCT03147664 on ClinicalTrials.gov