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Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.

NCT04755751 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 4

Last updated 2021-02-16

No results posted yet for this study

Summary

Our aims are to investigate the acute and long term effect of ERT on exercise capacity; comparing the effect of different ERT dosages (as prescribed by the clinician according to clinical judgment) and assessing the relationship between enzyme blood level and exercise capacity. Such evaluation may allow a more objective quantification of the response to ERT.

Conditions

  • Pompe Disease Infantile-Onset

Interventions

DIAGNOSTIC_TEST

Cardiopulmonary exercise testing (CPET)

CPET using a cycle ergometer (COSMED, Rome, Italy) beginning with a no resistance warm up lasting 1 to 3 minutes and followed with an incrementing resistance adapted to the patient's functional capacities according to the examiner's free judgment and ranging from no resistance (20 Rounds Per Minute (RPM)/minute) 5 to 20 Watts/minute on ramps or by successive stages

Sponsors & Collaborators

  • Rambam Health Care Campus

    lead OTHER

Principal Investigators

  • Lea Bentur · Rambam

Eligibility

Min Age
5 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-06-24
Primary Completion
2023-01-01
Completion
2024-01-01

Countries

  • Israel

Study Locations

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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 NCT04755751 on ClinicalTrials.gov