UnknownNot Applicable

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

Israel4 participantsStarted 2018-06-24

Plain-language 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.

Who can participate

Age range5 Years – 18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Pompe patients \>5 years that have been on alpha-glucosidase (GAA). Exclusion Criteria: * Oxygen saturation \> 90% in room air without ventilatory assistance. * Patients will be excluded if they required any invasive ventilation or if they required noninvasive ventilation while awake and upright

What they're measuring

Exercise capacity

Timeframe: 1-6 years

Trial details

NCT IDNCT04755751

SponsorRambam Health Care Campus

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2023-01-01

Contact for this trial

Lea Bentur

+972-4-7774360 l_bentur@rambam.health.gov.il

View on ClinicalTrials.gov More Pompe Disease Infantile-Onset trials