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 range

5 Years – 18 Years

Sex

ALL

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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