Status:
UNKNOWN
Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.
Lead Sponsor:
Rambam Health Care Campus
Conditions:
Pompe Disease Infantile-Onset
Eligibility:
All Genders
5-18 years
Brief 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) an...
Detailed Description
Pompe disease is a rare autosomal recessive metabolic myopathy caused by reduced or absence activity of the lysosomal enzyme acid alpha-glucosidase (GAA). Enzyme replacement therapy (ERT) with Myozyme...
Eligibility Criteria
Inclusion
- Pompe patients \>5 years that have been on alpha-glucosidase (GAA).
Exclusion
- 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
Key Trial Info
Start Date :
June 24 2018
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
January 1 2024
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT04755751
Start Date
June 24 2018
End Date
January 1 2024
Last Update
February 16 2021
Active Locations (1)
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1
Rambam Medical Center
Haifa, Israel