Ignite Creation Date:
2024-05-06 @ 3:47 PM
Last Modification Date:
2024-10-26 @ 1:57 PM
Study NCT ID:
NCT04755751
Status:
UNKNOWN
Last Update Posted:
2021-02-16
First Post:
2021-02-10
Brief Title:
Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease
Sponsor:
Rambam Health Care Campus
Organization:
Rambam Health Care Campus
Study Overview
Official Title:
Acute and Long Term Evaluation of Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease
Status:
UNKNOWN
Status Verified Date:
2021-02
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 and assessing the relationship between enzyme blood level and exercise capacity Such evaluation may allow a more objective quantification of the response to ERT
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 has significantly improved the prospect of patients with infantile Pompe disease IPD Most IPD patients show clinical improvement on therapy but deteriorate at different time points raising the possibility that increasing drug delivery might halt the progression of the disease Data on the effects of ERT therapy on physiological variables related to exercise tolerance is scarce
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
Methods A retrospective - prospective study evaluating pediatric patients with Pompe before and 2 days after ERT on multiple occasions and different dosing Evaluation included cardiopulmonary exercise testing CPET 6 minute-walking test 6MWT motor function test GMFM-88 and self-collected blood samples on a Guthrie card for enzyme blood levels
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
Methods A retrospective - prospective study evaluating pediatric patients with Pompe before and 2 days after ERT on multiple occasions and different dosing Evaluation included cardiopulmonary exercise testing CPET 6 minute-walking test 6MWT motor function test GMFM-88 and self-collected blood samples on a Guthrie card for enzyme blood levels
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None