Pompe Disease RWE Long-term Monitoring Program
ConditionPompe Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorNational Taiwan University Hospital
About this trial
Evaluate short- and long-term biomarker, motor function, respiratory function and safety with avalglucosidase alfa in IOPD and LOPD patients who switched from other ERT.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
None
Disqualifiers
Having severe allergic reaction to avalglucosidase alfa.
Patient/parent/legal guardian is not agreeing to provide signed informed consent, and the patient, if <18 years of age
Trial population
Pompe Patients receiving Avalglucosidase alfa
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Urinary Glc4/Hex4
the primary endpoints for effectiveness analysis of avalglucosidase alfa. Urinary Glc4/Hex4 is usually elevated in Pompe patients and are sensitive though nonspecific biomarkers for diagnosis and monitoring of IOPD or LOPD
Time frame
3yearsSecondary outcomes
1
Creatine kinase
Serum CK is usually elevated in Pompe patients and are sensitive though nonspecific biomarkers for diagnosis and monitoring of IOPD or LOPD (Kishnani et al., 2006; Li et al., 2021; Young et al., 2009).
Time frame
3years2
motor function (Quick Motor Function Test)
QMFT score is strongly correlated with proximal muscle strength, and significantly differentiated between patients with different levels of mobility (van Capelle et al., 2012), muscle function will be assessed by QMFT annually.
Time frame
3 years3
respiratory function (forced vital capacity).
Pulmonary function will be assessed by forced vital capacity. For infants, pulmonary function tests are difficult to administer and therefore data might be limited.
Time frame
3 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov