Higher Dose of Alglucosidase Alpha for Pompe Disease
ConditionGlycogen Storage Disease Type II
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-60
SponsorTaipei Veterans General Hospital, Taiwan
About this trial
This study is aimed to investigate that whether the higher dose ERT improve safety and clinical outcomes of Pompe disease patients. Also, wish to develop a new therapeutic recommendation and hope that it could improve the long-term outcomes of Pompe diesease patients.
Eligibility criteria
Qualifiers
Diagnosis of Pompe disease
Currently receiving ERT with regular clinic visits
Agree to sign informed consent
Disqualifiers
Lost follow-up for clinical visits
Allergy to Myozyme
Refuse to sign informed consent
Trial design
Treatments tested in this trial
- Alglucosidase Alfa
Treatment groups
No treatment groups listed
Locations
Sponsors and collaborators
Source ClinicalTrials.gov