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
This trial does not accept healthy volunteersQualifiers
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 population
The observational population included patients of Pompe disease who were diagnosed and treated in our series.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Alglucosidase Alfa
DrugStandard dose: 20-25 mg/kg every other week; Higher dose: \>25 mg/kg every other week.
Treatment groups
No treatment groups listed
Trial outcomes
Primary outcomes
1
Forced vital capacity
Pulmonary function test (Units: percentage of predictive value)
Time frame
For patient older than 3-year-old, first test before study, then every six months, up to 2-years.2
Peak expiratory flow
Pulmonary function test (Units: percentage of predictive value)
Time frame
For patient older than 3-year-old, first test before study, then every six months, up to 2-years.3
Polysomnography
Comprehensive test used to diagnose sleep disorders.
Time frame
For patient older than 6-month-old, first test before study, then every six months, up to 2-years.Secondary outcomes
1
uGLC4
Urine glucose tetrasaccharide (Units: mmol/mol Creatinine).
Time frame
uGLC4 will be tested before the treatment, then every three months, up to 2-years.2
CK
Blood creatine kinase (Units: units per liter, U/L).
Time frame
CK will be tested before the treatment, then every three months, up to 2-years.3
AST
Blood aspartate aminotransferase (Units: units per liter, U/L).
Time frame
AST will be tested before the treatment, then every three months, up to 2-years.4
ALT
Blood alanine aminotransferase (Units: units per liter, U/L).
Time frame
ALT will be tested before the treatment, then every three months, up to 2-years.Locations
Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: