Higher Dose of Alglucosidase Alpha for Pompe Disease

Study Overview
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Brief Summary
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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.

Official Title
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Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study

Conditions
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Glycogen Storage Disease Type II

Intervention / Treatment
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* Drug: Alglucosidase Alfa


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of Pompe disease Currently receiving ERT with regular clinic visits Agree to sign informed consent Exclusion Criteria: Lost follow-up for clinical visits Allergy to Myozyme Refuse to sign informed consent

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Alglucosidase Alfa|Standard dose: 20-25 mg/kg every other week; Higher dose: >25 mg/kg every other week.|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Forced vital capacity | Pulmonary function test (Units: percentage of predictive value) | For patient older than 3-year-old, first test before study, then every six months, up to 2-years. | 
| Peak expiratory flow | Pulmonary function test (Units: percentage of predictive value) | For patient older than 3-year-old, first test before study, then every six months, up to 2-years. | 
| Polysomnography | Comprehensive test used to diagnose sleep disorders. | For patient older than 6-month-old, first test before study, then every six months, up to 2-years. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| uGLC4 | Urine glucose tetrasaccharide (Units: mmol/mol Creatinine). | uGLC4 will be tested before the treatment, then every three months, up to 2-years. | 
| CK | Blood creatine kinase (Units: units per liter, U/L). | CK will be tested before the treatment, then every three months, up to 2-years. | 
| AST | Blood aspartate aminotransferase (Units: units per liter, U/L). | AST will be tested before the treatment, then every three months, up to 2-years. | 
| ALT | Blood alanine aminotransferase (Units: units per liter, U/L). | ALT will be tested before the treatment, then every three months, up to 2-years. | 
| Body weight | Change of body weight (Unit: kilogram, kg) | The body weight will be monitored before the treatment, then every two weeks, up to 2-years. | 
| Body height | Change of body height (Units: centimeter, cm) | The body height will be monitored before the treatment, then every two weeks, up to 2-years. | 
| Antibody titers | Alglucosidase alfa IgG antibody titer | First test will be one month later after first ERT, then every six months, up to 2-years. |