Higher Dose of Alglucosidase Alpha for Pompe Disease

Not yet recruiting

• Conditions: Glycogen Storage Disease Type II

• Registration Number: NCT05017402
• Lead Sponsor: Taipei Veterans General Hospital, Taiwan

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2021-08
• Study First Submitted: 2021-08-05
• Study First Submitted QC: 2021-08-17
• Last Update Submitted: 2021-08-17
• Study First Posted: 2021-08-23
• Last Update Posted: 2021-08-23
• Study Start: 2021-09-01
• Primary Completion: 2023-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

1. Diagnosis of Pompe disease
2. Currently receiving ERT with regular clinic visits
3. Agree to sign informed consent

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Exclusion Criteria

1. Lost follow-up for clinical visits
2. Allergy to Myozyme
3. Refuse to sign informed consent

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

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

Secondary Outcome Measures

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