Higher Dose of Alglucosidase Alpha for Pompe Disease

N/ANot Yet RecruitingNCT05017402

Taipei Veterans General Hospital, Taiwan36 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Glycogen Storage Disease Type II

Interventions

Alglucosidase AlfaDRUG

Standard dose: 20-25 mg/kg every other week; Higher dose: \>25 mg/kg every other week.

Eligibility

Sex: ALLMin age: 0 YearsMax age: 60 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Diagnosis of Pompe disease 2. Currently receiving ERT with regular clinic visits 3. Agree to sign informed consent Exclusion Criteria: 1. Lost follow-up for clinical visits 2. Allergy to Myozyme 3. Refuse to sign informed consent

Outcomes

Primary Outcomes

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.

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.

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

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.

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.

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.

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.

Body weight

Data from ClinicalTrials.gov

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