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Not yet recruiting NCT05017402

Higher Dose of Alglucosidase Alpha for Pompe Disease

— Observational Glycogen Storage Disease Type II

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Alglucosidase Alfa.
Who it may be relevant to
Registry conditions: Glycogen Storage Disease Type II. Basic parameters: 0 years — 60 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

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.

Interventions

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

Primary outcome measures

  • Forced vital capacity [Time frame: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.]
  • Peak expiratory flow [Time frame: For patient older than 3-year-old, first test before study, then every six months, up to 2-years.]
  • Polysomnography [Time frame: For patient older than 6-month-old, first test before study, then every six months, up to 2-years.]
Secondary outcome measures (7)
  • uGLC4 [Time frame: uGLC4 will be tested before the treatment, then every three months, up to 2-years.]
  • CK [Time frame: CK will be tested before the treatment, then every three months, up to 2-years.]
  • AST [Time frame: AST will be tested before the treatment, then every three months, up to 2-years.]
  • ALT [Time frame: ALT will be tested before the treatment, then every three months, up to 2-years.]
  • Body weight [Time frame: The body weight will be monitored before the treatment, then every two weeks, up to 2-years.]
  • Body height [Time frame: The body height will be monitored before the treatment, then every two weeks, up to 2-years.]
  • Antibody titers [Time frame: First test will be one month later after first ERT, then every six months, up to 2-years.]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT05017402 · 2021-02-019A

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗