Higher Dose of Alglucosidase Alpha for Pompe Disease

NCT05017402 | Not Yet Recruiting

• 1 other identifier: 2021-02-019A
• Study Type: observational
• Target: 36
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Glycogen Storage Disease Type II

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (7)

• uGLC4

  uGLC4 will be tested before the treatment, then every three months, up to 2-years.

• CK

  CK will be tested before the treatment, then every three months, up to 2-years.

• AST

  AST will be tested before the treatment, then every three months, up to 2-years.

• ALT

  ALT will be tested before the treatment, then every three months, up to 2-years.

• Body weight

  The body weight will be monitored before the treatment, then every two weeks, up to 2-years.

Interventions

Alglucosidase Alfa DRUG

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

Eligibility Criteria

• Age: 0 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

The observational population included patients of Pompe disease who were diagnosed and treated in our series.

You may qualify if:

• Diagnosis of Pompe disease
• Currently receiving ERT with regular clinic visits
• Agree to sign informed consent

You may not qualify if:

• Lost follow-up for clinical visits
• Allergy to Myozyme
• Refuse to sign informed consent

Sponsors & Collaborators

• Taipei Veterans General Hospital, Taiwan: lead

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Interventions

GAA protein, human

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2021
• First Posted: August 23, 2021
• Study Start: September 1, 2021
• Primary Completion: December 31, 2023
• Study Completion (Estimated): December 31, 2026
• Last Updated: August 23, 2021

Record last verified: 2021-08