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NCT Number: NCT05017402

Higher Dose of Alglucosidase Alpha for Pompe Disease

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.

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Key information

Conditions

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

Age range

0 year–60 year

Sex eligibility

All sexes

Study type

Observational

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Alglucosidase Alfa

Drug

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

Primary outcomes

  1. Forced vital capacity

    Time frame: 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)

  2. Peak expiratory flow

    Time frame: 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)

  3. Polysomnography

    Time frame: 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 outcomes

  1. uGLC4

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

    Urine glucose tetrasaccharide (Units: mmol/mol Creatinine).

  2. CK

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

    Blood creatine kinase (Units: units per liter, U/L).

  3. AST

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

    Blood aspartate aminotransferase (Units: units per liter, U/L).

  4. ALT

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

    Blood alanine aminotransferase (Units: units per liter, U/L).

  5. Body weight

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

    Change of body weight (Unit: kilogram, kg)

  6. Body height

    Time frame: The body height will be monitored before the treatment, then every two weeks, up to 2-years.

    Change of body height (Units: centimeter, cm)

  7. Antibody titers

    Time frame: First test will be one month later after first ERT, then every six months, up to 2-years.

    Alglucosidase alfa IgG antibody titer

Sponsors and collaborators

Lead sponsor

Taipei Veterans General Hospital, Taiwan

Other Gov

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2023
Study completion
2026
First posted
Aug 23, 2021
Registry last updated
Aug 23, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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