Who may be eligible for the Higher Dose of Alglucosidase Alpha for Pompe Disease trial?

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

ClinicalTrials.gov record NCT05017402

Higher Dose of Alglucosidase Alpha for Pompe Disease

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

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

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.The registry record was last updated on Aug 23, 2021, about 5 years ago, so the status shown may be out of date. The study team or the registry can confirm.

Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.

AgeUnder 1 month old to 60 years old

SexAll

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational cohort study follows people with Pompe disease who are receiving enzyme replacement therapy (alglucosidase alfa) at standard or higher doses. It collects safety and clinical outcome data over up to two years, including lung function, sleep studies, urine and blood biomarkers, growth measures, and antibody testing. The study aims to investigate whether higher-dose ERT affects safety and clinical outcomes and to inform dosing recommendations.

What it is studying. The study is collecting long-term clinical and safety data from people with Pompe disease treated with alglucosidase alfa at standard (20–25 mg/kg every other week) or higher (>25 mg/kg every other week) doses. Key areas of data are lung function and sleep testing, urine and blood biomarkers, growth measurements, and anti‑drug antibody levels.

What the researchers hope to learn. To gather long-term safety and clinical outcome data on higher-dose alglucosidase alfa in Pompe disease and to inform future dosing recommendations and understanding of long-term outcomes.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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 being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: Alglucosidase Alfa

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational study follows participants for up to 2 years with repeated tests and samples. Expect breathing tests (forced vital capacity and peak expiratory flow) at baseline and then every six months for those older than 3 years; sleep studies (overnight polysomnography) at baseline and every six months for those older than 6 months. Regular blood and urine tests (including uGLC4, CK, AST, ALT and antibody titers) will be collected—some every three months and some every six months—and body weight and height will be recorded about every two weeks. Participation requires providing informed consent and attending the scheduled clinic visits for these measurements.

How the study is structured. An observational cohort study where participants continue their usual enzyme replacement therapy and clinic care while study staff collect medical tests and records for up to two years. The study does not assign or change treatment. Planned monitoring includes periodic pulmonary function tests and polysomnography, regular urine and blood tests, frequent weight and height checks, and antibody testing according to the schedule in the registry.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
The registry says recruitment has not started. When is it expected to open, and can I register interest now?
No sites are listed on the registry yet. Where will the study take place?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on Pompe disease

Clinical trials32 trials listed for Pompe diseaseEvery trial the registry lists for Pompe disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Pompe disease researchLatest monthly update: September 2026. Includes: Antibody effects. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Sep 1, 2021 (estimated)
Primary completion: Dec 31, 2023 (estimated)
Study completion: Dec 31, 2026 (estimated)
Target enrollment: 36 participants (estimated)
Last updated on registry: Aug 23, 2021
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Taipei Veterans General Hospital, Taiwan

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins.

Terms used on this page · explanations by MedicalUpdates.ai

informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
follow-up: The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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