ClinicalTrials.gov record NCT06666413

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD

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

A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 4A study of a treatment that is already approved, to learn more about its long-term effects, safety or use in everyday practice.
AgeUp to 17 years old
SexAll
LocationChina
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.

A single-arm, open-label Phase 4 study giving avalglucosidase alfa by IV infusion every other week for 52 weeks (total study ≈64 weeks). It is for children under 18 with infantile-onset Pompe disease (symptoms starting before 12 months) with cardiomyopathy; both treatment-naïve children and those previously treated with alglucosidase alfa or avalglucosidase alfa may be eligible. People who previously received other enzyme-replacement therapies (ERTs) or any other Pompe disease treatment (including gene therapy) are excluded; other major medical conditions or congenital abnormalities may also exclude children.

What it is studying. The study is collecting safety information (adverse events, serious events, infusion reactions, lab tests, vital signs, ECGs) during treatment. It is also measuring clinical outcomes at Week 52 such as survival without invasive ventilation, heart muscle size (left ventricular mass Z score), growth (body growth Z scores), walking distance and motor scores where applicable, and motor development milestones.

What the researchers hope to learn. To evaluate the safety profile of avalglucosidase alfa in children with infantile-onset Pompe disease and to collect data on clinical outcomes (survival/ventilation status, cardiac measures, growth, motor function, and milestone achievement) over 52 weeks.

What this study is about

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

This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT. Study details include:

  • The study duration: total study duration is approximately 64 weeks.
  • Screening period of up to 8 weeks
  • Treatment period of 52 weeks
  • Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks)
  • The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.

Interventions being studied

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

Drug
  • Avalglucosidase alfa

Eligibility

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

Inclusion criteria

  • Participant must be <18 years of age, at the time of signing the informed consent.
  • Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.
  • Participants must have documented cardiomyopathy at the time of diagnosis.
  • Contraceptive use should be consistent with local regulations Participant's parent/legally authorized representative (LAR) must be capable of giving signed informed consent.

Exclusion criteria

  • Participants are excluded from the study if any of the following criteria apply:
  • Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
  • Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
  • Participants who have received an ERT other than alglucosidase alfa or avalglucosidase alfa, or any other treatment for Pompe disease, including gene therapy prior to the enrollment.
  • Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as IMP Participants who are anticipated to take prohibited therapy (ie, any other treatment for Pompe disease) during this study.
  • Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug before enrollment, whichever is longer, or are anticipated to take any other concurrent investigational treatments.
  • Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
  • The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

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.

After screening (up to 8 weeks) participants receive avalglucosidase alfa by intravenous infusion at 20 mg/kg every other week for a 52-week treatment period, followed by a short follow-up (typically 4 weeks; may be reduced to 2 weeks if the participant starts another study or commercial ERT). The study involves about 30 visits (29 site visits and one phone follow-up) with safety monitoring (adverse event reporting, lab tests, vital signs, ECG) and efficacy assessments such as heart ultrasound (echocardiography), growth measurements, motor tests (e.g., six‑minute walk test, quick motor function test where applicable) and a motor milestone checklist.

How the study is structured. Single-group, open-label Phase 4 treatment study. Participants receive avalglucosidase alfa 20 mg/kg by IV infusion every other week for 52 weeks with about 30 study contacts (29 site visits and one phone call). There is up to an 8-week screening period and a short follow-up after the last dose.

Questions you could ask

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

  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • 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
May 7, 2025
Primary completion
May 2, 2028 (estimated)
Study completion
May 2, 2028 (estimated)
Target enrollment
13 participants (estimated)
Last updated on registry
Mar 23, 2026
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
Genzyme, a Sanofi Company

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • Investigational Site Number: 1560001Recruiting

    Shanghai, 200127 · China

Terms used on this page · explanations by MedicalUpdates.ai

single group
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
treatment-naïve
Has not previously received the treatment, or the type of treatment, that the study is about.
Screening period
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
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.
legally authorized representative
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
prohibited therapy
A medicine that participants may not use during the study, usually because it could interfere with the study treatment or its measurement.
investigational
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
half-lives
The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
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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