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Study of Cipaglucosidase Alfa/Miglustat for Infantile-Onset Pompe Disease

This study is testing a combination treatment, cipaglucosidase alfa and miglustat, for children aged 0 to under 18 with Infantile-Onset Pompe Disease (IOPD), a genetic condition affecting muscle and organ function. Researchers want to understand how safe and effective this treatment is, how the body handles it, and if it causes an immune response. The study is looking for children who have already received enzyme replacement therapy (ERT) for at least six months and were diagnosed with hypertrophic cardiomyopathy (a heart muscle condition). The main goal is to see how many participants experience infusion-related reactions over 104 weeks. The current recruitment status is unclear.

Study design
This is a Phase 3, open-label study, meaning both you and the study team will know which treatment is being given. It plans to enroll 36 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for infusion-associated reactions for 104 weeks.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04808505

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Recruiting
PHASE3Up to 17InterventionalTreatment
Amicus Therapeutics
~36 participants
Updated 2026-01-22 on ClinicalTrials.gov
What's tested:Cipaglucosidase alfaMiglustat

At a glance

Recruiting sites
13 of 14 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Proportion of subjects with infusion-associated reactions (IARs)
Measured over 104 weeks

Conditions

Glycogen Storage Disease Type II Infantile Onset

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Where you'd take part

This study runs at 14 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • AOU Federico II

    Naples, Italyno site contact published

    Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohiono site contact published

    Recruiting

  • Duke University Early Phase Research Unit

    Durham, North Carolinano site contact published

    Recruiting

  • Erasmus MC, Sophia Kinderziekenhuis

    Rotterdam, Netherlandsno site contact published

    Recruiting

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, United Kingdomno site contact published

    Recruiting

  • SphinCS GmbH

    Höchheim, Germanyno site contact published

    Recruiting

  • The Emory Clinic

    Atlanta, Georgiano site contact published

    Recruiting

  • Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie

    Giessen, Germanyno site contact published

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

For Site
[email protected]
609-662-2000
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

What this trial measures

  • Proportion of subjects with infusion-associated reactions (IARs)104 weeks