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Observational Registry for Pompe Disease Treatments

This is a global study looking at the long-term effects of different treatments for Pompe disease, including cipaglucosidase alfa, miglustat, alglucosidase alfa, and avalglucosidase alfa. It also includes patients who are not currently receiving any treatment. The study aims to understand how safe and effective these treatments are over time, and how they impact your quality of life. You can join if you have a confirmed diagnosis of late-onset (LOPD) or infantile-onset (IOPD) Pompe disease. The main goal is to evaluate the long-term safety of these treatments over five years. The current status of this study is unclear.

Study design
This is a global, multicenter, observational registry planning to enroll 500 participants with Pompe disease.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will evaluate long-term safety of treatments over five years.

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

NCT06121011

A Global Prospective Observational Registry of Patients With Pompe Disease

Recruiting
Not specifiedAll AgesObservational
Amicus Therapeutics
~500 participants
Updated 2026-03-10 on ClinicalTrials.gov
What's tested:Cipaglucosidase alfaMiglustatAlglucosidase alfa or Avalglucosidase alfaUntreated

At a glance

Recruiting sites
29 of 41 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Evaluate long-term safety of Pompe disease treatments
Measured over 5 years

Conditions

Pompe Disease

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NCT06121011

Where you'd take part

This study runs at 41 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Aarhus Universitets hospital

    Aarhus C, Denmarkno site contact published

    Recruiting

  • Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari

    Bari, Italyno site contact published

    Not yet recruiting

  • Cambridge University - Addenbrooke's Hospital

    Cambridge, United Kingdomno site contact published

    Recruiting

  • Centre of Expertise for muscular diseases and peripheral neuropathies European Reference Network for Rare Neuromuscular Diseases

    Naples, Italyno site contact published

    Not yet recruiting

  • Cincinnati Children's Hospital

    Cincinnati, Ohiono site contact published

    Recruiting

  • Department of Neurosciences Rita Levi Montalcini, University of Torino

    Torino, Italyno site contact published

    Not yet recruiting

  • Dept of Pediatrics Erasmus MC - Sophia Children's Hospital

    Rotterdam, Netherlandsno site contact published

    Not yet recruiting

  • Duke University Medical Center

    Durham, North Carolinano 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.

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Eligibility criteria

Inclusion

Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping

Exclusion

Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP)

What this trial measures

  • Evaluate long-term safety of Pompe disease treatments5 years

    Data collection that describe the frequency of AEs/SAEs occurring in Pompe disease patients