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A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 500

Last updated 2026-03-10

No results posted yet for this study

Summary

This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate.

The objectives of the registry are:

* To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients
* To evaluate the long-term real-world effectiveness of Pompe disease treatments
* To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs)
* To describe the natural history of untreated Pompe disease

Conditions

  • Pompe Disease

Interventions

BIOLOGICAL

Cipaglucosidase alfa

Enzyme Replacement Therapy (ERT) via intravenous infusion

DRUG

Miglustat

Participants received ATB200 co-administered with AT2221 (Miglustat)

BIOLOGICAL

Alglucosidase alfa or Avalglucosidase alfa

Patients prescribed other commercially available ERT after local regulatory approval

OTHER

Untreated

Patients who are not currently receiving any medical therapy for Pompe disease.

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-16
Primary Completion
2034-12-20
Completion
2034-12-20

Countries

  • United States
  • Austria
  • Belgium
  • Denmark
  • Germany
  • Greece
  • Hungary
  • Italy
  • Netherlands
  • Poland
  • Slovenia
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06121011 on ClinicalTrials.gov