A Global Prospective Observational Registry of Patients With Pompe Disease

Condition(s) studied

Investigational drug(s) / intervention(s)

Cipaglucosidase alfa: Enzyme Replacement Therapy (ERT) via intravenous infusion

Miglustat: Participants received ATB200 co-administered with AT2221 (Miglustat)

Alglucosidase alfa or Avalglucosidase alfa: Patients prescribed other commercially available ERT after local regulatory approval

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

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

Eligibility

Primary outcome measure(s)

• Evaluate long-term safety of Pompe disease treatments — 5 years
  Data collection that describe the frequency of AEs/SAEs occurring in Pompe disease patients

Trial sites (41)

+ 1 more sites — see the full list on the official registry below.

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