A Global Prospective Observational Registry of Patients With Pompe Disease

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Amicus Therapeutics

Status

Enrolling

Conditions

Pompe Disease

Treatments

Other: Untreated
Biological: Alglucosidase alfa or Avalglucosidase alfa
Biological: Cipaglucosidase alfa
Drug: Miglustat

Study type

Observational

Funder types

Industry

Identifiers

NCT06121011
POM-005

Details and patient eligibility

About

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

Enrollment

500 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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

Trial design

500 participants in 3 patient groups

Cipaglucosidase alfa/Miglustat-treated patients
Treatment:
Drug: Miglustat
Biological: Cipaglucosidase alfa
Other Enyzme Replacement Therapy (ERT)-treated patients
Treatment:
Biological: Alglucosidase alfa or Avalglucosidase alfa
Untreated patients (those who are not currently receiving any medical therapy for Pompe disease)
Treatment:
Other: Untreated

Trial contacts and locations

8

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Central trial contact

For Site; For Patient

Data sourced from clinicaltrials.gov

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