A Global Prospective Observational Registry of Patients With Pompe Disease

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Amicus Therapeutics
Study ID
NCT06121011
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Cipaglucosidase alfa — BIOLOGICAL
    Enzyme Replacement Therapy (ERT) via intravenous infusion
  • Miglustat — DRUG
    Participants received ATB200 co-administered with AT2221 (Miglustat)
  • Alglucosidase alfa or Avalglucosidase alfa — BIOLOGICAL
    Patients prescribed other commercially available ERT after local regulatory approval
  • Untreated — OTHER
    Patients who are not currently receiving any medical therapy for Pompe disease.

Study Details

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

Key Dates

Start date
Feb 16, 2024
Status verified
Mar 2026
Primary completion
Dec 20, 2034
Completion
Dec 20, 2034

Study Design

Enrollment
500 participants (estimated)

Arms

  • Arm: Cipaglucosidase alfa/Miglustat-treated patients
  • Arm: Other Enyzme Replacement Therapy (ERT)-treated patients
  • Arm: Untreated patients (those who are not currently receiving any medical therapy for Pompe disease)

Primary Outcome Measure

Evaluate long-term safety of Pompe disease treatments [ Time Frame: 5 years ]

Central Contacts

Locations (15)

FacilityCityStateZIPSite coordinators
University of Arkansas Medical ScienceLittle RockArkansas72205-
University of California IrvineIrvineCalifornia92697-
Wolfson Children's HospitalJacksonvilleFlorida32207-
Emory UniversityAtlantaGeorgia30322-
Indiana University, IU Health Physicians NeurologyIndianapolisIndiana46202-
Washington University School of MedicineSt LouisMissouri63110-
NYU Neurogenetics, NYU Langone Medical CenterNew YorkNew York10017-
Duke University Medical CenterDurhamNorth Carolina27710-
Cincinnati Children's HospitalCincinnatiOhio45229-
University of Cincinnati Medical CenterCincinnatiOhio45219-
University of Pennsylvania Perelman Center for Advanced MedicinePhiladelphiaPennsylvania19104-
University of PittsburghPittsburghPennsylvania15219-
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15224-
University of UtahSalt Lake CityUtah84108-
Lysosomal and Rare Disorders Research and Treatment Center, Inc.FairfaxVirginia22030-

Find similar trials in Little Rock, AR

By research site
University of Arkansas Medical Science· Little Rock, ARUniversity of California Irvine· Irvine, CAWolfson Children's Hospital· Jacksonville, FLEmory University· Atlanta, GAIndiana University, IU Health Physicians Neurology· Indianapolis, INWashington University School of Medicine· St Louis, MO

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