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A Global Prospective Observational Registry of Patients With Pompe Disease

Sponsor
Amicus Therapeutics (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT06121011
Collaborator
(none)
500
Enrollment
2
Locations
120
Anticipated Duration (Months)
250
Patients Per Site
2.1
Patients Per Site Per Month

Study Details

Study Description

Brief 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

Condition or Disease Intervention/Treatment Phase
  • Biological: Cipaglucosidase alfa
  • Drug: Miglustat
  • Biological: Alglucosidase alfa or Avalglucosidase alfa
  • Other: Untreated

Study Design

Study Type:
Observational [Patient Registry]
Anticipated Enrollment :
500 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
A Global Prospective Observational Registry of Patients With Pompe Disease
Anticipated Study Start Date :
Nov 20, 2023
Anticipated Primary Completion Date :
Nov 20, 2033
Anticipated Study Completion Date :
Nov 20, 2033

Arms and Interventions

Arm Intervention/Treatment
Cipaglucosidase alfa/Miglustat-treated patients

Biological: Cipaglucosidase alfa
Enzyme Replacement Therapy (ERT) via intravenous infusion
Other Names:
  • ATB200, Pombiliti

    • Drug: Miglustat
    Participants received ATB200 co-administered with AT2221 (Miglustat)
    Other Names:
  • AT2221, Opfolda

    		•
    Other Enyzme Replacement Therapy (ERT)-treated patients

    Biological: Alglucosidase alfa or Avalglucosidase alfa
    Patients prescribed other commercially available ERT after local regulatory approval
    Other Names:
  • Myozyme, Lumizyme, Nexviazyme, Nexviadyme

    		•
    Untreated patients (those who are not currently receiving any medical therapy for Pompe disease)

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

    Outcome Measures

    Primary Outcome Measures

    1. Long-term safety [5 years]

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

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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)

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 NYU Langone Medical Center New York New York United States 10017
    2 Lysosomal and Rare Disorders Research and Treatment Center, Inc. Fairfax Virginia United States 22030

    Sponsors and Collaborators

    • Amicus Therapeutics

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Amicus Therapeutics
    ClinicalTrials.gov Identifier:
    NCT06121011
    Other Study ID Numbers:
    • POM-005
    First Posted:
    Nov 7, 2023
    Last Update Posted:
    Nov 7, 2023
    Last Verified:
    Nov 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Product Manufactured in and Exported from the U.S.:
    No
    Additional relevant MeSH terms:
    Glycogen Storage Disease Type II
    Miglustat

    Study Results

    No Results Posted as of Nov 7, 2023