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

N/A
Recruiting
Research Sponsored by Amicus Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 5 years
Awards & highlights

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

Eligible Conditions
  • Pompe Disease

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Evaluate long-term safety of Pompe disease treatments

Trial Design

3Treatment groups
Experimental Treatment
Group I: Untreated patients (those who are not currently receiving any medical therapy for Pompe disease)Experimental Treatment1 Intervention
Group II: Other Enyzme Replacement Therapy (ERT)-treated patientsExperimental Treatment1 Intervention
Group III: Cipaglucosidase alfa/Miglustat-treated patientsExperimental Treatment2 Interventions
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Miglustat
2020
Completed Phase 4
~210

Find a Location

Who is running the clinical trial?

Amicus TherapeuticsLead Sponsor
54 Previous Clinical Trials
2,224 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
~333 spots leftby Dec 2034