A Global Prospective Observational Registry of Patients With Pompe Disease
ConditionPompe Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorAmicus Therapeutics
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping
Disqualifiers
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 population
The study population in this registry will consist of patients with a diagnosis of Pompe disease (LOPD or IOPD) based on documented deficiency of acid α-glucosidase (GAA) enzyme activity and/or GAA genotyping, regardless of time since diagnosis.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Cipaglucosidase alfa
Biological/VaccineEnzyme Replacement Therapy (ERT) via intravenous infusion
Miglustat
DrugParticipants received ATB200 co-administered with AT2221 (Miglustat)
Alglucosidase alfa or Avalglucosidase alfa
Biological/VaccinePatients prescribed other commercially available ERT after local regulatory approval
Untreated
Other interventionPatients who are not currently receiving any medical therapy for Pompe disease.
Treatment groups
500 Participants
are divided into 3 treatment groupsGroup A: Cipaglucosidase alfa/Miglustat-treated patients2 interventions
Group B: Other Enyzme Replacement Therapy (ERT)-treated patients1 intervention
Group C: Untreated patients (those who are not currently receiving any medical therapy for Pompe disease)1 intervention
Trial outcomes
Primary outcomes
1
Evaluate long-term safety of Pompe disease treatments
Data collection that describe the frequency of AEs/SAEs occurring in Pompe disease patients
Time frame
5 yearsSponsors and contacts
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Source ClinicalTrials.gov