Observational Registry for Pompe Disease Patients
A Global Prospective Observational Registry of Patients With Pompe Disease
Recruiting
Observational Study
Pompe Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 500 (estimated)
- Sponsor
- Amicus Therapeutics · Industry
Who this trial is looking for
This trial is looking for patients with Pompe disease, including those with late-onset or infantile-onset types. Participants can be untreated or already receiving approved treatment, and will help us understand the long-term effects of therapies used for this condition.
Are You a Good Fit for This Trial?
You may be able to join if
- I have been diagnosed with late-onset Pompe disease.
- I have been diagnosed with infantile-onset Pompe disease.
- I have documented deficiency of GAA enzyme activity.
- I have undergone GAA genotyping.
You may not be able to join if
- I am currently in a clinical trial for Pompe disease.
- I am in a compassionate use program for Pompe disease.
- I am part of an expanded access program for Pompe disease.
Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
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 dise…
Trial Locations
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Eligibility Criteria
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 …
Contacts