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A Global Prospective Observational Registry of Patients With Pompe Disease

Join a global study to learn more about Pompe Disease.

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Phase N/A

Pompe Disease is a rare condition that affects muscles and organs. This study looks at people with two types of Pompe disease: Late-onset Pompe disease (LOPD) and Infantile-onset Pompe disease (IOPD). The study is observational, which means researchers will watch and learn without changing any treatments. Both people who are getting treatment and those who are not can join. The goal is to see how safe and effective treatments are and how they affect patients' lives over time.

Who can join? People with a confirmed Pompe disease diagnosis. However, if you are in another clinical trial or special program for new treatments, you cannot join.

  • This study is global, meaning it involves people from all over the world.
  • It is an observational registry, so you don’t change your current treatment.
  • There are no direct medical risks, only regular follow-ups.

If you have Pompe disease and want to help researchers understand it better, this study might be for you. You will be helping doctors learn more about how the disease and its treatments affect people over time.

Study details
    Pompe Disease

NCT06121011

Amicus Therapeutics

1 March 2025

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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FAQs

Learn more about clinical trials

What is a clinical trial?

A clinical trial is a study designed to test specific interventions or treatments' effectiveness and safety, paving the way for new, innovative healthcare solutions.

Why should I take part in a clinical trial?

Participating in a clinical trial provides early access to potentially effective treatments and directly contributes to the healthcare advancements that benefit us all.

How long does a clinical trial take place?

The duration of clinical trials varies. Some trials last weeks, some years, depending on the phase and intention of the trial.

Do I get compensated for taking part in clinical trials?

Compensation varies per trial. Some offer payment or reimbursement for time and travel, while others may not.

How safe are clinical trials?

Clinical trials follow strict ethical guidelines and protocols to safeguard participants' health. They are closely monitored and safety reviewed regularly.
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