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Pompe Disease Registry Protocol

Join our Pompe Disease Registry for future research insights and advancements.

Recruiting
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Phase N/A

Pompe Disease is a rare illness that affects muscles and movement. This study is a Registry which means it collects information from patients over time. It’s international, meaning it includes people from all over the world. Patients with Pompe disease can join to help doctors learn more about how the disease changes and how well treatments work.

The study looks at both past and future data, which is called retrospective and prospective. To join, you must have a confirmed diagnosis of Pompe disease. This means tests show your body doesn't have enough of a certain enzyme, GAA, or there are changes in your GAA genes. There are no other restrictions to join.

  • Participation is voluntary, which means you can choose to join or leave at any time.
  • There is no specific treatment given in the study, just tracking of your health data.
  • There is no financial compensation for participating, but your involvement can help improve future care for Pompe patients.
Study details
    Glycogen Storage Disease Type II
    Pompe Disease

NCT00231400

Genzyme, a Sanofi Company

13 December 2024

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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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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