Registry for Pompe Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to understand more about Pompe disease, a rare genetic disorder affecting muscle function. By collecting information from individuals with Pompe disease, both those receiving treatment and those who are not, researchers hope to learn about disease progression and the long-term effects of treatments like alglucosidase alfa. This information will help improve patient care and develop better treatment guidelines. Anyone with a confirmed diagnosis of Pompe disease, verified through specific tests, can join this trial. As a Phase 4 trial, this research focuses on understanding how the already FDA-approved treatment benefits more patients, offering valuable insights for participants.
Do I need to stop my current medications to join the trial?
The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the trial coordinators or your doctor.
Why are researchers excited about this trial?
Researchers are excited about the Pompe Disease Registry Protocol because it aims to collect comprehensive data on patients with Pompe disease, which can lead to a deeper understanding of the condition. Unlike treatments that focus solely on managing symptoms, this registry offers a broader perspective by tracking the natural progression of the disease in a real-world setting. The information gathered can help identify patterns, improve current treatments, and possibly lead to the development of new therapies that are more effective and tailored to patient needs.
Who Is on the Research Team?
Study Director
Principal Investigator
Genzyme, a Sanofi Company
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observational
Participants are observed to track the natural history and outcomes of Pompe disease
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Observational Registry
Find a Clinic Near You
Who Is Running the Clinical Trial?
Genzyme, a Sanofi Company
Lead Sponsor
David Meeker
Genzyme, a Sanofi Company
Chief Executive Officer since 2011
MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School
Jean-Paul Kress
Genzyme, a Sanofi Company
Chief Medical Officer since 2015
MD from Faculte Necker-Enfants Malades, Paris