Pregnancy Outcomes for Pompe Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to gather information about pregnancy experiences and outcomes for women with Pompe disease, a rare genetic disorder affecting muscle function. The researchers focus on observing any pregnancy complications and tracking infant growth, regardless of whether the women receive specific treatments for Pompe, such as enzyme replacement therapy (ERT). Women who are pregnant and part of the Pompe registry, with the proper medical documentation, are encouraged to join to help improve understanding of how Pompe disease affects pregnancy. The trial does not test new treatments, and participants will continue their usual care with their doctors. As an unphased trial, this study offers a unique opportunity to contribute to valuable research that could enhance care for future pregnancies affected by Pompe disease.
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop taking your current medications. It focuses on tracking pregnancy outcomes and allows standard care as determined by your doctor.
Why are researchers excited about this trial?
Researchers are excited about the Pompe Pregnancy Sub-Registry because it aims to gather vital information about Pompe disease during pregnancy, a topic with limited existing data. Unlike typical treatments for Pompe disease, such as enzyme replacement therapy (ERT) using drugs like alglucosidase alfa, this registry doesn't administer a new treatment but observes and collects data. This observational approach is unique because it seeks to understand how Pompe disease affects pregnancy and vice versa, potentially influencing future treatment protocols for pregnant patients. By focusing on real-world outcomes, researchers hope to uncover insights that could lead to better care and safety guidelines for expectant mothers with Pompe disease.
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
Tracking pregnancy outcomes, including complications and infant growth, in women with Pompe disease
Follow-up
Participants are monitored for safety and effectiveness after pregnancy
What Are the Treatments Tested in This Trial?
Interventions
- Observational Study
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
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