Registry for Fabry Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial focuses on understanding Fabry disease, a rare genetic disorder that affects the body's ability to break down a specific type of fat. Researchers aim to learn more about the disease's progression and its impact on patients over time, regardless of treatment status. They are also examining pregnancy outcomes for women with Fabry disease to understand how the disorder might affect pregnancies and children. Individuals diagnosed with Fabry disease or pregnant women with Fabry can consider joining this trial. Participants will not receive experimental treatments but will have their health monitored by their doctors. As an unphased trial, this study seeks to gather valuable insights into Fabry disease, offering participants the opportunity to contribute to a deeper understanding of their condition.
Do I need to stop my current medications to join the trial?
The trial does not require you to stop taking your current medications. You will continue to receive care as determined by your doctor.
Why are researchers excited about this trial?
Researchers are excited about the Fabry Disease Registry and Pregnancy Sub-registry because it aims to gather comprehensive data on Fabry disease, particularly in the context of pregnancy, which is not extensively covered by current treatments. Existing options for Fabry disease, like enzyme replacement therapy or chaperone therapy, focus on managing symptoms but don't provide insights into long-term outcomes or specific challenges faced during pregnancy. This registry and sub-registry offer a unique opportunity to collect valuable information that can guide future treatments and improve patient care, with an emphasis on understanding how pregnancy affects disease progression and treatment efficacy.
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
Registry Participation
Participants undergo clinical assessments and receive care as determined by the patient's treating physician. The registry tracks routine clinical outcomes for patients with Fabry disease.
Pregnancy Sub-registry
Tracks pregnancy outcomes, including complications and infant growth, in women with Fabry disease during pregnancy. Data collection includes medical and obstetric history, pregnancy, birth, and infant growth through month 36 postpartum.
Follow-up
Participants are monitored for long-term safety and effectiveness of treatments, including Fabrazyme®. This includes monitoring factors associated with the efficacy of Fabry disease treatments.
What Are the Treatments Tested in This Trial?
Interventions
- Fabry Disease Registry
- Fabry Pregnancy Sub-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