Fabry Disease Registry & Pregnancy Sub-registry

What this study is about

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary objectives of the Registry are: * To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; * To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; * To characterize and describe the Fabry population as a whole; * To evaluate the long-term safety and effectiveness of Fabrazyme® Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician. If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.

Who can join (things the study team will check)

✅ You may be able to join if…

• Fabry Registry: All patients with a confirmed diagnosis of Fabry disease who have signed the informed consent and patient authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte αGAL (alpha-galactosidase) enzyme activity and/or mutation(s) in the gene coding for αGAL.
• Fabry Pregnancy Sub-registry:
  • Eligible women must:
  • be enrolled in the Fabry Registry.
  • be pregnant, or have been pregnant with appropriate medical documentation available.
  • provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.

Where this trial is running

• University of Alabama Birmingham- Nephrology- Site Number : 840018, Birmingham, Alabama, United States
• University of Alabama Birmingham- Nephrology- Site Number : 840073, Birmingham, Alabama, United States
• Phoenix Children's Hospital- Site Number : 840003, Phoenix, Arizona, United States
• University of Arizona- Site Number : 840015, Tucson, Arizona, United States
• Arkansas Children's Hospital- Site Number : 840109, Little Rock, Arkansas, United States
• University of Arkansas for Medical Sciences- Site Number : 840113, Little Rock, Arkansas, United States
• University of California at Irvine- Site Number : 840036, Irvine, California, United States
• Southern California Permanente Medical Group- Site Number : 840108, Los Angeles, California, United States
• USC Health Sciences Center Dept of Genetics- Site Number : 840082, Los Angeles, California, United States
• University Of California Los Angeles- Site Number : 840088, Los Angeles, California, United States
• UCSF Benioff Children's Hospital - Oakland- Site Number : 840029, Oakland, California, United States
• Children's Hospital of Orange County- Site Number : 840074, Orange, California, United States

+ 272 more sites.

Who to contact

Trial Transparency · 800-633-1610 · contact-us@sanofi.com

It's completely normal to call and ask questions before deciding anything. Mention the study ID: NCT00196742.