Fabry Disease Registry & Pregnancy Sub-registry

Fabry Disease Clinical Trial

Official title: Fabry Disease Registry Protocol

Status Recruiting

Clinical Trial Summary

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.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Fabry Disease

• NCT number: NCT00196742
• Study type: Observational [Patient Registry]
• Source: Sanofi
• Contact: Trial Transparency
• Phone: 800-633-1610
• Email: Contact-Us@sanofi.com
• Status: Recruiting
• Start date: July 31, 2001
• Completion date: January 31, 2034