Fabry Disease Registry & Pregnancy Sub-registry
- Conditions
- Fabry Disease
- Registration Number
- NCT00196742
- Lead Sponsor
- Genzyme, a Sanofi Company
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 9000
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Fabry Registry: To evaluate the long-term safety and effectiveness of Fabrazyme® 33 years The primary purpose of this Registry is to describe the development and progression of Fabry disease in a representative global population. As Fabry is not a well-described disease this longitudinal program has a wide variety of "primary" outcomes including antibody testing as well as complication outcomes (eg, QoL, cognitive testing) which are measured over time. Additionally, as it is subject to what is collected by clinical sites at the time of visit it is unknown the amount of data that will be available for analyses.
Fabry Pregnancy Sub-registry: pregnancy outcomes, including complications and infant growth 33 years The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Fabry disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with agalsidase beta.
This Sub-registry augments the routine monitoring and data collection recommended by the Fabry Registry. The Sub-registry aims to collect patient assessments from the ante- and perinatal periods and postpartum follow-up. Neonatal assessments and periodic pediatric assessments will also be collected.
- Secondary Outcome Measures
Name Time Method Fabry Register: Monitor factors associated with the efficacy of Fabry disease treatments 33 years A secondary endpoint is to monitor those factors associated with the efficacy of Fabry disease treatments.
Trial Locations
- Locations (282)
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 Childrens 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
UCLA School Of Medicine- Site Number : 840088
🇺🇸Los Angeles, California, United States
Scroll for more (272 remaining)University of Alabama Birmingham- Nephrology- Site Number : 840018🇺🇸Birmingham, Alabama, United States