Fabry Disease Registry & Pregnancy Sub-registry
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|ClinicalTrials.gov Identifier: NCT00196742|
Recruitment Status : Recruiting
First Posted : September 20, 2005
Last Update Posted : January 13, 2023
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.
|Condition or disease|
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||9000 participants|
|Target Follow-Up Duration:||52 Weeks|
|Official Title:||Fabry Disease Registry Protocol|
|Actual Study Start Date :||July 31, 2001|
|Estimated Primary Completion Date :||January 31, 2034|
|Estimated Study Completion Date :||January 31, 2034|
Patients with Fabry disease
No experimental intervention is given. A patient with Fabry Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.
Pregnant women with confirmed diagnosis of Fabry
No experimental intervention is given. Pregnant women with confirmed diagnosis of Fabry that are participating in the Fabry Registry and consented to participate in the Fabry Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with agalsidase beta) and irrespective of the commercial product with which she may be treated.
- Fabry Registry: To evaluate the long-term safety and effectiveness of Fabrazyme® [ Time Frame: 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 [ Time Frame: 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.
- Fabry Register: Monitor factors associated with the efficacy of Fabry disease treatments [ Time Frame: 33 years ]A secondary endpoint is to monitor those factors associated with the efficacy of Fabry disease treatments.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00196742
|Contact: Trial Transparency email recommended (Toll free number for US & Canada)||800-633-1610 ext 1 then #||Contact-Us@sanofi.com|
|Contact: Fabry Registry HelpLine||617-591-5500|
|Study Director:||Study Director||Genzyme, a Sanofi Company|