Fabry Outcome Survey (FOS) (FOS)
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|ClinicalTrials.gov Identifier: NCT03289065|
Recruitment Status : Recruiting
First Posted : September 20, 2017
Last Update Posted : March 8, 2021
|Condition or disease|
19 MAY 2020: The temporary enrollment stop of new patients into this study due to the COVID-10 pandemic has been lifted in one or more countries, and the study is now again enrolling new patients. However, some countries/sites may still have paused the enrollment of new patients due to the pandemic.
24 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||4000 participants|
|Target Follow-Up Duration:||25 Years|
|Official Title:||Fabry Outcome Survey (FOS)|
|Actual Study Start Date :||April 1, 2001|
|Estimated Primary Completion Date :||April 1, 2030|
|Estimated Study Completion Date :||April 1, 2030|
FOS is a disease registry open to all Fabry participants irrespective of treatment status or type of treatment.
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline to year 20 ]An adverse event (AE) is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. An AE or ADR that meets one or more of the following criteria/outcomes is classified as SAE whether considered to be related to the pharmaceutical product or not: death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalizations, a persistent or significant disability or incapacity, a congenital anomaly or birth defect and important medical events.
- Number of Participants With Infusion-related Reactions (IRRs) [ Time Frame: Baseline to year 20 ]An infusion-related reaction (IRR) is defined as an AE that has been assessed as at least possibly related to treatment with Replagal and occurs during an infusion or up to 24 hours post Replagal infusion.
- Renal Function by Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Baseline to year 20 ]Renal function will be measured by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for adults and by Counahan-Barratt for children (<18 years).
- Left Ventricular Mass Index (LVMI) [ Time Frame: Baseline to year 20 ]Left ventricular mass index (LVMI) will be assessed from baseline or from birth (age at event) to evaluate the course of Fabry disease in participants who are currently untreated or are being treated with an approved Fabry treatment.
- Age at Mortality Event (survival) [ Time Frame: Baseline to year 20 ]Age at mortality event (survival) will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in Fabry Outcome Survey (FOS)
- Time to First Morbidity Event [ Time Frame: Baseline to year 20 ]Time to first morbidity event will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in FOS.
- Age at First Morbidity Event [ Time Frame: Baseline to year 20 ]Age at first morbidity event will be analysed with relevant split by demographic or baseline characteristic using Kaplan-Meier survival estimates with censoring at last visit in FOS.
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): NCT03289065
|Contact: Shire Contact||+1 866 842 5335||ClinicalTransparency@shire.com|
|United States, Massachusetts|
|Lexington, Massachusetts, United States, 02421|
|Contact: Central Contact ClinicalTransparency@shire.com|
|Principal Investigator: Shire PI|
|Study Director:||Study Director||Shire|