Fabry Outcome Survey (FOS)

Fabry Disease Clinical Trial

— FOS  

Official title:

Fabry Outcome Survey (FOS)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03289065
• Other study ID #: FOS
• Status: Completed
• Start date: April 1, 2001
• Est. completion date: September 30, 2021

Study information

• Verified date: November 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The purpose of this study is to collect data that will increase understanding of Fabry disease history and progression, in treated and untreated patients with Fabry disease. The data from FOS may provide guidance to healthcare professionals about disease treatment options.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4000
• Est. completion date: September 30, 2021
• Est. primary completion date: September 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Participants must have a documented diagnosis of Fabry disease

• This may include a genetic mutation analysis. The collection of the genetic mutation analysis result is optional and dependent on the participant providing their consent for this data to be used in the FOS registry.

• Participants can be untreated, currently or previously treated with Replagal, or any other approved treatment for Fabry disease.

2. Signed and dated written informed consent from the participant

• For participants aged less than (<) 18 years (or as per local regulation), parent and/or participant's legally authorized representative (LAR), and assent of the minor, where applicable, is necessary.

• If a participant is unable to read or if a legally acceptable representative is unable to read, an impartial witness should be present during the informed consent discussion and should sign and personally date the informed consent.

• Informed consent must be obtained from LARs for cognitively impaired participants when applicable.

Exclusion Criteria:

1. Participants currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials) will be excluded from the Registry.

Related Conditions & MeSH terms

• Fabry Disease

Locations

Country	Name	City	State
United States	Shire	Lexington	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	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.	Baseline to year 20
Primary	Number of Participants With Infusion-related Reactions (IRRs)	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.	Baseline to year 20
Primary	Renal Function by Estimated Glomerular Filtration Rate (eGFR)	Renal function will be measured by Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) for adults and by Counahan-Barratt for children (<18 years).	Baseline to year 20
Primary	Left Ventricular Mass Index (LVMI)	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.	Baseline to year 20
Primary	Age at Mortality Event (survival)	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)	Baseline to year 20
Primary	Time to First Morbidity Event	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.	Baseline to year 20
Primary	Age at First Morbidity Event	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.	Baseline to year 20