Fabry Outcome Survey (FOS) (FOS)
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ClinicalTrials.gov Identifier: NCT03289065 |
Recruitment Status :
Completed
First Posted : September 20, 2017
Last Update Posted : November 16, 2021
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Condition or disease |
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Fabry Disease |
Study Type : | Observational |
Actual Enrollment : | 4000 participants |
Observational Model: | Other |
Time Perspective: | Prospective |
Official Title: | Fabry Outcome Survey (FOS) |
Actual Study Start Date : | April 1, 2001 |
Actual Primary Completion Date : | September 30, 2021 |
Actual Study Completion Date : | September 30, 2021 |

Group/Cohort |
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FOS Participant
FOS is a disease registry open to all Fabry participants irrespective of treatment status or type of treatment.
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- 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.

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
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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.
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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.

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
United States, Massachusetts | |
Shire | |
Lexington, Massachusetts, United States, 02421 |
Study Director: | Study Director | Shire |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Shire |
ClinicalTrials.gov Identifier: | NCT03289065 |
Other Study ID Numbers: |
FOS |
First Posted: | September 20, 2017 Key Record Dates |
Last Update Posted: | November 16, 2021 |
Last Verified: | November 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement. |
Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) Informed Consent Form (ICF) Clinical Study Report (CSR) |
Access Criteria: | IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement. |
URL: | https://vivli.org/ourmember/takeda/ |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Genetic Fabry Disease Glycolipid lysosomal |
Fabry Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders |
Vascular Diseases Cardiovascular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |