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Fabry Outcome Survey (FOS) (FOS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03289065
Recruitment Status : Recruiting
First Posted : September 20, 2017
Last Update Posted : May 21, 2020
Sponsor:
Information provided by (Responsible Party):
Shire

Study Description
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Brief 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.

Condition or disease
Fabry Disease

Detailed Description:

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 Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 4000 participants
Observational Model: Other
Time Perspective: Prospective
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

Resource links provided by the National Library of Medicine


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


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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).

  4. 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.

  5. 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)

  6. 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.

  7. 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.


Eligibility Criteria
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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
Study Population
The FOS registry is open to participants with Fabry disease irrespective of their treatment. Participants may be untreated, currently treated or have been previously treated with Replagal, or any other approved treatment for Fabry disease. There is no predetermined sample size.
Criteria

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.

Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
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United States, Massachusetts
Shire Recruiting
Lexington, Massachusetts, United States, 02421
Contact: Central Contact       ClinicalTransparency@shire.com   
Principal Investigator: Shire PI         
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03289065    
Other Study ID Numbers: FOS
First Posted: September 20, 2017    Key Record Dates
Last Update Posted: May 21, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided 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 in the Data Sharing section of the www.shiretrials.com website. 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://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Shire:
Genetic
Fabry Disease
Glycolipid
lysosomal
Additional relevant MeSH terms:
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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