Fabry Outcome Survey (FOS) (FOS)

• ClinicalTrials.gov Identifier: NCT03289065
• Recruitment Status: Recruiting
• First Posted: September 20, 2017
• Last Update Posted: March 8, 2021
• Sponsor: Shire
• Information provided by (Responsible Party): Takeda ( Shire )

Study Description

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

• 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

Groups and Cohorts

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

Outcome Measures

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

• 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

Contacts

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

Locations

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

• Study Director: Study Director; Shire

More Information

Additional Information:

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

Feriozzi S, Linhart A, Ramaswami U, Kalampoki V, Gurevich A, Hughes D; Fabry Outcome Survey Study Group. Effects of Baseline Left Ventricular Hypertrophy and Decreased Renal Function on Cardiovascular and Renal Outcomes in Patients with Fabry Disease Treated with Agalsidase Alfa: A Fabry Outcome Survey Study. Clin Ther. 2020 Dec;42(12):2321-2330.e0. doi: 10.1016/j.clinthera.2020.10.007. Epub 2020 Nov 17.

Parini R, Pintos-Morell G, Hennermann JB, Hsu TR, Karabul N, Kalampoki V, Gurevich A, Ramaswami U; FOS Study Group. Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age. Drug Des Devel Ther. 2020 Jun 3;14:2149-2158. doi: 10.2147/DDDT.S249433. eCollection 2020.

Ramaswami U, Beck M, Hughes D, Kampmann C, Botha J, Pintos-Morell G, West ML, Niu DM, Nicholls K, Giugliani R; FOS Study Group. Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis. Drug Des Devel Ther. 2019 Oct 25;13:3705-3715. doi: 10.2147/DDDT.S207856. eCollection 2019.

• Responsible Party: Shire
• ClinicalTrials.gov Identifier: NCT03289065
• Other Study ID Numbers: FOS
• First Posted: September 20, 2017
• Last Update Posted: March 8, 2021
• Last Verified: March 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

Keywords provided by Takeda ( Shire ):

Genetic; Fabry Disease; Glycolipid; lysosomal

Additional relevant MeSH terms:

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