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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06052800
Other study ID # OBS17349
Secondary ID U1111-1287-7177
Status Recruiting
Phase
First received
Last updated
Start date September 13, 2023
Est. completion date September 4, 2026

Study information

Verified date December 2023
Source Sanofi
Contact Trial Transparency email recommended (Toll free for US & Can
Phone 800-633-1610
Email Contact-US@sanofi.com
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial Summary

This is a national, multicenter, observational, cohort study designed to assess clinical outcomes upon agalsidase beta treatment, to characterize the clinical manifestations, and to collect the natural history on male and female Fabry disease adult patients who carry the GLA IVS4. This study aims to retrospectively and prospectively investigate the disease natural history, clinical manifestations, and the treatment outcomes upon agalsidase beta in Fabry disease (FD) patients carrying the GLA IVS4 mutation from medical records, physician assessments, and patient-reported outcomes.


Description:

Study Design Time Perspective: Retrospective and Prospective


Recruitment information / eligibility

Status Recruiting
Enrollment 100
Est. completion date September 4, 2026
Est. primary completion date September 4, 2026
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria: - Provide signed informed consent. Cohort 1: - Male or female Fabry disease patient with documented GLA IVS4 in medical record. - Age = 18 years old at the time of signing informed consent. - The maximum proportion of female is 20% of cohort 1. - Patient who has received agalsidase beta treatment for at least 6 months. - The data of LVMI, LPWT, IVST and blood lyso-Gb3 concentration are all available within 6 months prior to agalsidase beta treatment initiation. Cohort 2: - Male or female Fabry disease patient with documented GLA IVS4 in medical record. - Age = 18 years old at the time of signing informed consent. - The maximum proportion of female is 20% of cohort 2. - Patient who plans to apply for the National Health Insurance Reimbursement for agalsidase beta medication. - The data of LVMI, LPWT, IVST and blood lyso-Gb3 concentration are all available within 6 months prior to agalsidase beta treatment initiation. Cohort 3: - Male or female Fabry disease patients with documented GLA IVS4 mutation in medical record. - Male patient is aged = 30 years old and female patient is aged = 40 years old at the time of signing informed consent. - The maximum proportion of female is 20% of cohort 3. - Patient who has never received agalsidase alpha or agalsidase beta treatment (ERT-naïve). - Elevated blood lyso-Gb3. - At least ONE of the following conditions documented in medical record: 1. cardiac parameter abnormalities (e.g. via imaging, electrophysiology, or biomarker); 2. at least one FD-related sign/symptom. - The data of LVMI, LPWT, IVST and blood lyso-Gb3 concentration are all available within 6 months. - Patients who are expected not to receive ERT or FD-specific treatment per investigator's judgement. Exclusion Criteria: - Any condition that, in the opinion of the Investigator, may interfere with patient's participation in the study, such as life expectancy of less than 6 months (e.g. diagnosed with malignancy, CAD) - Fabry patients who have severe heart disease (NYHA Class IV) or severe myocardial fibrosis per investigator judgement - Known non-Fabry disease infiltrative cardiomyopathy including amyloidosis - Known non-GLA genetic (e.g., sarcomeric, metabolic mutations) hypertrophic cardiomyopathy. - Patients who are receiving any Fabry disease-specific treatment (enzyme replacement therapy, chaperone therapy, substrate reduction therapy, or gene therapy) other than agalsidase beta for Fabry disease - Pregnancy or suspected pregnancy - Patient diagnosed with moderate to severe dementia - Unstable patient condition as judged by investigator (e.g., hypertension, diabetes, and systematic disease)

Study Design


Related Conditions & MeSH terms


Locations

Country Name City State
Taiwan Investigational Site Number :1580004 Taichung
Taiwan Investigational Site Number :1580005 Tainan
Taiwan Investigational Site Number :1580001 Taipei
Taiwan Investigational Site Number :1580002 Taipei
Taiwan Investigational Site Number :1580003 Taipei

Sponsors (1)

Lead Sponsor Collaborator
Sanofi

Country where clinical trial is conducted

Taiwan, 

Outcome

Type Measure Description Time frame Safety issue
Primary Cohort 1: change of Left ventricular mass index (LVMI) as measured by echocardiography in agalsidase beta-treated patients through study completion with a minimum of 2 years
Primary Cohort 2: change of Left ventricular mass index (LVMI) as measured by echocardiography in agalsidase beta-treated patients up to 1.5 years
Secondary Change of Left posterior wall thickness (LPWT) as measured by echocardiography For cohort 1, through study completion with a minimum of 2 years For cohort 2, 1.5 years For cohort 3, through study completion with a minimum of 1.5 years
Secondary Change of Interventricular septal thickness (IVST) as measured by echocardiography For cohort 1, through study completion with a minimum of 2 years For cohort 2, 1.5 years For cohort 3, through study completion with a minimum of 1.5 years
Secondary Change of blood Globotriaosylsphingosine (lyso-Gb3) concentration For cohort 1, through study completion with a minimum of 2 years For cohort 2, 1.5 years For cohort 3, through study completion with a minimum of 1.5 years
Secondary Change of left ventricular mass index (LVMI) as measured by echocardiography in ERT-naive patients For cohort 3, through study completion with a minimum of 1.5 years
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