A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (CARAT)

  • STATUS
    Recruiting
  • End date
    Sep 15, 2026
  • participants needed
    90
  • sponsor
    Genzyme, a Sanofi Company
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Updated on 15 July 2022
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hypertrophy
ventricular hypertrophy
migalastat
agalsidase beta
agalsidase alfa

Summary

This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.

  • Study visits will take place approximately every 3 to 6 months
  • Participants who complete the randomized period may continue to the open-label extension (OLE) to receive venglustat for up to an additional 18 months.

Description

Randomized period: the total duration will be up to approximately of 20 months (1 month screening 18 months of treatment and a possible follow-up period of 1 month if no participation in the open label extension period)

Open-label extension period: the total duration will be approximately 19 months (18 months of treatment and 1 month of follow-up period)

Details
Condition Fabry's Disease
Treatment agalsidase alfa, agalsidase beta (GZ419828), migalastat, venglustat (GZ402671)
Clinical Study IdentifierNCT05280548
SponsorGenzyme, a Sanofi Company
Last Modified on15 July 2022

Eligibility

 Yes No Not Sure

Inclusion Criteria

Male and female participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease
Participants may be receiving treatment with agalsidase alfa, agalsidase beta, or migalastat, or may be untreated
Left ventricular hypertrophy
Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant
A signed informed consent must be provided prior to any study-related procedures

Exclusion Criteria

History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery or kidney transplantation
History of seizures currently requiring treatment
Underlying medical condition that may cause or contribute to left ventricular hypertrophy
Asymmetric hypertrophy by cardiac MRI at screening
Advanced cardiac fibrosis, defined as significant late gadolinium enhancement beyond the basal inferolateral left ventricular wall on cardiac MRI
History of ongoing clinically significant cardiac arrythmia, prior or ongoing treatment for the above
Estimated glomerular filtration rate <60 mL/min/1.73m2
Presence of severe depression as measured by Beck's Depression Inventory (BDI)-II >28 and/or a history of an untreated, unstable major affective disorder within 1 year of the screening visit
Patients with hepatitis C, HIV, or hepatitis B infection
Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID-19 requiring hospitalization within 6 months of enrollment
History of drug and/or alcohol abuse
Moderate to severe hepatic impairment
History of or active hepatobiliary disease
Liver enzymes (alanine aminotransferase/aspartate aminotransferase) or total bilirubin >2 times the upper limit of normal
Strong or moderate inducers or inhibitors of cytochrome P450 CYP3A4 per Food and Drug Administration (FDA) classification within 14 days or 5 halflives, whichever is longer, prior to randomization
Known contraindication to undergoing MRI or known hypersensitivity to gadolinium-based contrast agents
The above information is not intended to contain all considerations relevant to a potential
participation in a clinical trial
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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