Venglustat for Fabry Disease
(CARAT Trial)

Recruiting in Palo Alto (17 mi)

+111 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Sanofi

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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 long-term extension (LTE) to receive venglustat for up to additional 45 months with the total study duration up to 5.3 years maximum.

Eligibility Criteria

Adults aged 18-65 with Fabry disease and heart enlargement (left ventricular hypertrophy) can join this trial. They may be new or existing patients treated with specific enzyme replacement therapies or migalastat. Participants must not be pregnant, breastfeeding, or donating sperm, and should have no severe liver issues, certain infections like HIV/Hepatitis B/C, recent use of strong drugs affecting the liver's drug processing enzymes, major cardiovascular events/surgeries/kidney transplant history, seizures requiring treatment, extreme cardiac fibrosis on MRI scans unrelated to Fabry disease.

Inclusion Criteria

My heart's left ventricle is thickened.

I am currently being treated with or have not received treatment for my condition.

Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant

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Exclusion Criteria

Asymmetric hypertrophy by cardiac MRI at screening if considered by central reader to be not related to Fabry disease

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

I tested positive for COVID-19 recently or was hospitalized due to it in the last 6 months.

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Treatment Details

Interventions

Agalsidase alfa (Enzyme Replacement Therapy)
Agalsidase beta (GZ419828) (Enzyme Replacement Therapy)
Migalastat (Chaperone Therapy)
Venglustat (GZ402671) (Substrate Reduction Therapy)

Trial OverviewThe study is testing Venglustat tablets against standard treatments for Fabry disease over an 18-month period to see if they're better at reducing heart enlargement. Patients will visit the clinic every few months and might continue in a long-term extension phase for another 18 months after the initial study.

Participant Groups

2Treatment groups

Experimental Treatment

Active Control

Group I: VenglustatExperimental Treatment1 Intervention

Participants will receive venglustat once daily, orally

Group II: Standard of Care TherapyActive Control3 Interventions

Participants will receive a locally approved Fabry therapy at the standard dose and schedule (in accordance with the locally approved prescribing information)