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Substrate Reduction Therapy

Venglustat for Fabry Disease (CARAT Trial)

Phase 3

Recruiting

Research Sponsored by Sanofi

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Left ventricular hypertrophy

Male and female participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease

Must not have

Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID-19 requiring hospitalization within 6 months of enrollment

Underlying medical condition that may cause or contribute to left ventricular hypertrophy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is an 18-month study to evaluate the effect of a new therapy on a heart issue in adults with Fabry Disease. Participants visit every 3-6 months and may continue in the study for up to 18 more months.

Who is the study for?

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.

What is being tested?

The 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.

What are the potential side effects?

While specific side effects are not listed here for Venglustat and other medications used in this trial (Agalsidase alfa/beta & Migalastat), common side effects could include allergic reactions to medication components, potential kidney/liver function changes due to underlying conditions related to Fabry Disease treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My heart's left ventricle is thickened.

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I am between 18 and 65 years old with a confirmed diagnosis and symptoms of Fabry disease.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I have a condition that could cause thickening of my heart muscle.

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My kidney function is reduced, with a filtration rate below 45 mL/min.

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I have hepatitis C, HIV, or hepatitis B.

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My liver is not working well.

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I have a history of liver or bile duct disease.

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I haven't taken strong or moderate drugs that affect liver enzymes in the last 14 days or more.

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My heart has severe scarring on more than half of its thickness in 3 or more areas.

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I am currently being treated for seizures.

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I have a history of heart or major cardiovascular issues, or kidney transplantation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

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)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Venglustat (GZ402671)

2023

Completed Phase 1

~30

0 / 12Eligibility criteria met