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

  • STATUS
    Recruiting
  • End date
    Jul 15, 2027
  • participants needed
    90
  • sponsor
    Sanofi
Send
Updated on 30 May 2024
See if I qualify
hypertrophy
ventricular hypertrophy
agalsidase beta
agalsidase alfa
migalastat

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 long-term extension (LTE) to receive venglustat for up to additional 34 months with the total study duration up to 4.4 years maximum.

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 long-term extension period)

Long-term extension period: the total duration will be from minimum 19 months (18 months of treatment and 1 month of follow-up period) to maximum 35 months (34 months of treatment and 1 month of follow-up period). The maximum total study duration is approximately 4.4 years

Details
Condition Fabry Disease
Age 18years - 65years
Treatment agalsidase alfa, venglustat (GZ402671), Agalsidase beta (GZ419828), Migalastat, Migalastat
Clinical Study IdentifierNCT05280548
SponsorSanofi
Last Modified on30 May 2024

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 if considered by central reader to be not related to Fabry disease
Advanced cardiac fibrosis, defined as significant late gadolinium enhancement affecting 3 or more segments involving >50% of myocardial thickness on screening cardiac MRI
History of clinically significant cardiac arrhythmia. Atrial fibrillation that is well controlled on a stable medical regimen for at least 12 months is not an exclusion if the CHA2DS2-VASc score is 0 for males or 1 for females
Estimated glomerular filtration rate <45 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 within 14 days or 5 half-lives, 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
Clear my responses
Read more

How to participate?

Step 1 Connect with a study center
We have submitted the contact information you provided to the research team at {{SITE_NAME}}. A copy of the message has been sent to your email for your records.
US Only: Would you like to be notified about other trials? Sign up for Patient Notification Services.
Sign up

Send a message

Enter your contact details to connect with study team

Investigator Avatar

Primary Contact

First name*
Last name*
Email*
Phone number*
Other language

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

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.

Learn more

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

Learn more

Similar trials to consider

Browse trials for

hypertrophy
ventricular hypertrophy
agalsidase beta
agalsidase alfa
migalastat

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer
Add a private note
  • abc Select a piece of text.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.