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Purpose

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.

Condition

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Venglustat 		Participants will receive venglustat once daily, orally
  • Drug: Venglustat (GZ402671)
    Tablet; Oral
Active Comparator
Standard of Care Therapy 		Participants will receive a locally approved Fabry therapy at the standard dose and schedule (in accordance with the locally approved prescribing information)
  • Drug: Agalsidase alfa
    Concentrate for solution for infusion; IV infusion
    Other names:
    • Replagal®
  • Drug: Agalsidase beta (GZ419828)
    Powder for concentrate for solution for infusion; IV infusion
    Other names:
    • Fabrazyme®
  • Drug: Migalastat
    Hard capsules; Oral
    Other names:
    • Galafold®

Recruiting Locations

University of Alabama at Birmingham (UAB) - The Kirklin Clin Site Number : 8400010
Birmingham, Alabama 35233-2110

More Details

Status
Recruiting
Sponsor
Sanofi

Study Contact

Trial Transparency email recommended (Toll free for US & Canada)
800-633-1610
Contact-US@sanofi.com

Detailed 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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.