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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline to 18 months
Awards & highlights
CARAT 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.Check my eligibility
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.See study design
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.
CARAT Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My heart's left ventricle is thickened.
Select...
I am between 18 and 65 years old with a confirmed diagnosis and symptoms of Fabry disease.
CARAT Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ from baseline to 18 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline to 18 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)
Secondary outcome measures
Change in Beck Depression Inventory-II (BDI-II) score
Change in T1 relaxation time, measured by cardiac MRI (central reading)
Change in global longitudinal strain, measured by echocardiography (central reading)
+6 moreCARAT 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
Find a Location
Who is running the clinical trial?
SanofiLead Sponsor
2,164 Previous Clinical Trials
3,514,890 Total Patients Enrolled
13 Trials studying Fabry Disease
2,169 Patients Enrolled for Fabry Disease
Genzyme, a Sanofi CompanyLead Sponsor
524 Previous Clinical Trials
85,539 Total Patients Enrolled
25 Trials studying Fabry Disease
14,685 Patients Enrolled for Fabry Disease
Clinical Sciences & Operations Clinical Sciences and OperationsStudy DirectorSanofi
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My heart's left ventricle is thickened.I am currently being treated with or have not received treatment for my condition.I tested positive for COVID-19 recently or was hospitalized due to it in the last 6 months.I have a condition that could cause thickening of my heart muscle.My kidney function is reduced, with a filtration rate below 45 mL/min.I have a heart rhythm problem but it's been stable for over a year.I have hepatitis C, HIV, or hepatitis B.My liver is not working well.I have a history of liver or bile duct disease.I am between 18 and 65 years old with a confirmed diagnosis and symptoms of Fabry disease.I haven't taken strong or moderate drugs that affect liver enzymes in the last 14 days or more.My heart has severe scarring on more than half of its thickness in 3 or more areas.I am currently being treated for seizures.I have a history of heart or major cardiovascular issues, or kidney transplantation.
Research Study Groups:
This trial has the following groups:- Group 1: Venglustat
- Group 2: Standard of Care Therapy
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What threats to patient health has Venglustat been shown to present?
"Our team at Power has evaluated the safety of Venglustat to be a 3, due to evidence-based data in Phase 3 trials that both demonstrate its efficacy and validate its security."
Answered by AI
Am I eligible to join this clinical experiment?
"People with fabry disease between 18 and 65 years old are eligible to apply for this clinical trial. Approximately 90 patients will be accepted."
Answered by AI
Are individuals of any age admitted to this investigation?
"The parameters of this medical trial dictate that the youngest possible enrollee is 18 years old and the oldest can be no older than 65."
Answered by AI
Are there a myriad of facilities conducting this research in the local area?
"According to the information available, 35 medical centres are participating in this study. These include Lysosomal and Rare Disorders Research and Treatment Center, Inc-Site Number:8400004 in Fairfax, Investigational Site Number :0400001 in Graz, and Investigational Site Number :2080001 as well as other sites across the world."
Answered by AI
Has this research endeavor opened up enrollment for participants?
"This investigation is currently accepting participants, as stated on clinicaltrials.gov. It has been open since the 3rd of May 2022 and was last updated on the 11th August 2023."
Answered by AI
What is the maximum enrolment capacity of this clinical trial?
"Genzyme, a Sanofi Company, is sponsoring this trial and require 90 eligible participants to begin. The study will be conducted at the Lysosomal and Rare Disorders Research and Treatment Center in Fairfax Virginia as well as an Investigational Site Number :0400001 located in Graz Madrid Comunidad De."
Answered by AI
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