Fabry DiseasePhase 3CARATNCT05280548
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
A Phase 3 study testing Venglustat (GZ402671), Agalsidase alfa and Agalsidase beta (GZ419828) in people with Fabry Disease, run by Sanofi at 54 sites in 19 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Venglustat (GZ402671), Agalsidase alfa and Agalsidase beta (GZ419828)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 104 participants
- Where
- 54 sitesin 19 countries
- Study dates
- 3 May 2022to 6 Dec 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
54 sites in 19 countries.
54 sites in 19 countries
United States8 sites
Emory University School of Medicine - Atlanta- Site Number : 8400009
Atlanta, Georgia, 30322
University of Alabama -The Kirklin Clinic- Site Number : 8400010
Birmingham, Alabama, 35233
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005
Chicago, Illinois, 60611
Renal Disease Research Institute- Site Number : 8400012
Dallas, Texas, 75204
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004
Fairfax, Virginia, 22030
Maryam Banikazemi, MD- Site Number : 8400001
Hawthorne, New York, 10532
University of California Los Angeles Medical Center- Site Number : 8400008
Los Angeles, California, 90095
University of Utah Health Hospital- Site Number : 8400006
Salt Lake City, Utah, 84132
Japan7 sites
Investigational Site Number : 3920004
Fukuoka, 814-0180
Investigational Site Number : 3920007
Kagoshima, Kagoshima-ken, 890-0064
Investigational Site Number : 3920003
Kagoshima, Kagoshima-ken, 890-8520
Investigational Site Number : 3920005
Kawasaki, Kanagawa, 215-0026
Investigational Site Number : 3920006
Sapporo, Hokkaido, 060-8648
Investigational Site Number : 3920002
Sendai, Miyagi, 980-8574
Investigational Site Number : 3920001
Tokyo, 105-8461
China5 sites
Investigational Site Number : 1560002
Beijing, 100034
Investigational Site Number : 1560005
Beijing, 100034
Investigational Site Number : 1560001
Chengdu, 610041
Investigational Site Number : 1560007
Guangzhou, 510080
Investigational Site Number : 1560003
Shanghai, 200025
Canada4 sites
Investigational Site Number : 1240003
Calgary, Alberta, T2M 0L6
Investigational Site Number : 1240006
Edmonton, Alberta, T6G 2B7
Investigational Site Number : 1240005
Toronto, Ontario, M5T 3H7
Investigational Site Number : 1240002
Vancouver, British Columbia, V5Z 1M9
Germany4 sites
Investigational Site Number : 2760003
Berlin, 10117
Investigational Site Number : 2760004
Hochheim am Main, 65239
Investigational Site Number : 2760005
Mainz, 55131
Investigational Site Number : 2760001
Würzburg, 97080
Italy4 sites
Investigational Site Number : 3800004
Bologna, 40138
Investigational Site Number : 3800001
Milan, Lombardy, 20122
Investigational Site Number : 3800002
Naples, Napoli, 80131
Investigational Site Number : 3800003
Naples, Napoli, 80131
Greece3 sites
Investigational Site Number : 3000002
Athens, 115 27
Investigational Site Number : 3000003
Athens, 124 62
Investigational Site Number : 3000001
Heraklion, 711 10
Spain3 sites
Investigational Site Number : 7240003
Alicante, 03010
Investigational Site Number : 7240002
Madrid, Madrid, Comunidad de, 28007
Investigational Site Number : 7240001
Pontevedra, 36312
Turkey (Türkiye)3 sites
Investigational Site Number : 7920001
Ankara, 06560
Investigational Site Number : 7920002
Istanbul, 34098
Investigational Site Number : 7920003
İzmit, 41000
Poland2 sites
Investigational Site Number : 6160001
Krakow, 31-202
Investigational Site Number : 6160003
Lodz, Lódzkie, 02-213
South Korea2 sites
Investigational Site Number : 4100001
Seoul, Seoul-teukbyeolsi, 03722
Investigational Site Number : 4100002
Yangsan, Gyeongsangnam-do, 50612
Taiwan2 sites
Investigational Site Number : 1580003
Taichung, 407
Investigational Site Number : 1580001
Taipei, 104
Netherlands1 site
Investigational Site Number : 5280001
Amsterdam, 1105 AZ
United Kingdom1 site
Investigational Site Number : 8260001
London, London, City of, NW3 2QG
About this study
Official title: A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy
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.
Read the 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 46 months (45 months of treatment and 1 month of follow-up period). The maximum total study duration is approximately 5.3 years
- Conditions
- Fabry Disease
- Being tested
- Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828), Migalastat
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Venglustat (GZ402671)Drug
Tablet; Oral
Agalsidase alfaDrug
Also called Replagal®
Concentrate for solution for infusion; IV infusion
Agalsidase beta (GZ419828)Drug
Also called Fabrazyme®
Powder for concentrate for solution for infusion; IV infusion
MigalastatDrug
Also called Galafold®
Hard capsules; Oral
| Study group | Role | What they receive |
|---|---|---|
| Venglustat | Experimental | Participants will receive venglustat once daily, orally Receives: Drug: Venglustat (GZ402671) |
| Standard of Care Therapy | Active comparator | Participants will receive a locally approved Fabry therapy at the standard dose and schedule (in accordance with the locally approved prescribing information) Receives: Drug: Agalsidase alfa; Drug: Agalsidase beta (GZ419828); Drug: Migalastat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)
Measured: from baseline to 18 months
Secondary outcomes (9)
Slope of estimated glomerular filtration rate (eGFR) as assessed by the chronic kidney disease epidemiology collaboration (CKD-EPI) creatinine equation
Measured: from baseline to 18 months
Change in T1 relaxation time, measured by cardiac MRI (central reading)
Measured: from baseline to 18 months
Change in global longitudinal strain, measured by echocardiography (central reading)
Measured: from baseline to 18 months
Percent Change in tiredness component of FD-PRO
Measured: from baseline to 18 months
Percent Change in swelling in lower extremities component of FD-PRO
Measured: from baseline to 18 months
Number of participants with adverse event (AE) and serious adverse event (SAE)
Measured: from baseline to 18 months
Change in Beck Depression Inventory-II (BDI-II) score
Measured: from baseline to 18 months
Change in the lens clarity by ophthalmological examination
Measured: from baseline to 18 months
Plasma venglustat concentrations at prespecified visits over the study duration
Measured: from baseline to 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2022
- Study start3 May 2022
- Primary completion22 May 2026
- Study completion6 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 104 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations Clinical Sciences and Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05280548
- Sponsor's protocol ID
- EFC16158
- Other IDs
- U1111-1266-5068, 2023-509715-91, 2021-002320-20, 2023
- Acronym
- CARAT
- Status last verified
- Jun 2026
- First submitted
- 4 Mar 2022
- Last updated
- 24 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy
- ClinicalTrials.gov ID
- NCT05280548
- Acronym / sponsor ID
- CARAT / EFC16158
- Conditions
- Fabry Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 104 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 3 May 2022 (actual)
- Primary completion
- 22 May 2026 (actual)
- Study completion
- 6 Dec 2027 (estimated)
- First posted
- 15 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 46 months (45 months of treatment and 1 month of follow-up period). The maximum total study duration is approximately 5.3 years
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Venglustat | Experimental | Drug: Venglustat (GZ402671)Participants will receive venglustat once daily, orally |
| Standard of Care Therapy | Active comparator | Drug: Agalsidase alfa, Drug: Agalsidase beta (GZ419828), Drug: MigalastatParticipants will receive a locally approved Fabry therapy at the standard dose and schedule (in accordance with the locally approved prescribing information) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Venglustat (GZ402671)Tablet; Oral | Drug | Venglustat |
| Agalsidase alfaAlso: Replagal®Concentrate for solution for infusion; IV infusion | Drug | Standard of Care Therapy |
| Agalsidase beta (GZ419828)Also: Fabrazyme®Powder for concentrate for solution for infusion; IV infusion | Drug | Standard of Care Therapy |
| MigalastatAlso: Galafold®Hard capsules; Oral | Drug | Standard of Care Therapy |
Primary outcome
Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)
Time frame: from baseline to 18 months
9 secondary outcomes
Slope of estimated glomerular filtration rate (eGFR) as assessed by the chronic kidney disease epidemiology collaboration (CKD-EPI) creatinine equation
Time frame: from baseline to 18 months
Change in T1 relaxation time, measured by cardiac MRI (central reading)
Time frame: from baseline to 18 months
Change in global longitudinal strain, measured by echocardiography (central reading)
Time frame: from baseline to 18 months
Percent Change in tiredness component of FD-PRO
Time frame: from baseline to 18 months
Percent Change in swelling in lower extremities component of FD-PRO
Time frame: from baseline to 18 months
Number of participants with adverse event (AE) and serious adverse event (SAE)
Time frame: from baseline to 18 months
Change in Beck Depression Inventory-II (BDI-II) score
Time frame: from baseline to 18 months
Change in the lens clarity by ophthalmological examination
Time frame: from baseline to 18 months
Plasma venglustat concentrations at prespecified visits over the study duration
Time frame: from baseline to 18 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2026.
- EFC16158 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations Clinical Sciences and Operations study director
Sanofi
54 sites in 19 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Venglustat (GZ402671), Agalsidase alfa and Agalsidase beta (GZ419828) in people with Fabry Disease, run by Sanofi at 54 sites in 19 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 24 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 54 sites in United States, Japan, China, Canada, Germany and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading) (time frame: from baseline to 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2026.