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NCT05280548 (venglustat in Fabry disease): BVCT ex-ante prediction vs. reported outcome

On 18 Apr 2025, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 30 Jul 2026 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially insufficient.

Clinical Trial

NCTID
NCT05280548
Acronym
CARAT
Drug Name
venglustat
Drug MoA
glucosylceramide synthase inhibitor
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Fabry disease
Human Patients
104
Sponsor
Sanofi
Phase
Phase 3

Prediction of , index 1984

Prospective Prediction
Prediction Index
1984
Prediction Date
Commercial Prediction
FAILURE (Commercially insufficient clinical benefit in eGFR/FD-PRO/left ventricular mass index inferior to pegunigalsidase alfa, agalsidase alfa or migalastat)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak clinical benefit in eGFR/FD-PRO/left ventricular mass index compared with placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
468 days in advance
Readout Data Interpretation
venglustat did not meet primary endpoint failing to demonstrate superior-to-SoC clinical benefit
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

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
Phase
Phase 3
Status
Active not recruiting
Enrollment
104
Lead sponsor
Sanofi
Conditions
Fabry Disease
Interventions
DRUG: Venglustat (GZ402671); DRUG: Agalsidase alfa; DRUG: Agalsidase beta (GZ419828); DRUG: Migalastat
Design
Randomized · Parallel · None
Primary purpose
Treatment
Start
2022-05-03
Primary completion
2026-05-22
Completion
2027-12-06
Primary outcomes
  • Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading) (from baseline to 18 months)

NCT05280548 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials