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NCT05206773 (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 29 Jan 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
NCT05206773
Acronym
PERIDOT
Drug Name
Venglustat (GZ402671)
Drug MoA
glucosylceramide synthase inhibitor
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Fabry Disease
Human Patients
122
Sponsor
Sanofi
Phase
Phase 3

Prediction of , index 1983

Prospective Prediction
Prediction Index
1983
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in FD-PRO inferior to pegunigalsidase alfa)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak clinical benefit in FD-PRO compared with placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
286 days in advance
Readout Data Interpretation
venglustat failed to meet the primary endpoint of FD-PRO compared with placebo
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, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months
Phase
Phase 3
Status
Active not recruiting
Enrollment
122
Lead sponsor
Sanofi
Conditions
Fabry Disease
Interventions
DRUG: Venglustat (GZ402671); DRUG: Placebo
Design
Randomized · Parallel · Double
Primary purpose
Treatment
Start
2022-03-11
Primary completion
2025-12-17
Completion
2027-01-04
Primary outcomes
  • Percent change from baseline at 6 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain) (From baseline to 6 months)
  • Percent change from baseline at 12 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain) (From baseline to 12 months)

NCT05206773 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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