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NCT05737784 (Elsunersen in SCN2A-DEE Epilepsy): BVCT ex-ante prediction vs. reported outcome

On 5 Jan 2024, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. The 6 Apr 2026 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially sufficient.

Clinical Trial

NCTID
NCT05737784
Acronym
EMBRAVE
Drug Name
Elsunersen
Drug MoA
anti-SCN2A
Drug Modality
antisense oligonucleotide (ASO)
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
SCN2A-DEE Epilepsy
Human Patients
60
Sponsor
Praxis Precision Medicines
Phase
Phase 1 Phase 2

Prediction of , index 1245

Prospective Prediction
Prediction Index
1245
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient clinical benefit for SCN2A-mutant patients)
Technical Prediction
SUCCESS (statistically significant clinical benefit for SCN2A-mutant patients)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
822 days in advance
Readout Data Interpretation
Elsunersen achieved 77% placebo-adjusted reduction in monthly seizures and disease modifying improvements (p=0.015)
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially sufficient
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Seamless, Clinical Trial to Investigate the Safety and Efficacy of Multiple Doses of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
60
Lead sponsor
Praxis Precision Medicines
Conditions
SCN2A-DEE; Epilepsy
Interventions
DRUG: PRAX-222 - Initial Dose; DRUG: PRAX-222 - Initial Ascending Doses; DRUG: PRAX-222 - Optional Ascending Doses; DRUG: PRAX-222 - Fixed Doses; PROCEDURE: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2023-04-13
Primary completion
2026-04-01
Completion
2028-02
Primary outcomes
  • Number of participants with treatment-emergent adverse events (Preliminary Safety, Dose Escalation) (Screening (-8 weeks) through up to 92 weeks)
  • Seizure frequency (Confirmatory Phase) (36 to 40 weeks)

NCT05737784 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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