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NCT05818553 (relutrigine in SCN2A and SCN8A developmental and epileptic…): BVCT ex-ante prediction vs. reported outcome

On 5 Jan 2024, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. 2 readouts followed: 3 Sep 2024, True Positive (TP); 4 Dec 2025, True Positive (TP).

Clinical Trial

NCTID
NCT05818553
Acronym
EMBOLD
Drug Name
relutrigine
Drug MoA
anti-SCN8A antagonist
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs)
Human Patients
77
Sponsor
Praxis Precision Medicines
Phase
Phase 2

Prediction of , index 1244

Prospective Prediction
Prediction Index
1244
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient clinical benefit for SCN8A-mutant patients)
Technical Prediction
SUCCESS (statistically significant clinical benefit more for SCN8A-mutant patients than for SCN2A-mutant patients)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
242 days in advance
Readout Data Interpretation
46% placebo-adjusted reduction in monthly motor seizure and thus primary endpoint met (whether efficacy is better for SCN8A-DEE is pending for further data).
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially sufficient
Prediction Classification
True Positive (TP)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
699 days in advance
Readout Data Interpretation
relutrigine achieved strong clinical efficacy that triggered early stop for efficacy at interim analysis
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 Phase 2,Double-Blind,Randomized Clinical Trial to Explore the Safety,Tolerability,Efficacy, and Pharmacokinetics of PRAX-562 in Pediatric Participants With Developmental and Epileptic Encephalopathies Followed by Open-Label Extension(OLE)
Phase
Phase 2, Phase 3
Status
Active not recruiting
Enrollment
77
Lead sponsor
Praxis Precision Medicines
Conditions
SCN2A Encephalopathy; SCN8A Encephalopathy
Interventions
DRUG: PRAX-562
Design
Randomized · Parallel · Triple
Primary purpose
Treatment
Start
2023-08-02
Primary completion
2025-11-12
Completion
2027-03-01
Primary outcomes
  • PART A (Cohorts 1 and 2) RDB: To evaluate the safety and tolerability of PRAX 562 in pediatric participants with SCN2A- and SCN8A- DEEs (16 weeks)
  • PART B (Cohorts 1 and 2) OLE: To evaluate the long-term safety and tolerability of PRAX-562 in pediatric participants with DEEs (48 weeks)

NCT05818553 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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