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NCT05898620 (NGN-401 in Rett Syndrome): BVCT ex-ante prediction vs. reported outcome

On 31 Jul 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 11 Nov 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically probably significant (yet weak overall clinical benefit considering both efficacy and toxicity) and commercially probably insufficient.

Clinical Trial

NCTID
NCT05898620
Acronym
EMBOLDEN
Drug Name
NGN-401
Drug MoA
AAV9-delivered miRNA-regulated MECP2 gene therapy
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Rett Syndrome
Human Patients
16
Sponsor
Neurogene
Phase
Phase 1 Phase 2

Prediction of , index 1710

Prospective Prediction
Prediction Index
1710
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
103 days in advance
Readout Data Interpretation
low-dose NGN401 achieved 2-score CGI-I from baseline; high-dose NGN401 achieved 33.3% SAE (1/3)
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically probably significant (yet weak overall clinical benefit considering both efficacy and toxicity) and commercially probably insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Baseline-Controlled, Open-Label, Multicenter, Single-Arm, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of NGN-401 in Subjects With Rett Syndrome (Embolden)
Phase
Phase 3
Status
Active not recruiting
Enrollment
33
Lead sponsor
Neurogene Inc.
Conditions
Rett Syndrome
Interventions
GENETIC: NGN-401
Design
Non randomized · Sequential · Single
Primary purpose
Treatment
Start
2023-06-13
Primary completion
2029-12
Completion
2029-12
Primary outcomes
  • Efficacy of NGN-401 (52 Weeks)

NCT05898620 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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