BioinvestGPT

BioinvestGPT ApS · Research use only

NCT06152237 (TSHA-102 in Rett Syndrome): BVCT ex-ante prediction vs. reported outcome

On 14 Feb 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 18 Jun 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically maybe significant and commercially insufficient.

Clinical Trial

NCTID
NCT06152237
Acronym
REVEAL
Drug Name
TSHA-102
Drug MoA
scAAV9-delivered miniMECP2 gene therapy
Drug Modality
AAV9-based gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Rett Syndrome
Human Patients
6
Sponsor
Taysha Gene Therapies
Phase
Phase 1

Prediction of , index 1464

Prospective Prediction
Prediction Index
1464
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
125 days in advance
Readout Data Interpretation
weak efficacy in seizure reduction (none is seizure free without anti-seizure medication).
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically maybe significant and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Multicenter, Open Label, Randomized, Dose-Escalation and Dose-Expansion Study of the Safety, Tolerability, and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females With Rett Syndrome
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
6
Lead sponsor
Taysha Gene Therapies, Inc.
Conditions
Rett Syndrome
Interventions
GENETIC: TSHA-102
Design
Randomized · Sequential · None
Primary purpose
Treatment
Start
2023-12-12
Primary completion
2028-11-02
Completion
2031-11-02
Primary outcomes
  • Primary Safety (Baseline through week 52)

NCT06152237 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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