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NCT04259281 (GTX-102 in Angelman Syndrome): BVCT ex-ante prediction vs. reported outcome

On 3 Jan 2024, BioinvestGPT's BVCT predicted technical success and commercial success for this trial. The 15 Apr 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially sufficient (no SoC).

Clinical Trial

NCTID
NCT04259281
Drug Name
GTX-102
Drug MoA
intrathecal UBE3A-AS ASO
Drug Modality
small interfering RNA (siRNA) antisense oligonucleotide
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Angelman Syndrome
Human Patients
74
Sponsor
Ultragenyx Pharmaceutical
Phase
Phase 1 Phase 2

Prediction of , index 1128

Prospective Prediction
Prediction Index
1128
Prediction Date
Commercial Prediction
SUCCESS (commercially sufficient clinical benefit)
Technical Prediction
SUCCESS (statistically significant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
103 days in advance
Readout Data Interpretation
rapid and clinically significant improvement resulting a total MRDI response of +2.0 (p<0.0001) at Day 170 and +2.0 (p=0.0007) at Day 338.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially sufficient (no SoC)
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS)
Phase
Phase 1, Phase 2
Status
Completed
Enrollment
74
Lead sponsor
Ultragenyx Pharmaceutical Inc
Conditions
Angelman Syndrome
Interventions
DRUG: GTX-102
Design
Non randomized · Parallel · None
Primary purpose
Treatment
Start
2020-02-24
Primary completion
2025-01-08
Completion
2025-01-08
Primary outcomes
  • Number of Participants with Adverse Events (AEs), Serious AEs (SAEs), Adverse Events of Special Interest (AESIs), AEs Leading to Discontinuation and Severity of AEs (Up to Day 337)

NCT04259281 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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