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NCT05089656 (OAV101 in Type 2 Spinal Muscular Atrophy): BVCT ex-ante prediction vs. reported outcome

On 1 Jul 2024, BioinvestGPT's BVCT predicted technical partial success and commercial failure for this trial. The 19 Mar 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially insufficient (1.88 point is below the 3-point minimal threshold of clinically meaningful improvement in HFMSE score (DOI-10.1007/s40120-024-00653-2).

Clinical Trial

NCTID
NCT05089656
Acronym
STEER
Drug Name
OAV101 (AVXS-101)
Drug MoA
rAAV9-mediated gene therapy with SMN1 transgene
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Type 2 Spinal Muscular Atrophy (SMA)
Human Patients
127
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3

Prediction of , index 1658

Prospective Prediction
Prediction Index
1658
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit in HFMSE at week 52 inferior to nusinersen)
Technical Prediction
partial SUCCESS (statistically maybe significant yet very weak clinical benefit in HFMSE at week 52)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
261 days in advance
Readout Data Interpretation
OAV101 achieved 1.88 point placebo-adjusted improvement in HFMSE score (p=0.0074); inferior to nusinersen’s 4.9 point placebo-adjusted improvement in HFMSE score (see add'l link below)
Press Release
Additional Readout Data
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially insufficient (1.88 point is below the 3-point minimal threshold of clinically meaningful improvement in HFMSE score (DOI-10.1007/s40120-024-00653-2)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory
Phase
Phase 3
Status
Completed
Enrollment
126
Lead sponsor
Novartis Pharmaceuticals
Conditions
Type 2 Spinal Muscular Atrophy
Interventions
GENETIC: OAV101; PROCEDURE: Sham control
Design
Randomized · Crossover · Quadruple
Primary purpose
Treatment
Start
2022-02-01
Primary completion
2024-11-12
Completion
2025-04-29
Results first posted
2025-12-08
Primary outcomes
  • Change From Baseline at the End of Period 1 in the Hammersmith Functional Motor Scale Expanded - Total Score - in the ≥ 2 to < 18 Years Age Group (Baseline, Week 52 (or Week 48))

NCT05089656 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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