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NCT04849741 (Zilganersen in Alexander Disease): BVCT ex-ante prediction vs. reported outcome

On 7 May 2025, BioinvestGPT's BVCT predicted technical partial success and commercial partial success for this trial. The 22 Sep 2025 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically borderline significant and commercially maybe sufficient (depending on whether 10MWT improvement exceeds MCID).

Clinical Trial

NCTID
NCT04849741
Drug Name
Zilganersen (ION373)
Drug MoA
degrades GFAP mRNA
Drug Modality
antisense oligonucleotide (ASO)
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Alexander Disease (AxD)
Human Patients
54
Sponsor
Ionis Pharmaceuticals
Phase
Phase 3

Prediction of , index 2013

Prospective Prediction
Prediction Index
2013
Prediction Date
Commercial Prediction
partial SUCCESS (commercially maybe sufficient yet weak clinical benefit in 10MWT at most near the minimal clinically important difference MCID)
Technical Prediction
partial SUCCESS (statistically maybe significant yet weak clinical benefit in 10MWT compared with placebo)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
138 days in advance
Readout Data Interpretation
Zilganersen achieved a statistically significant improvement in 10MWT at week 61 (mean difference 33.3% p=0.0412)
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically borderline significant and commercially maybe sufficient (depending on whether 10MWT improvement exceeds MCID)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients With Alexander Disease
Phase
Phase 3
Status
Active not recruiting
Enrollment
54
Lead sponsor
Ionis Pharmaceuticals, Inc.
Conditions
Alexander Disease
Interventions
DRUG: zilganersen; DRUG: Placebo
Design
Randomized · Parallel · Double
Primary purpose
Treatment
Start
2021-06-01
Primary completion
2025-08-22
Completion
2034-07
Primary outcomes
  • Percent Change from Baseline in the 10-Meter Walk Test (10MWT) (Baseline and Week 61)

NCT04849741 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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