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NCT04066244 (BLZ945 in Amyotrophic Lateral Sclerosis): BVCT ex-ante prediction vs. reported outcome

On 1 Jul 2024, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 26 Jul 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially insufficient.

Clinical Trial

NCTID
NCT04066244
Drug Name
BLZ945
Drug MoA
CSF1R inhibitor
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Amyotrophic Lateral Sclerosis (ALS)
Human Patients
28
Sponsor
Novartis
Phase
Phase 2

Prediction of , index 1638

Prospective Prediction
Prediction Index
1638
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient additive clinical benefit in ALSFRS-R)
Technical Prediction
FAILURE (statistically insignificant additive clinical benefit with moderate toxicity to accelerate ALS disease progression)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
25 days in advance
Readout Data Interpretation
not disclosed yet trial terminated after internal review of efficacy and toxicity available data
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
An Open-label, Adaptive Design Study in Patients With Amyotrophic Lateral Sclerosis (ALS) to Characterize Safety, Tolerability and Brain Microglia Response, as Measured by TSPO Binding, Following Multiple Doses of BLZ945 Using Positron Emission Tomography (PET) With the Radioligand [11C]-PBR28
Phase
Phase 2
Status
Terminated
Enrollment
28
Lead sponsor
Novartis Pharmaceuticals
Conditions
Amyotrophic Lateral Sclerosis
Interventions
DRUG: BLZ945
Design
Randomized · Sequential · None
Primary purpose
Treatment
Start
2019-12-30
Primary completion
2024-02-01
Completion
2024-02-01
Results first posted
2025-03-19
Primary outcomes
  • Cohorts 1-4 : Change From Baseline in Volume of Distribution (Vt) in Different Brain Regions for [11C]-PBR28 PET Scan (Baseline, day 5)
  • Cohort 5 (PET Sub-study): Change From Baseline in Volume of Distribution (Vt) in Different Brain Regions for [11C]-PBR28 PET Scan (Baseline, day 84)
  • Cohort 5: Change From Baseline in Esophageal Wall Thickness (Baseline, Day 84)
  • Cohort 5: Cardiac Valve Thickness at Day 84 Compared to Baseline (Baseline, Day 84)
  • Cohort 5: Cardiac Valve Stenosis at Day 84 Compared to Baseline (Baseline, Day 84)

NCT04066244 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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