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NCT05349721 (PTC857 in Amyotrophic Lateral Sclerosis): BVCT ex-ante prediction vs. reported outcome

On 6 Aug 2024, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 26 Nov 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
NCT05349721
Acronym
CARDINALS
Drug Name
PTC857
Drug MoA
ALOX15 inhibitor
Drug Modality
small molecule
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Amyotrophic Lateral Sclerosis (ALS)
Human Patients
307
Sponsor
PTC Therapeutics
Phase
Phase 2

Prediction of , index 568

Prospective Prediction
Prediction Index
568
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient additive clinical benefit in ALSFRS-R)
Technical Prediction
FAILURE (statistically insignificant additive clinical benefit in ALSFRS-R)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
112 days in advance
Readout Data Interpretation
PTC857 failed to achieve any primary or secondary efficacy endpoints
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
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Assess the Efficacy, Safety, Tolerability, PK, and Biomarker Effects of PTC857 in Adult Subjects With Amyotrophic Lateral Sclerosis (CARDINALS)
Phase
Phase 2
Status
Terminated
Enrollment
336
Lead sponsor
PTC Therapeutics
Conditions
Amyotrophic Lateral Sclerosis
Interventions
DRUG: PTC857; DRUG: Placebo
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2022-05-15
Primary completion
2024-09-26
Completion
2025-01-30
Results first posted
2025-08-01
Primary outcomes
  • Combined Assessment of Function (ALS Functional Rating Scale-Revised [ALSFRS-R]) and Survival (CAFS) Rank After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population) (Week 24)

NCT05349721 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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