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NCT04281485 (PF-06939926 in Duchenne Muscular Dystrophy): BVCT ex-ante prediction vs. reported outcome

On 15 Jan 2024, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 12 Jun 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially insufficient (SRP9001 at least meet some secondary endpoints).

Clinical Trial

NCTID
NCT04281485
Acronym
CIFFREO
Drug Name
PF-06939926
Drug MoA
rAAV9 vector carrying a minidystrophin gene
Drug Modality
gene therapy
Drug Class
Best-In-Class
Therapeutic Area
Musculoskeletal Diseases
Indication
Duchenne Muscular Dystrophy (DMD)
Human Patients
122
Sponsor
Pfizer
Phase
Phase 3

Prediction of , index 1414

Prospective Prediction
Prediction Index
1414
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient clinical benefit with inferior-to-SRP9001 efficacy)
Technical Prediction
FAILURE (statistically insignificant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
149 days in advance
Readout Data Interpretation
statistically insignificant for all primary/secondary endpoints.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant and commercially insufficient (SRP9001 at least meet some secondary endpoints)
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

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

Official title
A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Phase
Phase 3
Status
Active not recruiting
Enrollment
114
Lead sponsor
Pfizer
Conditions
Duchenne Muscular Dystrophy
Interventions
GENETIC: PF-06939926; OTHER: Placebo; OTHER: Placebo; GENETIC: PF-06939926
Design
Randomized · Parallel · Quadruple
Primary purpose
Treatment
Start
2020-11-05
Primary completion
2024-05-15
Completion
2039-04-15
Results first posted
2025-06-08
Primary outcomes
  • Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 (Baseline, Week 52)

NCT04281485 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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