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NCT04370054 (PF-07055480 /​ Giroctocogene Fitelparvovec in Hemophilia A): BVCT ex-ante prediction vs. reported outcome

On 15 Jan 2024, BioinvestGPT's BVCT predicted technical success and commercial partial success for this trial. The 24 Jul 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially sufficient.

Clinical Trial

NCTID
NCT04370054
Acronym
AFFINE
Drug Name
PF-07055480 /​ Giroctocogene Fitelparvovec
Drug MoA
rAAV6 vector carrying a B-domain-deleted F8 gene
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Hematologic Diseases
Indication
Hemophilia A
Human Patients
76
Sponsor
Pfizer / Sangamo Therapeutics
Phase
Phase 3

Prediction of , index 1413

Prospective Prediction
Prediction Index
1413
Prediction Date
Commercial Prediction
partial SUCCESS (commercially sufficient clinical benefit with superior-to-Roctavian efficacy and non-superior-to-Roctavian toxicity)
Technical Prediction
SUCCESS (statistically significant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
191 days in advance
Readout Data Interpretation
superior efficacy (98.3% ABR reduction vs 84% ABR reduction by Roctavian) and non-superior toxicity (20% SAE vs 16.4% SAE).
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially sufficient
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants With Moderately Severe to Severe Hemophilia A(FVIII:C≤1%)
Phase
Phase 3
Status
Active not recruiting
Enrollment
77
Lead sponsor
Pfizer
Conditions
Hemophilia A
Interventions
BIOLOGICAL: PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Design
Single group · None
Primary purpose
Treatment
Start
2020-08-18
Primary completion
2024-06-17
Completion
2026-10-25
Results first posted
2025-08-17
Primary outcomes
  • Total Annualized Bleeding Rate (ABR) (FVIII Prophylaxis arm: a minimum of 7.4 months, up to maximum of 32.3 months (Pre-infusion period); PF-07055480 arm: Week 12 through at least 15 months of follow-up, maximum follow up was of 44.4 months (Post-infusion period))

NCT04370054 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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