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NCT04747431 (PBFT02 in Frontotemporal Dementia and Progranulin Mutations): BVCT ex-ante prediction vs. reported outcome

On 13 May 2024, BioinvestGPT's BVCT predicted technical partial success and commercial partial success for this trial. The 16 Sep 2024 readout is classified on the dashboard as True Positive (TP), a correct prediction. Recorded clinical-benefit conclusion: statistically significant and commercially TBD.

Clinical Trial

NCTID
NCT04747431
Acronym
upliFT-D
Drug Name
PBFT02
Drug MoA
AAV1-mediated GRN gene therapy
Drug Modality
gene therapy
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Frontotemporal Dementia and Progranulin Mutations (FTD-GRN)
Human Patients
15
Sponsor
Passage Bio
Phase
Phase 1 Phase 2

Prediction of , index 1607

Prospective Prediction
Prediction Index
1607
Prediction Date
Commercial Prediction
partial SUCCESS (commercially maybe sufficient clinical benefit in CDR + NACC FTLD)
Technical Prediction
partial SUCCESS (statistically significant yet weak clinical benefit in CDR + NACC FTLD)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
126 days in advance
Readout Data Interpretation
PBFT02 increased CSF PGRN expression up to 6-10 fold exceeding the range found in healthy adult controls; yet plasma PGRN expression remained below healthy reference levels.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically significant and commercially TBD
Prediction Classification
True Positive (TP)

ClinicalTrials.gov record

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

Official title
A Phase 1b Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacodynamic Effects of a Single Dose of PBFT02 Delivered Into the ICM of Adults With FTD and Mutations in the GRN or C9ORF72 Genes
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
30
Lead sponsor
Passage Bio, Inc.
Conditions
Frontotemporal Dementia; FTD; FTD-GRN; Dementia Frontotemporal; C9orf72
Interventions
DRUG: PBFT02
Design
Non randomized · Sequential · None
Primary purpose
Treatment
Start
2021-09-14
Primary completion
2028-08
Completion
2031-08
Primary outcomes
  • Number of Participants with Treatment-Related AEs and SAEs (Up to 5 years (multiple visits))
  • Change in Nerve Conduction Velocity and Amplitude from Baseline on Nerve Conduction Studies (From baseline to 5 years (multiple visits))
  • Change in Cellular and Humoral Response Against the Vector and Transgene in Serum (From baseline to 5 years (multiple visits))

NCT04747431 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

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