Penn Blood Disorder Center

Trustees of The University of Pennsylvania

Hemophilia A

Hemophilia B

Pfizer

condition

allSponsorName

gender

Male

Female

overallStatus

Recruiting

Not yet recruiting

Active, not recruiting

Enrolling by invitation

Completed

Terminated

Withdrawn

Suspended

Unknown

type

Interventional

Observational

Expanded Access

phase

Early Phase 1

Phase 1

Phase 2

Phase 3

Phase 4

Not Applicable

funderTypes

Industry

Other U.S. Federal agency

All others

ageGroup

Child (birth-17)

Adult (18-64)

Older Adult (65+)

This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objective of the study will evaluate the annualized bleeding rate \[ABR\] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

Trustees of The University of Pennsylvania | Penn Blood Disorder Center

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A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)

Trial sponsors