中等度から重度の血友病Bの成人男性におけるPF-06838435を用いた第IX因子遺伝子治療の有効性と安全性を評価する研究

基本情報

NCT ID

NCT03861273

ステータス

実施中（募集終了）

試験のフェーズ

第3相

試験タイプ

介入

目標被験者数

51

治験依頼者名

Pfizer

概要

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).

対象疾患

介入

依頼者（Sponsor）

実施施設 (4)