After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial
After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial
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DOI：
10.1089/hum.2022.29198.bfs
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发表时间：
2022-02-01
影响因子：
4.2
通讯作者：
Philippidis，Alex
中科院分区：
文献类型：
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作者：
Philippidis，Alex
在辉瑞公司承认开放标签 C3391001 研究中一名年轻男性参与者死亡后，FDA 暂停了一项 Ib 期临床试验，该试验评估辉瑞公司的微型肌营养不良蛋白基因疗法候选药物 PF-06939926 治疗杜氏肌营养不良症 (DMD) 的效果。该患者参加了该试验的非动态队列 (NCT03362502)，这是一项首次人体/首次患者、多中心、非随机、剂量递增、安全性和耐受性研究。该研究旨在评估单次静脉输注 PF-06939926 对 DMD 患者的流动和非流动受试者的影响。辉瑞表示：“我们代表辉瑞的每个人，向他的家人、朋友和最亲近的人表示同情。”“目前，我们尚未掌握完整的信息，正在积极与试验现场研究人员合作，以了解发生的情况。”1 试验中的筛查和给药已暂停辉瑞则与该研究的独立外部数据监测委员会一起审查数据。
THE FDA HAS imposed a clinical hold on a Phase Ib trial assessing Pfizer’s mini-dystrophin gene therapy candidate PF-06939926 in Duchenne muscular dystrophy (DMD), after the company acknowledged the death of a young male participant in the openlabel C3391001 study. The patient participated in the nonambulatory cohort of the trial (NCT03362502), a first-in-human/first-in-patient, multicenter, nonrandomized, ascending dose, safety, and tolerability study. The study was designed to assess a single intravenous infusion of PF-06939926 in ambulatory and nonambulatory subjects with DMD.‘‘On behalf of everyone at Pfizer, we extend our sympathies to his family, friends and those closest to his care,’’Pfizer stated.‘‘At this time, we do not yet have complete information and are actively working with the trial site investigator to understand what happened.’’1 Screening and dosing in the trial has been paused while Pfizer reviews data with the study’s independent External Data Monitoring Committee.