A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- Pfizer
- 入组人数
- 10
- 试验地点
- 25
- 主要终点
- Number of participants with abnormal and clinically relevant changes in neurological examinations
研究概览
简要总结
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
详细描述
The study will assess the safety and tolerability of fordadistrogene movaparvovec gene therapy. Approximately 10 participants will be enrolled in the study and receive a single IV infusion of PF-06939926; there is no placebo arm. The study includes boys who are at least 2 years old and less than 4 years old (including 3 year olds up until their 4th birthday). All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.
The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52). All participants will be followed in the study for 5 years after treatment with gene therapy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 3 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of DMD by prior genetic testing.
排除标准
- •Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive.
- •Positive test performed by Pfizer for neutralizing antibodies to AAV
- •Any prior treatment with gene therapy.
- •Any treatment designed to increase dystrophin expression within 6 months prior to screening (including, but not limited to, exon-skipping and nonsense read through).
- •Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD.
- •Abnormality in specified laboratory tests, including blood counts, liver and kidney function.
结局指标
主要结局
Number of participants with abnormal and clinically relevant changes in neurological examinations
时间窗: Through Week 52
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
时间窗: Through Week 52
Number of participants with abnormal hematology test results
时间窗: Through Week 52
Blood samples will be collected from subjects for the analysis of hematology
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
时间窗: Through Week 52
Number of participants with abnormal and clinically relevant changes on echocardiogram
时间窗: Through Week 52
Number of participants with abnormal biochemistry test results
时间窗: Through Week 52
Blood samples will be collected from subjects for the analysis of biochemistry
Number of participants with abnormal urine analysis
时间窗: Through Week 52
Urine samples will be collected from subjects for the analysis of urine
Number of participants with abnormal and clinically relevant changes in body weight
时间窗: Through Week 52
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
时间窗: Through Week 52
Number of participants with abnormal and clinically relevant changes in vital signs
时间窗: Through Week 52
次要结局
- Distribution of mini-dystrophin expression in muscle(At Week 9, Week 52 and Year 5 (if available))
- Number of participants with abnormal urine analysis(Through 5 years)
- Number of participants with abnormal and clinically relevant changes in neurological examinations(Through 5 years)
- Number of participants with abnormal and clinically relevant changes in body weight(Through 5 years)
- Level of mini-dystrophin expression in muscle(At Week 9, Week 52 and Year 5 (if available))
- Number of participants with abnormal biochemistry test results(Through 5 years)
- Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)(Through 5 years)
- Number of participants with abnormal and clinically relevant changes on echocardiogram(Through 5 years)
- Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events(Through 5 years)
- Number of participants with abnormal and clinically relevant changes in vital signs(Through 5 years)
- Number of participants with abnormal hematology test results(Through 5 years)
- Number of participants with abnormal and clinically relevant changes on cardiac troponin I(Through 5 years)