Establishing a Cost-effective Return of Results to Parents of Boys in VISION-DMD Clinical Trials

摘要(行政副刊) 在这个补充项目中，我们将研究一种创新和具有成本效益的返乡方法 杜兴男童家庭的个体和总体非遗传临床试验数据 肌营养不良症(DMD)参加安慰剂试验(n=168)。对此的评估 该方法将涉及社区利益相关者的广泛投入和个人和 汇总VBP15-002、003和LTE中参与者的试验数据。一个集中的赞助商- 启动的设计将用于重新同意并将患者与他们的数据连接起来，使用 单一协调员保护患者和行业研究团队之间的隐私防火墙。 在返回数据之前和之后，将对儿童的父母和 医生关于道德和后勤的培训。然后，该信息将通知返回数据到 VBP15-004的参与者。家长资助金是SBIR第二阶段和2B NINDS奖，用于 支持VBP15-002和VBP15-003治疗DMD的三项临床试验[48例患者， 4-7年；完成]；VBP15-004[120例患者，4-7年，2019年入选完成]。

Abstract (Administrative Supplement) In this supplemental project, we will study an innovative and cost-effective approach to returning individual and aggregate, non-genetic clinical trial data to families of boys with Duchenne muscular dystrophy (DMD) participating in vamorolone trials (n=168). The assessment of this approach will involve extensive community stakeholder input and return of individual and aggregate trial data to participants in VBP15-002, 003, and LTE. A centralized, sponsor- initiated design will be utilized for re-consent and connecting patients with their data, using a single coordinator to preserve privacy firewall between patients and industry study teams. Before and following return of data, surveys will be administered to the child’s parents and physicians regarding ethics and logistics. This information will then inform return of data to participants in VBP15-004. The Parent Grant is a SBIR Phase 2 and 2B NINDS award for support of three clinical trials of vamorolone in DMD (VBP15-002 and VBP15-003 [48 patients, 4-7 years; completed]; VBP15-004 [120 patients, 4-7 years, enrollment complete 2019).