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

摘要（行政补充） 在这个补充项目中，我们将研究一个创新和具有成本效益的方法， 杜氏症男孩家庭的个体和汇总非遗传临床试验数据 参与vamorolone试验的肌营养不良症（DMD）患者（n=168）。对此的评价 方法将涉及广泛的社区利益相关者的投入和个人的回报， 将试验数据汇总至VBP 15 -002、003和LTE中的受试者。一个集中的，赞助商- 启动的设计将用于重新知情同意，并使用 单一协调员，以保护患者和行业研究团队之间的隐私防火墙。 在数据返回之前和之后，将对儿童的父母进行调查， 关于伦理和后勤的医生。然后，此信息将通知将数据返回给 VBP 15 -004中的参与者。父母补助金是SBIR第2阶段和2B NINDS奖， 支持在DMD中进行的三项瓦莫龙临床试验（VBP 15 -002和VBP 15 -003 [48名患者， 4-7年;已完成]; VBP 15 -004 [120例患者，4-7年，2019年完成入组）。

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).