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

在 VISION-DMD 临床试验中建立具有成本效益的结果返回给男孩父母

• 批准号: 9929267
• 负责人: Paula R Clemens
• 金额: $ 11万
• 依托单位: REVERAGEN BIOPHARMA, INC.
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-07-01 至 2020-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9929267
• 关键词: Administrative Supplement; Attitude; Authorization documentation; Award; Bioethics; Blinded; Caregivers; Child; Childhood; Chronic; Clinic; Clinical; Clinical Data; Clinical Trials; Communities; Conscious; Consent; Country; Data; Data Aggregation; Data Reporting; Databases; Degenerative Disorder; Disclosure; Disease; Double-Blind Method; Duchenne muscular dystrophy; Effectiveness; Enrollment; Ethics; European; Family; Family Physicians; Feedback; Fostering; Foundations; Goals; Human Resources; Individual; Industry; Informed Consent; International; Interview; Legal; Logistics; Mendelian disorder; Methods; Modeling; National Institute of Neurological Disorders and Stroke; Ownership; Parents; Participant; Patients; Perception; Personal Satisfaction; Phase; Physicians; Privacy; Process; Research; Research Personnel; Site; Small Business Innovation Research Grant; Son; Surveys; Trust; Uncertainty; arm; boys; cost; cost effective; data sharing; design; experience; firewall; health data; improved; innovation; knowledge base; non-genetic; open data; open label; parent grant; practical application; preservation; randomized trial; recruit; response; satisfaction; social media; therapeutic development

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).

摘要（行政补充） 在这个补充项目中，我们将研究一种创新且具有成本效益的返回方法 对Duchenne男孩家族的个人和骨料，非遗传临床试验数据 参加Vamorollone试验的肌肉营养不良（DMD）（n = 168）。评估 方法将涉及广泛的社区利益相关者的意见和个人回报 向VBP15-002，003和LTE中的参与者进行汇总试验数据。集中的赞助商 - 启动设计将用于使用A 单个协调员在患者和行业研究团队之间保留隐私防火墙。 数据返回之前和之后，将向孩子的父母和 关于伦理和物流的医生。然后，此信息将向数据返还到 VBP15-004的参与者。家长赠款是SBIR 2阶段2和2B NINDS奖 DMD中Vamorone的三项临床试验的支持（VBP15-002和VBP15-003 [48例，48例， 4 - 7年；完全的]; VBP15-004 [120例患者，4 - 7年，入学率完成2019年）。