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

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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).