(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).
This project is a practical application of return of individual and aggregate clinical trial data to participants in an open-label clinical trial of vamorolone in Duchenne muscular dystrophy. Sponsor-initiated, centralized design aimed to limit burden on study sites. Stakeholder input via interviews, and parental/physician feedback via surveys, will inform return of data to participants in the blinded, pivotal trial.
| Hoffman, Eric P; Riddle, Valerie; Siegler, Maxime A et al. (2018) Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Steroids 134:43-52 |
| Conklin, Laurie S; Damsker, Jesse M; Hoffman, Eric P et al. (2018) Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug. Pharmacol Res 136:140-150 |
| Hathout, Yetrib; Conklin, Laurie S; Seol, Haeri et al. (2016) Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children. Sci Rep 6:31727 |
