Lead Investigator: Alexandre Betourne, Critical Path Institute
Title of Proposal Research: Sharing of Progressive Supranuclear Palsy (PSP) data with the Rare Disease Cures Accelerator – Data and Analytics Platform for Analysis of Outcome Measures and Other Purposes
Vivli Data Request: 5989
Funding Source:
Government – RDCA-DAP is funded by cooperative grant 2U18FD005320-06 from the FDA.
Commercial – Perhaps. Once the integrated database has been built and the community has agreed on analysis that could help resolve specific drug development issues, it is possible that such analysis may be supported by the companies.
Potential Conflicts of Interest: I run D-RSC, which is a non-profit consortium developing drug development tools that are suitable for regulatory uses. The consortium is supported by financial contributions of 8 drug companies and Parent Project Muscular Dystrophy, which contribute to the intellectual development of consortium projects. Companies are not permitted under the user agreement to discuss individual drug programs, and all results are made publicaly available after submission to the regulatory authorities (FDA and EMA). The requested data are for integration into the Rare Disease Cures Accelerator – Data and Analytics Platform, which is supported entirely through government grants.
Summary of the Proposed Research:
Progressive Supranuclear Palsy (PSP) is a rare disease where the first clinical trials have been completed, but no therapies have been approved The Rare Disease Cures Accelerator – Data and Analytics Platform (RDCA-DAP) is a data sharing and analytics platform set up with the goal of integrating rare disease datasets into larger sets of data for the purpose of developing tools to accelerate drug development across rare diseases. Integration of these PSP datasets with others from the community (currently accessing data from Biogen and academic sites) will increase the size of dataset that can be used for analysis. RDCA-DAP hopes to integrate these datasets, standardize them using CDISC standards to format the data for regulatory submissions and to provide access to the data through its analytics platform (degree of access to be determined with the data custodian). The data will then be available to C-Path quantitative medicine specialists for analysis and development of models of outcome assessments to help improve future clinical trial design (and other potential solutions).
Requested Studies:
An Extension Study of ABBV-8E12 in Progressive Supranuclear Palsy (PSP)
Sponsor: AbbVie
Study ID: NCT03391765
Sponsor ID: M15-563
A Randomized, Double-Blind, Placebo-Controlled Multiple Dose Study to Assess Efficacy, Safety, Tolerability, and Pharmacokinetics of ABBV-8E12 in Progressive Supranuclear Palsy
Sponsor: AbbVie
Study ID: NCT02985879
Sponsor ID: M15-562