Advancing Craniosynostosis Treatment (ACT) Rare Diseases Consortium Administrative Core

Children'S Hosp Of Philadelphia, Philadelphia PA

Investigators

Abstract

Modified Project Summary/Abstract Section ABSTRACT: ADMINISTRATIVE CORE The Advancing Craniosynostosis Treatment (ACT) Rare Diseases Consortium brings together 4 leading craniofacial centers and PEDSnet to study 5 craniosynostosis (CS) syndromes: Apert, Pfeiffer, Crouzon, Muenke, and Saethre-Chotzen. The Administration Core is established to enable the operational success of ACT across all participating sites and readily integrate ACT into the Rare Diseases Clinical Research Network (RDCRN). To achieve this, the Administration Core will have the following main efforts: 1) Operationalizing ACT across sites and RDCRN, through leadership, administrative support, communication, establishment of consortium-wide procedures and collaboration with the Data Management and Coordinating Center (DMCC). 2) Ensuring regulatory compliance through oversight of clinical projects, human subjects research, and data management. 3) Partnering with patient groups to engage patient populations with syndromic CS. 4) Providing administrative oversight for the Pilot/Feasibility Governance Core and Career Enhancement Core to enable each Core’s respective mission. The leadership team of the ACT Administrative Core comprises of experts in craniofacial surgery, genetics, pediatrics, dentistry, biostatistics, bioinformatics, and clinical data management. Additionally, all administrative personnel have extensive experience that will optimize the success of the Administrative Core. Overall, the Administrative Core sets forth the critical organizational infrastructure for productivity and collaboration across all ACT sites and compliance and data sharing across DMCC and RDCRN.