ARISEN (Autoimmunity, Rasmussen’s, Inflammation & Status Epilepticus research Network)

Administrative Core - Project Summary The Administrative Core for ARISEN (Autoimmunity, Rasmussen’s, Inflammation & Status Epilepticus research Network) will ensure successful completion of the aims in this proposal. The goal of ARISEN is to improve outcomes in patients with autoimmune encephalitis (AE), Rasmussen syndrome, and NORSE (new onset refractory status epilepticus). These are rare diseases, and thus a multi-center registry will optimize our ability to understand the natural history, identify biomarkers, improve clinical trial readiness, and optimize treatments. The proposed ARISEN administrative core leverages the infrastructure and prior success of two consortia: (1) CONNECT (CONquering Inflammation and Epilepsies ConsorTium), which is the first multi-center prospective pediatric-focused U.S.-based registry for AE and Rasmussen’s, and (2) the NORSE Institute which includes children and adults. MPI Dr. Gombolay started and has led CONNECT with co-I Dr. Stredny experience with handling multiple institutions’ IRBs, subaward contracts, and data use/material transfer agreements. Dr. Gombolay also has assembled a large multicenter retrospective study that is the largest pediatric anti-NMDA receptor encephalitis (anti-NMDARE) database to date with data from 17 institutions on 298 children under 18 years of age diagnosed with anti-NMDARE. Dr. Murray (MPI) started collaborating with Dr. Gombolay in 2022 and supervised the collection of data for this study from Texas Children’s Hospital, her prior institution before joining Emory and CHOA as the new Chief Research Officer in April 2024. Drs. Gombolay and Murray will also draw on the experience from Dr. Clardy, PI of the multicenter U01 ExTINGUISH trial through NeuroNEXT, and Dr. Hirsch, PI of the multicenter NORSE registry. This team of strong leaders has the expertise to achieve the goals of this proposal. The ARISEN Administrative Core will provide oversight, coordination, and logistical support for the successful operation of the ARISEN registry. We have carefully selected a network of 24 sites that individually and synergistically provide experience within these diseases, along with a complementary breadth of expertise to effectively and comprehensively address the aims of ARISEN. Both CONNECT and NORSE have demonstrated successful recruitment of patients for biorepository research. Emory has provided scientific, regulatory, biostatistical, laboratory/ biorepository and data management support among the sites. The Administrative Core will promote a cohesive and inclusive network within ARISEN and patient advocacy groups (PAGs) (Aim 1) and will facilitate communication among all the sites, the External Advisory Committee, and key stakeholders from the PAGs (Aim 2). One of our PAGs will distribute the patient enrollment fees to the sites. The Administrative Core will provide effective support of the ARISEN Registry including maintaining the ARISEN registry’s website and overseeing the research projects along with input from the External Advisory Committee. The core will work closely within the Rare Disease Clinical Research Network (RDCRN) and with the RDCRN Data Management and Coordinating Center (DMCC). Project Number: 1U54HD122209-01 | Fiscal Year: 2025 | NIH Institute/Center: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Principal Investigator: GRACE GOMBOLAY | Institution: EMORY UNIVERSITY, ATLANTA, GA | Award Amount: $784,729 | Activity Code: U54 | Study Section: Special Emphasis Panel[ZTR1 RD-4 (02)] View on NIH RePORTER: https://reporter.nih.gov/project-details/11173108