2020 Fall Virtual CTSA Program Meeting

Christopher P. Austin, M.D. Director, NCATS

Welcome Session November 19, 2020 Congressional efforts to respond to the COVID-19 pandemic Four supplemental funding bills, including CARES Act

Title Law Overview NIH Impact Coronavirus Aid, PL 116-136 Total: $2.3 Trillion (est.) FY 2020 Relief, and (3/27/20) Funds available until 9/30/24 to prevent, Economic Provides one-time payments to families, loans prepare for, and respond to coronavirus, Security (CARES) to businesses, emergency spending for federal domestically or internationally: Act agencies, aid to states and local governments, an expanded social safety net for the jobless, NHLBI: $103,400,000 and a cash infusion for the nation's hospitals. NIAID: $706,000,000 (includes $156,000,000 for facilities) NIBIB: $ 60,000,000 NLM: $ 10,000,000 NCATS: $ 36,000,000 OD: $ 30,000,000 (Common Fund)

House and Senate have been unable to agree on a fifth Supplemental https://ncats.nih.gov/covid19-translational-approach NCATS OpenData Portal https://opendata.ncats.nih.gov/covid19/

Run as many screens and make our data against as many COVID against all immediately available targets as NCATS can… approved drugs… to the world The National COVID-19 Cohort Collaborative (N3C)

• Making available vast amounts of clinical EHR data for speeding COVID-19 research and improving patient care • Centralized, secure data enclave that provides access to medical record data from people diagnosed with COVID-19 across the US

• Harnesses resources of Clinical and Translational Sciences Awards (CTSA) Program hubs and Center for Data to Health (CD2H)

https://ncats.nih.gov/n3c NCATS Convalescent Plasma RCTs Multisite trials run through CTSA TIN and hubs • NYU/Einstein CONTAIN Trial (NCT04364737) . Begun in April during peak of NYC caseload, target enrollment n=300 . Expanded in August to additional sites and target enrollment to n=1000 . Current active sites: 16 . Current total enrollment (as of November 11): 437 • Vanderbilt PassItOnII Trial (Passive Immunity Trial Of Nation II for COVID-19) (NCT04362176) . Also begun in April, during peak of Nashville caseload . Also expanded in August to include additional sites and target enrollment to n=1000 . Current active sites: 11 . Current total enrollment (as of November 11): 163

Funded by NCATS CARES Supplemental and Operation Warp Speed ACTIV-1 Immunomodulators Trial NCATS CTSA Trial Innovation Network/Hubs + CRO Launched October 16, 2020

Randomized, placebo-controlled master protocol evaluating multiple therapeutic agents for the treatment of moderately or severely ill patients infected with SARS-Cov-2 associated cytokine storm

Evaluate each agent with respect to speed of recovery, mortality, illness severity, and hospital resource utilization. Each agent will be evaluated as add-on therapy to remdesivir (provided for all participants) plus the standard of care in use at local clinics, including convalescent plasma and dexamethasone in accordance with national guidelines.

Patient Population: Hospitalized adults (≥18 years old) with COVID-19, including patients both in and out of the ICU

STUDY AGENTS OUTCOME MEASURES

• Infliximab (Remicade): anti-TNFα mAb Primary Endpoint Elevated TNF-α levels in severe COVID-19 • Time to Recovery by Day 29 • Abatacept (Orencia): CTLA-4-Ig fusion protein Key Secondary Endpoints Binds to Ag presenting cells limiting T-cell activation • Clinical Status on day 15 & Day 29 defined • Cenicriviroc (CVC): SM CCR2/CCR5 antagonist by COVID 8-point ordinal scale • 14-Day and 28-Day mortality Decreases activity of infiltrating macrophages 7 Departing Steering Committee Members 2018 - 2020

Barry Coller, M.D. Phil Kern, M.D. William Powderly, M.D. Rockefeller University University of Kentucky Washington University in St. Louis UL1 Member UL1 Member UL1 Member SC Co-Chair Welcome, New Steering Committee Members! 2021 - 2023

Don McClain, M.D. David McPherson, M.D. Muredach Reilly, M.D. Wake Forest University University of Texas Health Columbia University UL1 Member Science Center at Houston UL1 Member UL1 Member CTSA Program Steering Committee Co-Chair Appointments

John Buse, M.D. Laura James, M.D. University of North Carolina, Chapel Hill University of Arkansas UL1 Member UL1 Member 2021 SC Co-Chair 2021 SC Co-Chair Designate 2022 SC Co-Chair CTSA Program Steering Committee Highlights Barry Coller, M.D. November 19, 2020 CTSA Program Steering Committee & Journal of Clinical and Translational Science COVID-19 Special Edition

• Virtual visits Synergistically combined: • Interacting with the FDA: IND and EUA • Prioritizing studies unrelated to the emergency • Modifying laboratory testing via Home Brew during the emergency • Pharmacy: procurement, compounding, formulation • Risk: benefit analyses related to re-opening studies • Biorepositories and risks to research personnel • N3C and role of informatics • Protecting research personnel • Community engagement • Health disparities • Prioritizing studies of COVID-19 • IRB procedures, AAHRPP, OHRP • Informed consent

This Photo by Unknown Author is licensed under CC BY-NC 13

Key takeaways from yesterday’s CTSA Program Steering Committee meeting CTSA Program Highlights

Michael G. Kurilla, MD-PhD November 19, 2020

FY20 CTSA Program Overview

• CTSA Program • New Hubs • New PIs • PI Accomplishments • Diversity and Re-Entry Support • Miscellaneous

• Trans-NIH & Trans-Agency Activities

• Addressing COVID-19

• New DCI Staff FY20 CTSA Program Hubs 18 New CTSA Program PIs (per Notices of Award 10/1/2019 - 10/28/2020)

Megan H. Bair-Merritt, MD, John M. Arthur, MD, PhD* MSCE* Alexander G. Bassuk, MD, PhD Elisabet Borsheim, University of Arkansas for Boston University Medical University of Iowa PhD* Medical Sciences Campus KL2 University of Arkansas for KL2 UL1 Medical Sciences KL2

Ginny Bumgardner, MD, PhD* Mario Castro, MD, MPH Richard T. D’Aquila, MD The Ohio State University Michelle A. Chui, PharmD, PhD* University of Kansas Medical Northwestern University TL1 University of Wisconsin-Madison Center TL1 at Chicago UL1 UL1

Michael Demetriou, MD, PhD* Brownsyne Tucker Edmonds, Leonard E. Egede, MD, MS Patrick A. Flume, MD* University of California at Irvine MD, MPH, MS Medical College of Medical University of South KL2 Indiana University-Purdue Wisconsin Carolina University at Indianapolis KL2 UL1 KL2

Carolyn J. Greene, PhD* Claudia Lucchinetti, MD Julie Lumeng, MD* Susanna McColley, MD University of Arkansas for Mayo Clinic Rochester University of Michigan Northwestern University at Medical Sciences UL1 at Ann Arbor Chicago KL2 UL1 TL1

*Multiple Principal Investigators 19 New CTSA Program PIs (per Notices of Award 10/1/2019 - 10/28/2020)

Duane A. Mitchell, MD, PhD Sharon M. Moe, MD* Ingrid E. Nygaard, MD, MS* Ruth O’Hara, PhD University of Florida Indiana University University of Utah Stanford University UL1 UL1 KL2 UL1

Nicholas J. Shaheen, MD, Shane A. Phillips PT, PhD, FAHA Richard Saitz, MD* Sarah J. Schlesinger, MD MPH* University of Illinois at Chicago Boston University The Rockefeller University University of North Carolina KL2 Medical Campus KL2 Chapel Hill UL1 UL1

Louise Walter, MD† Leena Sharma, MD Lauren Trepanier, DVM, PhD* Michael W. Varner, MD* University of California, Northwestern University at University of Wisconsin- University of Utah San Francisco Chicago Madison KL2 KL2 KL2 TL1

* Multiple Principal Investigators † Contact PI for Bauer (1 year) 20

NIH welcomes two new Institute Directors from CTSA hubs

Lindsey A. Criswell, M.D., M.P.H., D.Sc. will direct the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

• Currently Vice Chancellor of Research at the University of California San Francisco (UCSF) • Former Co-PI UCSF

Michael F. Chiang, M.D. will direct the National Eye Institute (NEI)

• Currently Knowles Professor of Ophthalmology & Medical Informatics and Clinical Epidemiology at Oregon Health & Science University (OHSU) • Mentor for KL2 and TL1 participants at OHSU 21 Dr. Marcella Nunez-Smith named as one of three co-chairs of COVID-19 Transition Advisory Board

“Everyone is affected by this pandemic, yet the burden is disproportionate. We know communities of color are grieving at high rates and are facing substantial economic impact. The transition advisory board is setting a course for everyone in our country to experience recovery.” – Dr. Marcella Nunez-Smith

• Yale University Associate Professor of Internal Medicine, Public Health, and Management • Associate Dean for Health Equity Research at the Yale School of Medicine • Deputy Director of the Yale Center for Clinical Investigation (CTSA) • Founding Director of the Equity Research and Innovation Center (ERIC) Photo credit: Robert A Lisak • Director of the Center for Research Engagement (CRE) Katy B. Kozhimannil, PhD, MPA 2020 Heinz Award in Public Policy recipient for her work on rising maternal mortality rates in the rural U.S. and especially among women of color

Her research has found that pregnant women in rural areas are 9% more likely to die from complications than those in more populated areas. Her work on how doulas could reduce maternal complications and racial inequities helped drive policy change in Minnesota: In 2013, the state passed legislation for Medicaid coverage of doula care.

• Health policy researcher at the University of Minnesota

• Community Engagement Team at UMN CTSA Hub • Part of the Mayo Maternal Mortality Admin Supplement (lead PI: Regan Theiler) “Acute Teleobstetrics services for management of maternal health emergencies.” 22 Congratulations to these newly elected members of the National Academy of Medicine

For identifying the For his expertise in For pioneering the genetic basis of more scientific inference and development of novel than 45 monogenic research reproducibility, methods to engage conditions (two of which utilizing diverse methods traditionally hard-to-reach bear her name) across a to inform public decisions communities in the wide range of diseases, about medical design and conduct of and for leading the interventions. His work clinical research, pivotal study of newborn has led to a series of effectively integrating screening for spinal critical contributions to participant and muscular atrophy. She national deliberative community perspectives. was the original plaintiff bodies, including medical Her innovations have in the Supreme Court journals, funders, transformed nationwide Wendy K. Chung, case on gene patents, Steven N. insurers, the courts, and Consuelo H. Wilkins, and global data science MD, PhD, Columbia which overturned the Goodman, MD, the NAM. MD, MSCI, Vanderbilt initiatives by addressing University Irving ability to patent genes. MHS, PhD, Stanford University Medical disparities in the Medical Center University School of Center research participation. Medicine CTSA Program Diversity/Re-Entry Supplement Awardees

Ange-Therese Akono, Alina Gavrila, MD, Franck Diaz Vasquez, Chenghui Li, PhD, MA PhD, MS MMSC PhD, MS Arkansas Northwestern Harvard Medical Wake Forest School

Sabrina Scroggins, PhD Peter Jenkins, MD, Aderonke Bamgbose Adam Lietzan, DMD, Alexis Dunn, MSN, BSN Iowa MS Pederson, MD, BA PhD Emory University Indiana Northwestern UNC Where are they now? do Aisha Langford, Ph.D, M.P.H. • Assistant Professor, Department of Population Health, New York University School of Medicine

• 2016: CTSA Program Diversity Supplement (NCATS): study use of recruitment support tools • 2018: Career Development Award (NHLBI): Explore patient & clinician preferences for hypertension management

• Focus on health communication to improve individual decision making & reduce health disparities

• Co-Direct of the NYU CTSA Recruitment and Retention Core (RRC)

• Associate editor for two journals: Patient Education and Counseling and Clinical Trials

CTSA Program Diversity Supplement Award to NYU UL1TR002548 med.nyu.edu/faculty/aisha-Langford Recognition Beyond NCATS: NIH Co-Funding

Collaboration Support For Awards with Better Integrate Behavioral and Social Sciences (BSS) with other Health-Related OBSSR 3 Sciences (NOT-OD-19-147 - TL1) Development of Training Modules in Genomic Medicine for Health Care NHGRI 3 Professionals (NOT-HG-20-020 - UL1)

CTSA Program KL2 Institutional Career Development Awards as part of Trans-NIH INvestigation of Co-occurring conditions across the Lifespan to Understand 4 (NICHD Lead) Down syndrome INCLUDE) Project (NOT-OD-20-022 - KL2) Trans-NIH Expand Vaping Research and Understand EVALI (E-cigarette vaping associated 1 (NHLBI Lead) lung injury) (NOT-HL-19-724 - UL1) Trans-NIH Research on Bioethical Issues (NOT-OD-20-038 - UL1/U01) 4 NIDCD Lancet Commission on Hearing Loss (Unsolicited - UL1) 1 All of Us Expansion of All of Us Sites and Collaboration with N3C (Unsolicited - UL1) 3 Trans-NIH Research Focused on Maternal Mortality (NOT-OD-20-104 - UL1) 11 Recognition Beyond NCATS: Operation Warp Speed

• Convalescent Plasma RCTs • Convalescent Plasma/Antibody Assays • ACTIV-1 Immunomodulator RCTs

27 Enormous Response by CTSA Community to the Pandemic

General Research Areas

Specific Research Areas

. Hundreds of Projects . A few will be highlighted in tomorrow afternoon’s COVID-19 session 28 Proactively Share Accomplishments with your Program Officer

29 Announcing new DCI staff for FY20

Gallya Gannot, D.M.D., Ph.D. Mercedes Rubio, Ph.D. Audie Atienza, Ph.D. Sarah Dunsmore, Ph.D. Jamie Doyle, Ph.D. Program Director Program Director Program Director Program Director, TIN Program Director

Danielle Stewart Miriam Perkins, M.A.T. Mary Purucker, M.D., Ph.D. Monica Carter, M.B.A. Staff Assistant Program Analyst Contractor, Welcome back! Program Analyst ncats.nih.gov @ncats_nih_gov @ncats.nih.gov NIH-NCATS

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