Pre-Conference examines trends in stroke clinical trial designs

Sub1Carmichael_largeClinical trial design is changing in stroke. New areas of clinical research include adaptive trial design, new outcome measures, imaging and serum biomarkers. Students, trainees and early career professionals are poised to take active roles in this changing landscape of stroke clinical research. Expert faculty will discuss new ways to test therapies and principles in stroke during the Tuesday, Feb. 10, “ISC Pre-Conference Symposium II: (for Students/Trainee/Early Career): Emerging Trends for Stroke Trials: Biomarkers, Adaptive Trial Design, Repair Trials and New Endpoints.”

“Our goal is to get the participants energized and focused on stroke research to help guide and strengthen their careers,” said Symposium Moderator S. Thomas Carmichael, MD, PhD, professor of neurology, vice chair for research and programs in neurology at the David Geffen School of Medicine and Co-director of the Broad Stem Cell Center at the University of California, Los Angeles.

In developing this program, Carmichael queried clinical scientists for input on the most interesting and emerging trends in stroke clinical trial design and research. He then narrowed the topics to those with the most academic output on PubMed and with the most rigorous science for trainees.

The afternoon will begin with a discussion on “Adaptive Trial Design” with Jordan Elm, PhD, research assistant professor of biostatistics at the Medical University of South Carolina, Charleston.

“Jordan will go over how we might  measure early on responses related to an important clinical variable and change randomization and patient entry. That’s a fundamental element of adaptive trial design,” Carmichael said.

Steven C. Cramer, MD, professor and vice chair of research in neurology, professor of anatomy and neurobiology, and the clinical director of the Sue and Bill Gross Stem Cell Research Center at the University of California, Irvine, will examine a critically emerging field during “Appropriate Design/Endpoints for Neural Repair Trials.”

“Neural repair after stroke is a completely different biology than that present in acute stroke. The measurement of outcomes in the acute and chronic period differ, particularly in the specific aspects of motor, sensory or cognitive recovery that are used to determine the success of a therapy. Steve is a leader in the design of neural repair trials,” Carmichael said.

Christian Foerch, MD, neurologist at Goethe University, Frankfurt, Germany, will share his insights on biomarkers during “Phase 2 Trial Biomarker Endpoint Designs.”

“Dr. Foerch and others have been at the forefront of a scientific movement to establish predictors of recovery, such as protein levels in blood or specific changes in brain imaging, that are outside of the time-consuming and inexact measurement of patient behavioral change after stroke. There has been a lot scientific energy in this field of biomarkers but not much actual achievement,” Carmichael said. “Now there is progress in understanding the type and nature of biomarkers to apply to acute stroke trials.”

Up next, Jeffrey L. Saver, MD, professor of neurology, director of the Stroke and Vascular Neurology Program in the department of neurology and medical director of the Stroke Center at the University of California, Los Angeles, will present “Approaches to Consent in Acute Trials Including Exception from Informed Consent and Short-Form Consents.”

“Jeff has been involved in many important stroke trials. His most recent trial really pushed the envelope of stroke care forward into the ambulance — right at the edge of the first medical interaction with the stroke patient. This FAST-MAG trial, actually gave therapy in the ambulance,” Carmichael said. “Jeff has thought a lot about how we might achieve the kind of consent necessary for a clinical trial in a setting in which traditional concepts of consent are somewhat degraded — degraded by time because you have to move fast and degraded by neurological condition because people may have language impairments or the inability physically to consent.”

An expert in clinical design innovation in stroke, Philip M. Bath, MD, Stroke Association Professor of Stroke Medicine and chair and head of the Division of Clinical Neuroscience at the University of Nottingham, United Kingdom, will give the final presentation on “Prevention Trial Endpoint Designs.”

“He will look at how we deal with some of the outcome measures in acute stroke trials. Many of them involve ordinal measures, and we can come up with ways of grouping them so we can push them to give us greater utility,” Carmichael said.

He added that the program would be exciting because changes in the field of clinical stroke research go against some long-held rules.

“In many ways stroke is moving ahead as other clinical research areas have done the same, such as the cancer and cardiology fields. This symposium will allow trainees and early stage investigators to understand these changes,” Carmichael said. “With this rapid change in stroke there are many opportunities in which a trainee can have a significant impact.”