ALS Studies And Lessons Learned From COVID-19

By Tomislav Babic, MD, Ph.D., Barry Dussault, Analgesia, Addiction, Stephen Kirzinger, MD, Chris Mason, Michael F. Murphy, MD, Ph.D., Nathalie Richard, Jeanine Ronniger, Konstantin Schupachenko, Lucie Undus, MD, Ph.D.

Disruption is the hallmark of the novel coronavirus (COVID-19) pandemic. Unless one lives and works in total isolation, all aspects of life have been disrupted to a greater or lesser degree — how individuals work, study, shop, recreate, travel, and more. At the same time, there has been significant regional variability in terms of the impact of disruptions due to changing epidemiological situations and regulations.

The disruptions caused by COVID-19 have complicated both the initiation and execution of clinical trials involving therapies targeting amyotrophic lateral sclerosis (ALS). In areas where stay-at-home orders or lockdowns have been put in place, individuals participating in a clinical trial may be unable to travel to a trial center or, because of possible risk factors, may be unwilling to travel to clinics for assessments. Conversely, ALS trial centers may be constrained from allowing entry to individuals who are infected with coronavirus. The study teams may have been directed to work from home. Pharmacies may be closed or operating on a restricted schedule; delivery services may operate inconsistently or on a delayed schedule; even the health care personnel working at the trial center may have been redeployed to an emergency department (ED) or intensive care unit (ICU) in response to a surge of coronavirus patients. The FDA, NIH, and IRB promulgated new clinical trial guidelines as the pandemic evolved. The Northeast ALS Consortium (NEALS) and European Network for Cure of ALS (ENCALS) also published ALS patient recommendations that would affect how clinical trials could be conducted.

For ALS trial sponsors and CROs alike, the need to protect the health of study participants, the study team, on-site health care professionals, and the broader community in which the trial centers are located is of paramount importance. The question is, how to ensure this protection and conduct the trial? The answers lie in preemptive mitigation and adaptive processes that can be implemented flexibly as conditions evolve.