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The Argus Trial: Just Another Case of Canadians Waiting for Access to Clinical Trials

Earlier this week, Epygenix Therapeutics shared that the first Canadian participant was dosed in their latest clinical trial, which they believe will be pivotal to the treatment of Dravet Syndrome (Epygenix Therapeutics, 2022). Epygenix is a clinical-stage biopharmaceutical company focused on genetically screening, discovering and developing safe and effective treatments for rare and intractable forms of epilepsy (Epygenix Therapeutics, 2022). Through their latest discovery, clemizole hydrochloride, they are hoping to decrease the incidence of seizures in patients with Dravet Syndrome (Epygenix Therapeutics, 2022).


Dravet Syndrome is a severe form of epilepsy in which patients experience frequent, prolonged seizures which are triggered by high body temperature (Sullivan, Knupp, and Wirrell, 2018). Patients with Dravet Syndrome often also experience developmental delay, speech impairment, ataxia, hypotonia, sleep disturbances, and many other health problems as well (Sullivan, Knupp, and Wirrell, 2018). Dravet Syndrome often appears in the first year of life in an otherwise healthy infant, with seizures lasting longer than 5 minutes (Sullivan, Knupp, and Wirrell, 2018). Additionally, patients often experience incoordination (ataxia) and low muscle tone (hypotonia) from a very early age which continues to affect them throughout their lives (Sullivan, Knupp, and Wirrell, 2018). As such, Dravet Syndrome can have significant impacts on a patient’s quality of life and participation in the activities that are meaningful to them.


Currently, there is no cure for Dravet Syndrome with many treatments currently attempting to reduce seizure frequency and duration (Sullivan, Knupp, and Wirrell, 2018). While a number of different treatments currently exist, this new treatment, called EPX-100 is the first of its kind to suppress seizures through its action on the serotonin signalling pathway (Dravet Syndrome News, 2020).


The first Canadian participant, an individual suffering from Dravet Syndrome, is now participating in a Phase 2 20-week randomized, double-blind, placebo-controlled trial (Epygenix Therapeutics, 2022). Recruitment for this clinical trial began across a variety of sites in the United States in July 2020 (ClinicalTrials.gov, 2022). After about a 2-year delay, a Canadian participant was finally dosed earlier this week, about 693 days since the clinical trial began (ClinicalTrials.gov, 2022). The first Canadian site for this clinical trial is at the Children’s Hospital of Eastern Ontario in Ottawa (ClinicalTrials.gov, 2022). The Canadian principal investigator, Dr. Sharon Whiting, the Vice-Dean of Faculty Affairs of the Faculty of Medicines at the University of Ottawa, is a paediatric neurologist who completed her fellowship in epilepsy.


Gaining access to this clinical trial marks a significant stride forward for the Dravet Syndrome patient population in Canada. Yet, once again, Canadian patients are left to wonder why it is that they must wait for access to clinical trials, when their neighbours in the United States can get access to treatments so much sooner.


As early as 2009, clinicians, researchers, patients and their families began to vocalise their fears that clinical trials were leaving Canada, with the number of phase II and III clinical trials decreasing significantly between 2002-2009 (Silversides, 2009). This trend has continued in recent years, with a continuing decrease in the number of clinical trials taking place in Canada every year (CITE).


Even when looking at Dravet Syndrome, there are 63 clinical trials currently operating in the United States (Clinicaltrials.gov, 2022), 17 in Europe (European Clinical Trial Register, 2022), and yet only 9 in Canada (Health Products Canada, 2022).


This trend is often seen with other clinical conditions as well. Research has identified that there are significant barriers to clinical trials in Canada, and therefore many pharmaceutical companies are deciding to pursue their clinical trials elsewhere (Bentley et al., 2020). Key stakeholders have identified that there is insufficient stable funding to foster clinical trial infrastructure in Canada and inadequate support to retain knowledgeable staff (Bentley et al., 2020). In addition, Canada is falling behind on the adoption of strict cost-recovery policies, leading to fewer academic trials (Bentley et al., 2020). In addition, there is an overreliance on the funding of clinical trials in Canada by the pharmaceutical industry, with insufficient investment from governments and public grant agencies (Bentley et al., 2020).


A decrease in the number of clinical trials in Canada results in delayed access to treatments for all patients, with this barrier impacting patients with rare diseases even more significantly. As patients continue to wait for access to treatments via clinical trials, they continue to experience decreased health outcomes, quality of life, and may experience avoidable symptom worsening or even death.


As such, there is an urgent need for new and innovative pharmaceutical innovation that is socially motivated. We need to engage with all relevant stakeholders to develop collaborative processes, programs, policies, and procedures to break the conventional pharmaceutical practices to ensure that patients with rare diseases gain safe, effective, and timely access to treatments to address their unmet medical needs.

 

References


Bentley, C., Sundquist, S., Dancey, J., & Peacock, S. (2020). Barriers to conducting cancer trials in Canada: An analysis of key informant interviews. Current Oncology, 27(3), e307-e312. 10.3747/co.27.5707


ClinicalTrials.gov. (2020, July 8). EPX-100 (Clemizole Hydrochloride) as Add-on Therapy to Control Convulsive Seizures in Patients With Dravet Syndrome - Full Text View - ClinicalTrials.gov. Clinical Trials. Retrieved June 2, 2022, from https://www.clinicaltrials.gov/ct2/show/NCT04462770?term=NCT04462770&draw=2&rank=1


Dravet Syndrome News. (2020, January 29). EPX-100. Dravet Syndrome News. Retrieved June 2, 2022, from https://dravetsyndromenews.com/epx-100/


Epygenix Therapeutics. (2022, June 1). First Canadian Participant Dosed in ARGUS Trial, A Phase 2 Potentially Pivotal Study of EPX-100 Treatment of Dravet Syndrome. GlobeNewswire. Retrieved June 2, 2022, from https://www.globenewswire.com/news-release/2022/06/01/2453872/0/en/First-Canadian-Participant-Dosed-in-ARGUS-Trial-A-Phase-2-Potentially-Pivotal-Study-of-EPX-100-Treatment-of-Dravet-Syndrome.html


European Clinical Trials Register. (2022, June 1). Dravet Syndrome Clinical Trials. European Clinical Drug Register. Retrieved June 2, 2022, from https://www.clinicaltrialsregister.eu/ctr-search/search?query=dravet+syndrome


Health Products Canada. (2022, June 1). Clinical Trial Query - Dravet Syndrome. Government of Canada. Retrieved June 2, 2022, from https://health-products.canada.ca/ctdb-bdec/search-recherche.do;jsessionid=2F A008DB6B1AAEAC737C1AF07C994B80


Silversides, A. (2009). Clinical trials: The muddled Canadian landscape. Canadian Medical Association Journal, 180(1), 20-22. 10.1503/cmaj.081897


Sullivan, J., Knupp, K., & Wirrell, E. (2018, March 1). Dravet Syndrome - NORD. National Organization for Rare Disorders. Retrieved June 2, 2022, from https://rarediseases.org/rare-diseases/dravet-syndrome-spectrum/


Photo credit: Mato Napo (2021). Boy in Blue Mask Giving Injection [Image]. https://unsplash.com/photos/1moqJf2GVpo

 

Written by: Shir Grunebaum (MSc)


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