In Toronto, an international study is underway for an experimental drug for people with Autism Spectrum Disorder (ASD), and the community is divided on the use of the drug manufactured by the sponsor Hoffmann-La Roche. Called Balovaptan, the drug could potentially help people on the spectrum better manage normal challenges from communications to social engagement. The study lead is Dr. Evdokia Anagnostou, an investigator with Toronto’s Holland-Bloorview Kids Rehabilitation Hospital.
Autism Spectrum Disorder
Multinational drug developer Roche reports that ASD is associated with a wide range of symptoms grouped into two main categories including 1) core and 2) associated. Core symptoms include impairments of social interaction and communication, not to mention repetitive behavior and restricted interests, all of which have a significant impact on daily functioning. Associated symptoms can include anxiety, seizures, language disability, sensory issues, attention deficit, mood alternations, and others.
ASD is four times more commonly diagnosed in boys than girls. In America, it is estimated that 1 in 42 boys and 1 in 189 girls have ASD. The World Health Organization reports that the global prevalence of ASD is approximately one in every 160 people and is 0.3% of the global burden of disease. In the European Union, it is estimated that the prevalence of ASD can range from 57 to 67 per 100,000 children.
The study evaluates the efficacy, safety, and pharmacokinetics of 10 mg of oral administration balovaptan once a day (QD) compared with matching placebo in adults (18 years and older) with autism spectrum disorder (ASD). In the multi-site Phase III study sponsor, Roche seeks 350 participants. It commenced in August 2018 and concludes in March 2023.
One of the Canadian sites, reports Toronto City News, is Holland-Bloorview, which on the ground for this international study will distribute half the participants the drug and the other half a placebo. Other Canadian investigational sites include Okanagan Clinical Trials, University of Western Ontario, and McGill University. Dr
A Participation Evaluation Screening
Before signing up a participant, the study team must first screen participants to determine whether or not they are the right fit for this study. The participating investigators and their medial staff must help them determine the participant’s history, as well as any risks or benefits possibly derived from this study.
What is Balovaptan?
By January 2018, the U.S. Food and Drug Administration (FDA) had declared Breakthrough Therapy Designation for balovaptan in individuals with autism spectrum disorder (ASD). This status was awarded due to the output of a Phase II adult clinical trial known as VANILLA (Vasopressin Antagonist to improve social communication in Autism) study.
Known as RG7314, it is a selective molecule antagonist of the vasopressin V1A receptor, which is under development by the Swiss company for the treatment of autism. Ongoing clinical trials by the end of 2019 include a Phase III clinical trial for adults and a Phase II for the treatment for children for this indication. A current Phase III adult study is called V1aduct, and the latest Phase II child study is known as Av1ation.
Canadian Site Clinical Investigator
Dr. Evdokia Anagnostou, the study’s lead researcher at Holland-Bloorview Kids Rehabilitation Hospital, notes that the experimental drug is “regulating hormones in our brains that have to do with how we perceive the social world, whether we understand social cues, how we relate to other people, and how we develop close bonds and relationships.” She noted, “The idea would be, people who have difficulty with everyday social functions, may have one more potential option for thinking of interventions if they choose to use them.”
Dr. Anagnostou notes on the experimental drug’s safety that “Although we know quite a bit about its safety, we don’t have a final decision on it, so we monitor people closely.”
Groups such as Autism Ontario, representing families across the province are supportive of the study, believing this drug shows the potential to help those struggling with social interactions due to ASD. Although the news channel reported, other groups have concerns about the ethics of the study. Dr. Anagnostou hits back that the ethical thing to do is to offer those struggling with ASD and in need “options about how to improve their well-being and their functioning.”
Margaret Spoelstra, Executive Director of Autism Ontario, supports the study and emphasizes that the other ways to support those struggling with autism, is to open more doors for services and supports for those afflicted by the disorder.
Some Growing Concerns of Overly Broad Diagnoses
At least in Canada, there are some growing calls of concern that ASD is over-diagnosed. A Canadian researcher and psychiatrist point to a meta-analysis published in JAMA Psychiatry where investigators in Denmark and Quebec analyzed 11 previous major reviews on people with autism and people with the rest of the population. One of the study’s authors, Laurent Mottron, a research psychiatrist at the mental health unit of Montreal’s Riviere-des-Prairies Hospital and a study author notes, the challenge centers on the actual criteria to the point that a diagnosis can become essentially meaningless. On the other hand, researchers from providers such as Holland-Bloorview will counter that ASD diagnosis is based on criteria such as poor social skills, difficulties maintaining relationships, etc.
Holland-Bloorview Kids Rehabilitation Hospital
Holland-Bloorview Kids Rehabilitation Hospital offers both inpatient and outpatient services and strive to provide exceptional care for clients and families. The provider dates back to the 19th century when a group of dedicated women organized to get built a facility for children with disabilities and chronic illnesses. From 2018-2019 they served 8,313 clients, recorded 455 inpatient admissions, 53,491 outpatient visits and recorded 2,310 unique diagnoses.
The provider’s Bloorview Research Institute (BRI) is recognized in Canada and around the world for its leadership in the field of childhood disability research. See the research page for ongoing studies.
Lead Research/Investigator in Toronto, Canada
Dr. Evdokia Anagnostou, the study’s lead researcher at Holland-Bloorview Kids Rehabilitation Hospital
Call to Action: Do you have a loved one with an ASD diagnosis that you think could benefit from this study? Depending on where you are located, you can call the sponsor for more information. Feel free to contact TrialSite News as well, as our research team can help out.