Addressing ‘highly complex’ ASD clinical trials: Unique challenges and novel outcomes

By Melissa Fassbender contact

- Last updated on GMT

(Image: Getty/Radchynsky)
(Image: Getty/Radchynsky)

Related tags: Clinical trial

Inclusion criteria, standardized procedures, and high-quality clinician training are key to conducting clinical trials in individuals with autism spectrum disorder, says industry expert.

Pamela Ventola PhD recently joined the advisory committee for Q BioMed’s QBM-001 clinical program. QBM-001 is being developed and tested for the treatment of minimally verbal or non-verbal toddlers with an autism spectrum disorder (ASD).

Ventola is a clinical psychologist and assistant professor at Yale University's Child Study Center and also directs the Rare Disease and Pediatric Center of Excellence at Cogstate.

As part of her research, Ventola is using eye-tracking paradigms as a novel outcome of clinical trials in ASD. To further discuss her work, Outsourcing-Pharma caught up with Ventola (PV) to discuss the unique challenges involved and how they can be addressed.

OSP:What are the unique challenges to conducting clinical trials with patients with autism spectrum disorders?

Conducting trials with individuals with ASD is highly complex. ASD is a heterogeneous condition, so inclusion criteria are key. Similarly, ASD is a behaviorally-defined disorder, so confirming diagnosis by experienced clinicians is also crucial.

Once subjects are enrolled in the study, completing the necessary outcome assessments is also important. Many children with ASD have challenging behaviors and limited language and social motivation, so experienced and highly trained clinicians are needed in order to obtain accurate data.

OSP:How can these challenges be faced?

These challenges can be addressed by employing high-quality clinician training as part of the trial start-up process.

Also, clear and standardized procedures (methodologies defined in the study protocol) across sites and across raters will increase data quality.

OSP:How is informed consent addressed for patients with autism spectrum disorders?

For children, the parents have legal rights to make decisions about the subjects. For adults, often parents have completed legal proceedings in order to make medical decisions for their children even after the individual has reached legal age (parents have legal guardianship).

Regardless of who can legally consent to study participation, for all subjects, site staff explains the procedures to the subject in language that the he/she can understand, often with visual supports as well, particularly for younger children.

For subjects who are able based on cognitive functioning, they provide assent to participate in the trial. This process of consent and assent is typically directed by the site’s Institutional Review Board (IRB).

OSP:In what ways are clinical outcomes assessed?

Given a number of complexities in ASD, there are no consensus outcomes in ASD. ASD is a heterogeneous condition, so no one outcome measure can be used for all individuals across age and cognitive functioning levels.

Additionally, the core features in ASD are abstract and difficult to measure. Furthermore, many of the available outcomes are broad and not sufficiently precise or granular in order to detect change.

Lastly, many of the available outcomes are also subjective and rely on the judgment of clinicians or caregivers, as opposed to direct, objective measures. Given these complexities, outcome measures vary between trials. They include caregiver report measures, such as the Vineland Adaptive Behavior Scales, clinician-reported measures, such as the Clinical Global Impression Scale, or direct assessments with the child, such as measures of language production (expressive language).

There are also a range of more experimental outcome measures that are investigating biological markers of treatment response. Examples of such approaches include neuroimaging and eye tracking.

OSP:How are you using eye-tracking paradigms as a novel outcome of clinical trials in ASD?

We developed novel eye-tracking paradigms to determine how eye tracking approaches can be used as outcome measures for clinical trials in ASD. My laboratory in collaboration with Dr. Fred Shic and his laboratory at the University of Washington, developed paradigms specifically designed to measure the key behaviors targeted our clinical trial (e.g., reciprocity or going back and forth in a conversation).

The children completed the eye tracking sessions before, at mid-point, and following participation in the treatment program. We are now looking for changes in gaze patterns that may serve as an objective marker of treatment response.

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