Supporting Schizophrenia Clinical Trials: Keys to a Successful Study

Schizophrenia is a common, severe mental illness that affects how a person thinks, feels, and behaves. While symptoms may differ between patients, they are generally categorized into positive (e.g., hallucinations), negative (e.g., loss of motivation), and cognitive (e.g., trouble processing information) symptoms. (2024 National Institute of Mental Health) Schizophrenia onset typically occurs in late adolescence to early adulthood, affecting approximately 1% of the population.

Despite current treatment options, schizophrenia can be both chronic and disabling. According to a 2016 report, less than 11% of patients with schizophrenia hold gainful employment. That number decreases to less than 6% if the patient has multiple admissions. (Davidson et al., 2015)

While modern treatments for schizophrenia are effective for about half of patients, they focus on improving primarily positive symptoms of the disorder (e.g., hallucinations and thought disorders). (Stępnicki et al., 2018) Researchers are continuing to explore new treatment options to improve the long-term prognosis of patients.

Conducting Effective Schizophrenia Clinical Trials

In general, successful psychiatric trials require rigorous design, strategic planning, and vigilant monitoring and management.

There are many things to consider when planning a study within schizophrenia. As a contract research organization (CRO) that has supported over 150 CNS trials, including supporting schizophrenia clinical trials, PharPoint suggests consideration of the following:

• A Thoughtful Trial Design
• Consideration of Patient Traits
• Strategic Planning to Reduce High Drop Out Rates

A Thoughtful Trial Design: Questions to Ask as You Plan Your Schizophrenia Clinical Trial

An experienced, multi-functional team should be engaged early to ensure the design of a trial that properly balances multiple priorities — meeting a sponsor’s goals while appealing to patients, sites, and regulators.

You may want to consider questions such as:

• Would an adaptive study design benefit your trial?
• What’s your plan for blinding and randomization?
• How will you ensure rater consistency?
• Have you clearly planned for the potential use of rescue medication during study conduct?
• If your study design puts your trial at risk of a high number of missed visits, could telehealth options help mitigate this?

Consideration of Patient Traits

Studies must be planned with a clear understanding and consideration of patient traits – including how they may impact protocol clarity, enrollment, and overall patient-centricity.

Due to the nature of the disease, a protocol should clearly define the duration of the illness (e.g., first episode vs. chronic illness). The protocol should also clearly outline requirements for documenting response to previous treatment(s).

Exclusion criteria, including comorbidities, must be carefully considered and outlined to ensure a study’s validity without overly limiting enrollment potential. Schizophrenia is associated with substantial psychiatric and medical comorbidity, with research suggesting 50% of patients with schizophrenia have at least one comorbid condition. (Green et al., 2003)

Strategic Planning to Reduce High Dropout Rates

Trials recruiting patients with schizophrenia typically encounter a high drop out rate that must be accounted for in order to meet enrollment goals.

Studies on non-compliance among persons who have schizophrenia found the average non-compliance with pharmacotherapy to be shown at 42%, with a rate of missed medical appointments estimated at 24%. Studies evaluating psychosocial treatments may encounter a lower non-compliance rate, with a 2010 report indicating a drop out rate of approximately 13%. (Villeneuve et al., 2010)

Strategies to mitigate the risk of non-compliance and reduce drop out may include:

• Continually assessing adverse events and potential rescue strategies for non-responders;
• Working to better understand participant barriers with a site staff assessment of patient commitment to attend next study visit; and
• Leveraging participants’ external support systems and resources.

Get CRO Support for Your Upcoming Schizophrenia Clinical Trial

In conclusion, navigating the complexities of schizophrenia clinical trials requires experience, strategic planning, and a patient-centered approach.

For more information about how PharPoint’s experts can help you meet goals in your upcoming study, contact our team.

Sources

1. Villeneuve K, Potvin S, Lesage A, Nicole L. Meta-analysis of rates of drop-out from psychosocial treatment among persons with schizophrenia spectrum disorder. Schizophrenia Research. May 10, 2010. Accessed October 18, 2024. https://www.sciencedirect.com/science/article/abs/pii/S0920996410012569.

2. Green AI, Canuso CM, Brenner MJ, Wojcik JD. Detection and management of comorbidity in patients with … Psychiatric Clinics. March 2003. Accessed October 18, 2024. https://www.psych.theclinics.com/article/S0193-953X(02)00014-X/fulltext.

3. Stępnicki P, Kondej M, Kaczor AA. Current concepts and treatments of schizophrenia. MDPI. August 20, 2018. Accessed October 18, 2024. https://www.mdpi.com/1420-3049/23/8/2087.

4. Davidson M, Kapara O, Goldberg S, Yoffe R, Noy S, Weiser M. A nation-wide study on the percentage of schizophrenia and bipolar disorder patients who earn minimum wage or above. OUP Academic. March 20, 2015. Accessed October 18, 2024. https://academic.oup.com/schizophreniabulletin/article/42/2/443/2518910.

5. Schizophrenia. National Institute of Mental Health. April 2024. Accessed October 18, 2024. https://www.nimh.nih.gov/health/topics/schizophrenia.