This is a post by Autism Speaks’ Assistant Director of Science Communication and Special Projects Leanne Chukoskie, Ph.D.
On Friday, a short post with a link to a clinical trial led by Curemark was highlighted in e-Speaks and on Autism Speaks’ Facebook page. The post generated a many questions and comments, some of which could be addressed with more information about clinical trials in general.
Clinical trials are research tools for studying the health and well-being of people. Clinical trials are not always focused on treatment, but may include studies of better methods for diagnosis, screening or improving quality of life. A description of types of clinical trials is available at an informative website provided by the National Institutes of Health in the FAQ.
In addition to the different types of trials conducted, any clinical trial involving a new drug enters a phased series of tests to assess the safety and efficacy of the drug for a particular population. A Phase I clinical trial is small and establishes safety and appropriate dosage. In Phase II, the trial is expanded to include more subjects so a better estimate of effectiveness and safety can be established. Phase III trials can be conducted after preliminary evidence for the effectiveness and safety of the drug has been favorable. In this phase of study, the effectiveness of the treatment, any potential side effects of the treatment are closely monitored in a larger population. Also this phase typically includes a comparison of the treatment under study with other drugs available for the same condition. A Phase IV clinical trial is called “post-market” research because it is conducted after a drug has been made available for use in the general population. Phase IV studies typically include several thousand participants and help to refine aspects of the treatment’s best usage and ideal treatment candidate.
The Curemark study noted in Friday’s post is a Phase III treatment trial that aims to compare the effectiveness of a compound called CM-AT versus a placebo administered 3x per day for 90 days. This particular trial is “double-blind” meaning that neither the research participants nor the clinical staff administering the treatment know whether a subject is receiving the active compound or a placebo at the time the treatment is given. Double-blinded studies are believed to produce more objective results because the outcomes are not influenced by the subjects’ or clinicians’ expectations about the treatment. Treatment information will be revealed for all subjects when the blind is broken at the end of the study at which point an analysis of the effectiveness of the new treatment versus control can be compared.
The clinicaltrials.gov website lists 219 studies that result in a search for “autism.” A smaller number (110) are seeking volunteers. The treatments being assessed include behavioral therapies, different drug compounds, transcranial magnetic stimulation (TMS) and others from researchers all over the world. 12 of the 219 studies were sponsored by Autism Speaks and nine of those are actively recruiting. We encourage anyone interested in participating in research to seek more information about these studies.