When clinical trials get cancelled and do not lead to an FDA approved drug, it can cause emotional challenges for participants who had started seeing improvements during the trial phase. A recent article, published by the Chicago Tribune, reported that the clinical trial using the drug Arbaclofen for fragile X syndrome came to a halt as it did not meet the main goal of the study related to social behavior. The trial was a joint effort by Seaside Therapeutics and Roche Pharmaceuticals. Fragile X syndrome is a genetic condition and does not have any known treatment yet. It affects approximately 1 in 100,000 people in the US.
Participants were in the age range of 12 to 25 years old and several of these participants had actually started seeing improvements in their social interaction. The study raised hopes for so many mothers whose kids started seeing improvements in vocabulary and social behavior. This story highlights the complexities of clinical study results and the challenges in bringing new drugs in the market. It also portrays how participants and their families start perceiving the study as their only hope for diseases that have no known cures. This is known as therapeutic misconception. From the point of view of the trial, it is a misconception to think that it has anything to do with meaningful personal therapy or amelioration of disease. From the point of view of the individual and family, it is often the only hope in a sea of suffering. A link to the story is here.
If you or someone you know would like to participate in a clinical trial and would like to get connected to the right researcher, please visit us at Reg4All.