A response from LuMind Research Down Syndrome President and CEO Carolyn Cronin regarding the end of the Roche Clinical Trial for CLEMATIS.
Like you we were disappointed to learn about the end of Roche Clinical Trial for CLEMATIS, the Phase II study investigating the efficacy and safety of basmisanil (RG1662) in adults and adolescents with Down syndrome. Roche issued a statement saying that while there was not a safety related reason to end the study, there was no significant difference between those who took the drug and those that took the placebo in the adult (18-30 years old) or adolescent (12-17 years old) study groups.
We are hearing from many of you with a family member in the clinical trial that your experience was different. You saw positive changes.
Scientists and researchers rely on numbers and statistical results and hold dearly to empirical data. But the changes you witnessed in your loved one and the hope you felt about this drug also have great value.
In December 2014, we wrote a post on our plus15 blog about the story of Fragile X and one family’s quest for treatments for their son Andy (NPR Story “A Family’s Long Search For Fragile X Drug Finds Frustration, Hope”). The family started a foundation, funded research, and almost 20 years later saw positive results for their son in a clinical trial. They were devastated when the results of the clinical trial indicated no significant change in the group overall.
In the article the father, also a scientist, stated, “Most clinical trials have some people who do really well even when the overall result is not necessarily great.”
Even though the experimental Fragile X drug is no longer available, Andy’s family believes in the changes they saw and recommitted themselves to the research effort so the next clinical trial or the one after that will be successful.
“In clinical research there are often disappointments from the laboratories through clinical trials,” said Dr. Michael Harpold, LuMind RDS’s chief scientific officer. “We often say we learn as much from negative results as we do from the successes – and that’s true. Roche further announced that they are committed to sharing the scientific results and what has been learned from these clinical trials at an upcoming scientific congress and through www.clinicaltrials.com. This will be an important step for researchers and LuMind RDS to be able to review the results in detail to determine insights and further advancing research to find safe and effective drug therapies to improve cognition in individuals with Ds.”
Although not the result we all desired, there will be important information from the Roche study.
At LuMind RDS, we feel your disappointment about the Roche trial and are seeking answers. Part of that quest is to continue funding other lines of discovery, because like the family in the Fragile X story, we believe we will find safe and effective drug therapies to improve cognition in our loved ones with Down syndrome.
We are in contact with Roche and will share what we learn with you.