Statement from LuMind Research Down Syndrome Foundation on the Roche Clinical Trials

Categories: Blog, Clinical Trials, Ds Research, News

From LuMind RDS Board Chairman, Ryan Hartman

RocheWe thank you for turning to LuMind Research Down Syndrome with your questions and comments about the news of the Roche CLEMATIS Phase 2 clinical trial for RG1662/Basmisanil. As the largest source of private funding for Down syndrome cognition research, we are in contact with many people from our community as well as representatives from Roche.

We want the Down syndrome community and especially those parents who saw changes in their children, to know we are listening to you, hearing you, and are working with Roche to understand the path forward.

Earlier this week, Roche issued a statement explaining that upon the completion of the clinical trial, which was conducted on people with Down syndrome, ages 12-30, there were no differences between individuals receiving RG1662 and individuals receiving the placebo. Roche went on to state that because of the results, including agreement within the younger 12-17 year old age range, they were halting the RG1662 Phase 2 clinical trial in individuals with Ds ages 6-11 years old.

We are hearing from those of you who had loved ones participating in the clinical trials that you saw different results – especially in children in the 6-11 age range.

First, we feel compelled to clarify our relationship with Roche and the CLEMATIS clinical trials. We fund research at leading universities and help these labs demonstrate viable discoveries to firms such as Roche. While LuMind RDS funded fundamental and translational research that led the way to the opportunity for clinical trials, we are not financially invested in the Roche clinical trials (we have not provided funds nor have been given funds); we are not currently privy to the results, or hold any patents. Our work with biopharma firms typically involves engaging with leadership in biopharma companies and communicating research results hoping to bring these exciting opportunities to the attention of the firms, who then invest time and money in the next phases of the pipeline – development and clinical trials.

Soon after Roche issued its statement, representatives of LuMind Research Down Syndrome were on a call with members of the CLEMATIS team at Roche. The Roche representatives described the preliminary results and decisions concerning the RG1662/Basmisanil clinical trials.

According to LuMind RDS’s Chief Scientific Officer Dr. Michael Harpold, who was on the call with Roche and is familiar with the study, the double blind research protocol used is the “gold-standard” for clinical trials. With a double blind protocol, none of the participants and their families, researchers, or clinicians involved in the trial know which participants receive the study drug or the placebo during the trials. Typically, this information is “unblinded” following the completion of the trial so that the researchers can begin the analyses of the trial results. He further explained that while he too is disappointed in the results, we won’t know what lessons can be learned from the study until the “unblinded” data and the results are fully analyzed and presented.

Roche also stated their continued commitment to making the data and results available to the wider research community, including LuMind RDS. The release of this information is necessary to assess why efficacy was not detected, formulate potential appropriate ways any reasons might be further addressed (including through additional research), and ultimately apply this knowledge to other Ds clinical trials. But analysis of the full data for presentation will take time.

We absolutely understand that asking for your patience is not a satisfying response, especially when many of you indicated you saw responses.

On the call, the Roche representatives expressed tremendous gratitude to the participants and the Ds community for their support and making the trials possible. They emphasized this was the largest clinical trial in individuals with Ds conducted to date and provided strong proof that such trials were possible – a critical accomplishment for the Ds community.

We are also mindful of the significant investment of time and money firms like Roche make in the pursuit of these drug therapies, developing the drugs, and running clinical trials. Without these biopharma firms believing in our vision, in most instances the research we fund would remain in the laboratories and never come to reality.

While we share your disappointment in the news from the CLEMATIS study, together we are pioneering research that will ultimately lead to an effective treatment for cognition and memory dysfunction in our loved ones with Down syndrome. As pioneers charting new grounds, we understand that along the way we will stumble. It is our responsibility to our loved ones and to the parents and families who support us that we learn from these challenges so the next opportunity is even more successful. So we will continue working with the Principal Investigators and Roche to ensure that the investment in this trial is a major contributor to the next trial.

We will continue to keep you informed of any further developments as they evolve. Thank you.