Lochmüller Lab

The Lochmüller Lab is excited to share that as of March 14, 2022 our site at the Children’s Hospital of Eastern Ontario (CHEO) has registered its tenth participant for AMO’s Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy (REACH CDM) clinical trial.

This is a significant milestone for our involvement in the trial: through enrolling our tenth participant we have not only met but exceeded our site’s recruitment goal. A special thanks to our clinical research team at CHEO (Emilie Hill-Smith, Laura Thompson, Isabelle Fisette-Paul Hus  and Matthew MacDonald) who are involved in every facet of the site’s clinical work, as well as to the wonderful patients and families who participate.

“We are proud to be a site that is bringing global trials to the Canadian myotonic dystrophy community and are thrilled to see the enthusiasm shown for the REACH CDM trial. Eastern Canada has one of the highest prevalence rates for myotonic dystrophy worldwide. Until now, there have not been any drug trials for children with the severe, congenital form of myotonic dystrophy. This recruitment milestone is a testament to the engagement we see from the Canadian myotonic dystrophy community when trials are available to them and shows the international community that this part of Canada is one of the best in the world to run clinical trials in this rare condition.”

• Dr. Hanns Lochmüller

The Lochmüller Lab at CHEO is one of several trial sites for REACH CDM, with the Children’s Hospital London Health Sciences Centre (LHSC) a second Canadian site, and others actively enrolling patients in the United States, New Zealand, and Australia. In total the REACH CDM study will enroll 56 patients across all sites and has recently passed the 65% threshold for enrollment.

“We are very proud of our progress to date. With 12 global research centers now actively recruiting, we also very much appreciate the support from our outstanding team of investigators as well as the patients and caregivers for their continued commitment to participating in this study despite the challenging circumstances caused by the COVID-19 pandemic.”

• Dr. Michael Snape, Chief Scientific Officer at AMO Pharma

REACH CDM is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1. On completion of the REACH CDM study, all patients have the opportunity to transition to the REACH CDM X study, a 52-week open-label study designed to evaluate the long-term safety and efficacy of AMO-02.

To learn more about the REACH CDM trial including a list of treatment centers, visit www.reachcdm.com.  A list of all clinical trials the Lochmüller Lab is currently involved with is available on the lab website.