22/05/2024
Ottawa, Ontario — Wednesday May 22, 2024
A new drug for children and youth with Congenital myotonic dystrophy (CDM), a rare genetic disorder, is showing promise following a clinical trial led by CHEO researchers. Dr. Hanns Lochmüller, Senior Scientist at the CHEO Research Institute (CHEO RI), recently presented the results of the AMO Pharma Phase 2/3 clinical study, REACH CDM, at an international conference in Nijmegen, The Netherlands (IDMC 2024). The study involved children and adolescents with CDM, a rare genetic disorder presenting with muscle weakness in infants and causing issues in breathing, sucking and swallowing. As people affected by CDM approach adulthood, they display the progressive features of the adult-onset form of the disease, which affects multiple body systems including muscle wasting, stiffness, and cardiac arythmia.
The drug known as tideglusib provided clinically significant improvements in walking, cognition, and activities of daily living and was generally safe and well tolerated, with no reported severe adverse events, but did not reach significance in the primary endpoint, an 11-item rating scale.
“The results of the REACH CDM Study showed measures of efficacy benefit across multiple symptom areas, and we are very excited that this clinical development program will continue to move forward,” said Dr. Lochmuller, lead investigator in the REACH CDM Clinical Study at CHEO. “We remain very hopeful that this investigational therapy can have a transformative impact on the health of patients living with this ultra-rare, severe muscular dystrophy in the years ahead.“
Clinical research trials are situated at the forefront of medicine, with new discoveries and treatments rapidly advancing the prospect of therapeutics for previously untreatable diseases. CHEO clinical researchers conduct trials to help to build the critical body of evidence for efficacy and approval of new therapies and play an instrumental role in providing Canadian patients with pathways to access cutting-edge treatments.
Based on the positive study outcomes of the REACH CDM Phase 2/3 study, AMO Pharma recently announced plans to conduct a Phase 3 study in adults to support a future submission for drug approval in children, adolescents and adults affected by Myotonic dystrophy.
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.
May 20 is Clinical Trials Day. The CHEO RI is home to over 80 ongoing regulated trials to provide better care for children and youth in our community and beyond!