The research article, Phase 1 Open-Label Study of Omigapil in Patients With LAMA2- or COL6-Related Dystrophy, summarizing the first-ever CMD clinical trial of a therapeutic small molecule in CMD has been published in the Journal of Neurology Genetics.
"Sending my deepest gratitude to Cure CMD for your support of this work, without which this study would not have been possible! I sincerely hope that the CMD community finds the manuscript to be a tribute to the effort of our wonderful patients with COL6 and LAMA2-related dystrophy, and their families," shares Dr. Reghan Foley, primary author of the paper and clinical researcher who lead the lengthy effort to complete and publish this landmark study.
"The publication of this study is a very important step for: 1) demonstrating that successful recruitment and running of a phase 1 clinical trial in these patient populations is possible and 2) helping the design and running of future clinical trials for incredibly special individuals affected with congenital muscular dystrophy."
While the CMD community was incredibly disappointed that further investigation of omigapil did not continue after this Phase 1 trial, this study has opened doors for future clinical trials in CMD.
Shares Cure CMD's Executive Director, Rachel Alvarez, "It was a real blow to our community when the study’s sponsor did not continue on to a Phase 2 trial to study the drug’s potential efficacy. We can never truly express our appreciation to the affected individuals and their families who participated in this trial, with kiddos enduring long airplane trips, early mornings, needle sticks, and a ban on chocolate (!!) to help ensure this trial was successful. It is important that they know that the lack of a Phase 2 omigapil study is in no way a reflection of their dedication and sacrifice. While omigapil is not currently being studied further, there are other promising treatments actively being investigated. And we've shown the pharmaceutical world that CMD is ready for clinical trials."