Stiris Research Inc. Announces Recruitment Closed in Phase II-III Congenital Myotonic Dystrophy Trial

Stiris Research (“Stiris”) an entrepreneurial clinical trial management company focused on the management of Phase I-IIIB trials, today announces that recruitment has closed on a Phase II/III Congenital Myotonic Dystrophy (CMD) trial.

“We are very excited to announce that recruitment has officially been closed for our Phase II-III Congenital Myotonic Dystrophy study. This is an important and exciting milestone towards a treatment for CMD (DM1) which currently has limited treatment options. We are extremely grateful to all of the subjects, their families/caregivers, and the investigative sites for their collaboration and dedication to the study to date. We look forward to moving towards database lock and receiving the study results which are anticipated in mid-2023!” said Madison Pearce, Project Manager.

Congenital Myotonic Dystrophy (CMD) is a rare early childhood form of myotonic dystrophy (also known as Steinert’s disease) with symptoms generally beginning to show in childhood or later in life, however symptoms of congenital myotonic dystrophy are evident from birth. CMD is generally characterized by diminished muscle tone (hypotonia), progressive muscle weakness and degeneration (atrophy). CMD only occurs when the mother already has myotonic dystrophy (although she may not be aware of this) and passes it on to her child in a more severe form. Myotonic, meaning “involving muscle stiffness” and Dystrophy, meaning “muscle wasting, and weakness” have a devastating impact on these children. CMD varies considerably in severity, symptoms and progression from child to child, but in some cases can be fatal, especially in the early weeks of life (www.musculardystrophyuk.org) and (https://rarediseases.org/rare-diseases/congenital-muscular-dystrophy).