Satellos Bioscience, Inc. is leading a clinical trial to see how safe and effective an oral medication (pill) called SAT-3247 works in children between the ages of 7-9 years of age living with Duchenne muscular dystrophy (DMD).
SAT-3247 is an experimental treatment that may help the body to generate new muscle. It supports special repair cells in the muscle (called satellite cells) and is considered a form of stem cell therapy. SAT-3247 is designed to help muscle cells grow. It works by blocking a protein (called AAK1) that interferes with muscle repair, allowing muscle stem cells to divide normally and make new muscle cells again. Because it works on a process shared by all people with DMD, this treatment could potentially be used regardless of the specific genetic pathogenic variant that causes their DMD.
This study is an early-stage (phase 2a) clinical trial for boys with DMD who can walk and are between 7-9 years of age.
The clinical trial team hopes to enroll approximately 50 children worldwide. Each child will be randomly assigned to receive either SAT-3247 or a placebo (a look-alike pill with no active medical ingredient). This is a double blind study meaning that neither the parents nor the study doctors will know whether the participant is receiving the placebo or the study drug (STA-3247).
Participants will take the STA-3247 once a day on weekdays (for 5 consecutive days) for 12 weeks.
Before starting, children will go through a screening period to make sure the study is a good fit for them. After that, there will be one starting (baseline) visit, one telephone check-in after week 1, and then in-person follow-up visits during week 4, 8, and 12 of the study. Use of steroids and other DMD medications will be noted so that results can be compared fairly.
This trial is currently recruiting participants in several countries, including the U.S., the UK, Europe, Australia, and Serbia. A new site is also opening soon in Ottawa, Canada at the Children’s Hospital of Eastern Ontario. If you are interested in learning more information, or checking to see if your child is eligible, please contact the study coordinator, Emilie Hill-Smith, at [email protected].
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: