Our genes provide instructions (genetic code) to our cells to make proteins that play important roles in our bodies. People with Duchenne have a mutation (a change) in the genetic code for dystrophin protein. That means that no dystrophin is produced. Without working dystrophin protein, the muscles break down losing strength and function over time. Over time, the heart is also affected in people with Duchenne, a common cause of early mortality.
PGN-EDO51 is a type of investigational drug called an exon skipping agent that is designed to correct the underlying cause of Duchenne. PGN-EDO51 was designed to help cells “skip” over a segment (called an exon) of the transcript (section of mRNA) that codes for dystrophin protein so that the body can create a shorter, functional dystrophin protein. PGN-EDO51 was designed to enter cells efficiently, potentially improving potency over the first generation Duchenne oligonucleotide therapies.
In this study, PGN-EDO51 will be given via intravenous (IV) infusion every 4 weeks; 4 doses in the first part of the study, 27 doses in the long-term extension for a total of 31 doses over a period of 2.5 years. Each infusion takes about an hour. The study staff will observe you after each infusion. You may be asked on certain visits to stay overnight at or near the study clinic prior to and/or after an infusion.
This is an open-label study, meaning that everyone who agrees to take part in the study and meets the required criteria will receive PGN-EDO51. All study participants will receive support and monitoring by a qualified healthcare team over a participation period of about 2.5 years.
An investigational drug is a substance that is being tested and may or may not be approved by regulatory authorities, like the US Food and Drug Administration (FDA) or Health Canada, for treatment of this condition. It can only be used in clinical research studies like the CONNECT1-EDO51 study.