The CONNECT1-EDO51 clinical trial is a research study for people who are living with Duchenne muscular dystrophy (Duchenne). The study will test whether an investigational drug, called PGN-EDO51, is safe and tolerable for boys and men with Duchenne. CONNECT1-EDO51 is an open-label study, meaning that everyone who agrees to take part in the study and meets the required criteria to participate will receive PGN-EDO51.
Study participants may be able to join the study if they meet the following requirements:
Other study requirements will apply.
The first part of the CONNECT1-EDO51 study will have 10 study visits over a period of 3 months. Up to 3 of these study visits may occur at your home rather than at the study clinic.
The second part of the study is a long-term extension which will last about 2 years (108 weeks) that you can choose to join once you complete the first part of the study and confirm eligibility.
If you complete both parts of the study your participation will last approximately 2.5 years.
You will have a total of 2 muscle biopsies if you complete the entire study.
If you are interested in participating, the study team will schedule a screening visit to see if you qualify for the study. At this visit you will:
If you qualify and choose to enroll in the study, you will receive the study drug, PGN-EDO51, every 4 weeks for a total of 4 doses in the first part of the study. If you choose to join the long-term extension after completing the first part of the study, you will continue to receive the study drug every 4 weeks for approximately 2 years. During the long-term extension period, you will receive an additional 27 doses of PGN-EDO51, which may be given at the clinic or at home.
Participants will be compensated for their study visit time and reimbursed for travel expenses.
Participation in a clinical study is voluntary. You can ask any questions you have and may leave the study at any time, for any reason.
If you have additional questions about participating in this clinical research study, please send an email to clinicaltrials@pepgen.com to learn more.