The CONNECT2-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.
For the first 6 months of the study, you will be randomly assigned to receive monthly IV infusions (through a needle in a vein) of either PGN-EDO51 or placebo. The placebo will look like PGN-EDO51 but does not contain any active medicine. You will have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during these 6 months. During the rest of the study (about 2 years), you will receive PGN-EDO51 only (no placebo).
Study participants may be able to join the study if they meet the following requirements:
Male by birth and at least 6 years old
Diagnosed with Duchenne amenable to exon 51 skipping (genetic testing will be required to join)
Weigh at least 25 kg (about 55 lbs)
Willing to have a total of two open muscle biopsies (to collect muscle tissue samples)
Other study requirements will apply. If you have additional questions about participating in this clinical research study, please send an email to clinicaltrials@pepgen.com to learn more.
Participants will be compensated for their study visit time and reimbursed for travel expenses.
Participation in the CONNECT2-EDO51 double-blind, randomized, placebo-controlled part of the study lasts about 6 months and involves up to 16 visits to the study site. There is also an open-label 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. If you complete the entire study your participation will last approximately 2.5 years. You will have a total of 2 muscle biopsies over the course of the study if you complete the entire study. Study participants can expect the following:
The Informed Consent Form (ICF) contains information about the study including its goals, duration, benefits, risks, tests and procedures.
Receive study health assessments to confirm if you qualify for the study.
If you qualify and enroll, you will receive monthly infusions of either PGN-EDO51 or placebo for about 6 months (total of 7 infusions). You will have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during these 6 months.
You will receive regular study assessments throughout the study. These assessments may include muscle exercises to check how your muscles are working. You will also have blood and urine tests, a heart test (ECG), a breathing test (spirometry), and 2 open muscle biopsies, and you will be asked to fill in questionnaires.
You will have a follow-up visit once the Treatment Period is complete to check your overall health and complete some assessments. This will be your final visit in the first part of the study.
You will have the choice, if you are eligible, to continue in a long-term extension period during which you will receive monthly infusions of PGN-EDO51 (no placebo) for 2 more years.
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.
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 first generation oligo therapies.
For the first 6 months of the study during the double-blind period, PGN-EDO51 or placebo will be given via intravenous (IV) infusion every 4 weeks for a total of 7 doses during this period. In the open-label long-term extension period, a total of 27 doses of PGN-EDO51 (no placebo during this period) will be given via IV infusion every 4 weeks over a period of about 2 years. Each infusion takes about an hour. The study staff will observe you after each infusion. You may be asked to stay overnight at or near the study clinic after an infusion.
This is a double-blind, randomized, placebo-controlled study, meaning that both you and the study doctor and research staff will not know if you are receiving PGN-EDO51 or placebo during the first part of the study, which lasts about 6 months. You will have a 75% chance of receiving PGN-EDO51 and 25% chance of receiving placebo (no active drug) during the first part of the study. After that period, if you are eligible, you will have the option to continue into the open-label long-term extension part for the study where all participants will receive PGN-EDO51 (no placebo) for a period of 2 years.
All study participants will receive support and monitoring by a qualified healthcare team over a participation period of about 2.5 years if they complete the entire study.
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 The European Medicines Agency (EMA), for treatment of this condition. It can only be used in clinical research studies like the CONNECT2-EDO51 study.
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 (or change) in the genetic code for dystrophin protein. That means that no dystrophin is produced. Without functional dystrophin protein, the muscles break down and strength is lost over time. Over time, the heart is also affected in people with Duchenne, a common cause of early mortality.
Here are some common questions and answers about study participation.
Clinical research studies are designed to answer specific questions about the safety or effectiveness of investigational drugs, vaccines, other experimental approaches, or new ways of using existing treatments. Studies are important for medical advancement. Current treatments for diseases and conditions are only available because of study volunteers, and what we learn from the CONNECT2-EDO51 study could help accelerate the development of new therapies and inform future research, which may help the Duchenne community in the future.
Before enrolling in a clinical trial, you must sign an Informed Consent Form (ICF). The ICF contains information about the study, including study goals, how long the study will last, benefits and risks, and the tests and procedures you will receive.
Study participation usually involves visiting a clinic regularly, taking or receiving an investigational medicine or placebo, and having assessments done to monitor your health. More details about specific study participation can be provided to you by the site study staff or investigator.
Participation in clinical research studies is your choice, and you may stop at any time. Talk to your doctor for additional information on clinical research studies and to learn more about eligibility for available studies like the CONNECT2-EDO51 Study.
Costs associated with the study, including costs for the investigational drug, study supplies, study visits, and/or necessary tests, are covered. You will be compensated for your study visit time and will be eligible for reasonable travel expense reimbursement.
You will play an active role in your own health care, will receive frequent health check-ups as part of your participation, and may help others by contributing to what we know about Duchenne. As the therapy is investigational, its potential benefits are still being studied.
If you have additional questions about participating in this clinical research study, please send an email to clinicaltrials@pepgen.com.