Are you living with Duchenne muscular dystrophy (Duchenne)?

Consider joining the CONNECT2-EDO51 study, which is researching an investigational drug, called PGN-EDO51, to see if it is safe and tolerable for boys and men with Duchenne. PGN-EDO51 is an investigational drug, meaning that it has not been approved for any use by any health authorities, such as the US Food and Drug Administration (FDA) or The European Medicines Agency (EMA).

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Are you living with Duchenne muscular dystrophy (Duchenne)?

Consider joining the CONNECT2-EDO51 study, which is researching an investigational drug, called PGN-EDO51, to see if it is safe and tolerable for boys and men with Duchenne. PGN-EDO51 is an investigational drug, meaning that it has not been approved for any use by any health authorities, such as the US Food and Drug Administration (FDA) or The European Medicines Agency (EMA).

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What is the CONNECT2-EDO51 study?

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).

Who can join the CONNECT2-EDO51 study?

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.

What will happen during the study?

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:

Review & Sign the Informed Consent Form

The Informed Consent Form (ICF) contains information about the study including its goals, duration, benefits, risks, tests and procedures.

Screening Period

Receive study health assessments to confirm if you qualify for the study.

Study Treatment Period

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.

Follow-up Visit

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.

Roll-over into Open-Label Long-Term Extension

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.

If you have additional questions about participating in this clinical research study, please send an email to clinicaltrials@pepgen.com to learn more.

About the Study Drug

What is the study drug?

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.

Will I receive the study drug?

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.

What is an “investigational drug?”

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.

What is Duchenne Muscular Dystrophy (Duchenne)?

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.

Where can I learn more about Duchenne?

Overview of Clinical Research Studies

Here are some common questions and answers about study participation.

If you have additional questions about participating in this clinical research study, please send an email to clinicaltrials@pepgen.com.

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