RecruitingPHASE2INTERVENTIONAL

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

This study is designed to explore a new potential medicine, PGN-EDODM1, for individuals living with Myotonic Dystrophy Type 1 (DM1). The main goal is to understand how safe the medicine is and if people can tolerate it well when given multiple times. To do this, some participants will receive PGN-EDODM1, while others will receive a placebo – a dummy medicine that looks the same but contains no active drug. This comparison helps researchers see if any changes are due to the new medicine. The study is open to both men and women between the ages of 16 and 65 who have been diagnosed with DM1.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

PepGen Inc

Enrolment target

Start

10 Dec 2024

Estimated completion

01 Mar 2027

Myotonic Dystrophy 1

What is this study about?

You might have heard about clinical trials, which are research studies that test new medicines to see if they are safe and effective. This particular study is investigating a new medicine called PGN-EDODM1. It's being tested for a condition called Myotonic Dystrophy Type 1, often shortened to DM1. The main purpose of this study is to learn more about this experimental medicine, focusing on its safety and how well people can tolerate it. By 'tolerating it well,' we mean if people experience side effects and how manageable those side effects are, if any occur.

In this type of study, often called a 'Phase 2' study, a group of people will receive the new medicine, PGN-EDODM1, while another group will receive a 'placebo.' A placebo looks exactly like the study medicine but doesn't contain any active ingredients. Comparing the effects in both groups helps the research team understand if any changes observed are due to the actual medicine or other factors. This is a common and important step in developing new treatments.

Understanding the safety of any new medicine is the most important thing. This study will carefully monitor participants to see how their bodies react to PGN-EDODM1. The information gathered will help doctors and scientists decide if this medicine should be studied further in future, larger trials. Your participation could help advance our understanding of DM1 and potentially lead to new treatment options down the line.

Key takeaways

This study is testing a new medicine (PGN-EDODM1) for Myotonic Dystrophy Type 1 (DM1).
Its main goal is to check the medicine's safety and how well people tolerate it.
Some participants will get the actual medicine, others a placebo (dummy medicine).
It's open to adults aged 16 to 65 with a confirmed DM1 diagnosis.
Participation involves clinic visits, tests, and potentially a muscle biopsy.
Your involvement could help advance future treatments for DM1.

Who may be eligible?

To join this study, doctors will first check if you meet certain requirements. You must have a confirmed diagnosis of Myotonic Dystrophy Type 1 (DM1). This means tests have shown a specific genetic change (at least 100 CTG repeats in the DMPK gene). You also need to experience muscle stiffness, a common symptom of DM1, known as myotonia. Your body mass index (BMI) should be less than 35.0 kg/m², which is a measure relating your weight to your height.

You also need to have enough muscle in a specific part of your lower leg (called the tibialis anterior muscle) for a small biopsy (a tissue sample) to be taken safely. This is important for the research team to understand the effects of the medicine.

There are also some reasons why you might not be able to join. For example, if you were born with congenital DM1 (a severe form of DM1 present from birth) or have other significant health problems that could make your participation unsafe or interfere with the study's results. If you've recently used other experimental drugs or certain medications for myotonia, you might also need to wait a period before joining.

Quick self-check

I have a definite diagnosis of Myotonic Dystrophy Type 1.
I have muscle stiffness (myotonia).
My age is between 16 and 65 years old.
I do not have congenital DM1 (DM1 that started at birth).
I haven't used experimental drugs recently (within the last 30 days or longer for some drugs).
My doctor confirms I don't have other major health issues that would interfere with the study.

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you would receive either the investigational medicine PGN-EDODM1 or a placebo (a dummy treatment). Exactly how many times and how often you would receive it would be explained to you. You would have several visits to the study clinic for various assessments. These assessments might include physical exams, blood and urine tests, heart checks, and other evaluations to monitor your health and how your body is reacting to the medicine.

One part of the study involves a small muscle biopsy from your lower leg. This is a procedure where a tiny piece of muscle tissue is carefully removed for examination. Throughout the study, the research team will keep a close eye on any side effects you might experience and your overall well-being. The total duration of your participation in the study, including all visits and follow-up, would be clearly explained by the study team.

Potential risks and benefits

Taking part in any clinical trial involves both potential benefits and risks. You might not directly benefit from this study, as it's designed to gather information about the new medicine rather than being a guaranteed treatment. However, your participation could help researchers learn more about DM1 and potentially help others in the future. Potential risks could include side effects from the study medicine or discomfort from procedures like blood draws or the muscle biopsy. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (8)

University of Calgary
Calgary, Canada· Recruiting
Ottawa Hospital Research Institute (OHRI)
Ottawa, Canada· Recruiting
CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Canada· Recruiting
Montreal Neurological Institute
Montreal, Canada· Recruiting
Pacific Clinical Research Network Auckland
Takapuna, New Zealand· Active not recruiting
Salford Royal Hospital
Salford, United Kingdom· Recruiting
University College London Hospitals NHS Foundation Trust
London, United Kingdom· Recruiting
Newcastle Upon Tyne Hospitals
Newcastle upon Tyne, United Kingdom· Recruiting

Common questions

What is Myotonic Dystrophy Type 1?

Myotonic Dystrophy Type 1 (DM1) is a genetic condition that causes muscles to weaken and waste away over time. It can affect many parts of the body.

What is a placebo?

A placebo is a substance or treatment that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers see if the real medicine is having an effect.

Will I know if I'm getting the study medicine or the placebo?

In this type of study, participants usually don't know whether they are receiving the active medicine or the placebo. This helps prevent bias in the results.

What is a muscle biopsy?

A muscle biopsy is a small procedure where a tiny piece of muscle tissue is removed, usually with a needle, so doctors can examine it more closely.

Can I leave the study at any time?

Yes, absolutely. Your participation is completely voluntary, and you can choose to leave the study at any point without it affecting your ongoing medical care.

How to find out more

PepGen

clinicaltrials@pepgen.com 781-797-0979 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.