At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- PGN-EDODM1 (drug), Placebo (other)
- How long the study runs
- Study runs about 27 months (dates as stated)
- About the drug or intervention
- PGN-EDODM1 — drug: Administered by intravenous (IV) infusion · Placebo — other: Administered by intravenous (IV) infusion
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a medicine called PGN-EDODM1 in people with myotonic dystrophy type 1 (DM1), a condition that affects the muscles. It is run by PepGen Inc. What the study involves and what the medicine does is not stated — ask the trial team.
Who can take part
- A confirmed diagnosis of DM1, based on a genetic test showing at least 100 CTG repeats in the DMPK gene
- Myotonia (muscles that are slow to relax after tightening)
- Enough muscle in both shin muscles (at the front of the lower leg) for a small muscle sample to be safely taken with a needle
- A body mass index (BMI) under 35.0 kg/m²
Who may not be able to
- Congenital DM1 (present from birth)
- Any serious health condition, past or present, that could affect the study's safety checks
- Screening blood or lab tests that the study doctor considers abnormal in an important way
- Taking medicines specifically for myotonia in the 2 weeks before screening
- Breathing test showing less than 40% of the expected amount of air exhaled (forced vital capacity, or FVC)
- Having taken an experimental drug, device, or product within 30 days or 5 half-lives of the study drug (whichever is longer) before screening
What taking part involves
- • Not stated — ask the trial team
Time commitment: The number of visits and how long the study lasts are not stated — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 16 Years to 65 Years
- Who
- All
- Number of participants
- 24
- Started
- 2024-12-10
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for myotonic dystrophy 1
- • Phase2 - 24 participants
- • The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo
Who can take part?
- • Ages 16 Years to 65 Years
- • Diagnosed with myotonic dystrophy 1
Where?
- • Salford - Salford Royal Hospital
- • London - University College London Hospitals NHS Foundation Trust
- • Cardiff - Cardiff and Vale Hospital
- • Newcastle upon Tyne - Newcastle Upon Tyne Hospitals
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 16 Years - 65 Years
- Who can join: All genders
What the study is looking for
- ✓Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
- ✓Presence of myotonia
- ✓Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
- ✓Body Mass Index (BMI) of \< 35.0 kg/m\^2
Who cannot take part
- ✗Congenital DM1
- ✗Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- ✗Abnormal laboratory tests at screening considered clinically significant by the Investigator
- ✗Medications specific for the treatment of myotonia within 2 weeks prior to screening
- ✗Percent predicted forced vital capacity (FVC) \<40%
See the full criteria
Where Is This Study? (4 UK sites)
Salford Royal Hospital
Salford, United Kingdom
University College London Hospitals NHS Foundation Trust
London, United Kingdom
Cardiff and Vale Hospital
Cardiff, United Kingdom
Newcastle Upon Tyne Hospitals
Newcastle upon Tyne NE7 7DN, United Kingdom
How to Get in Touch
PepGen
Sponsor contactCONTACT