Investigating PGN-EDODM1 for Myotonic Dystrophy Type 1

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical study evaluates the safety and tolerability of an investigational medication, PGN-EDODM1, in individuals diagnosed with myotonic dystrophy type 1 (DM1). Participants will receive either PGN-EDODM1 or a placebo in multiple administrations to assess the drug's effects. The study aims to determine how well the medication is tolerated compared to a control group, focusing on individuals with specific muscle function criteria and genetic markers associated with DM1.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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

Exclusion Criteria:

* 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%
* Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening

Note: Other inclusion and exclusion criteria may apply.

Where it's running

• University of Calgary
  Calgary, Alberta, Canada Recruiting
• Ottawa Hospital Research Institute (OHRI)
  Ottawa, Ontario, Canada Recruiting
• CIUSSS du Saguenay-Lac-Saint-Jean
  Chicoutimi, Quebec, Canada Recruiting
• Montreal Neurological Institute
  Montreal, Quebec, Canada Recruiting
• Pacific Clinical Research Network Auckland
  Takapuna, Auckland, New Zealand Recruiting
• Pusan National University Yangsan Hospital
  Pusan, Gyeongsangnam-do, South Korea Recruiting

Study contacts

• Study coordinator: PepGen
• Email: clinicaltrials@pepgen.com
• Phone: 781-797-0979

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Understanding long-term progression in myotonic dystrophy type 1 (DM1)

  This study will follow adults with DM1 for at least four years to see how walking, hand function, muscle strength, heart rhythms, and lung function change over time.

  Recruiting Virginia Commonwealth University · Richmond, Virginia · NCT07700225

• Spanish National Registry for Myotonic Dystrophy Type 1

  This registry gathers health, clinical, and genetic information from people in Spain with DM1 to see how the condition changes over time and help match patients to future trials.

  Recruiting at 8 sites Fundació Institut Germans Trias i Pujol · Multiple Locations, Andalusia and 7 other locations · NCT07385443

• Long-term PGN-EDODM1 extension for people with myotonic dystrophy type 1

  This open-label extension will try continued PGN-EDODM1 in people with myotonic dystrophy type 1 who completed a prior PGN-EDODM1 study to see how safe and tolerable it is over time.

  Recruiting at 3 sites Phase 2 PepGen Inc · Calgary, Alberta and 2 other locations · NCT07220603

• Wearable monitoring of walking and arm movement in adults with neuromuscular or metabolic movement disorders

  This project uses a wrist/ankle wearable to continuously monitor walking and upper-limb movement in ambulant adults with neuromuscular diseases or obesity to see if digital measures like stride speed track real-life motor function.

  Recruiting Centre Hospitalier Universitaire de Liege · Liège · NCT07136844

• Testing ATX-01 for myotonic dystrophy type 1

  This study is testing a new drug called ATX-01 to see if it is safe and well-tolerated in people with myotonic dystrophy type 1.

  Recruiting at 12 sites Phase 1/2 ARTHEx Biotech S.L. · Los Angeles, California and 11 other locations · NCT06300307

• Collecting data on muscle disorders using wearable devices

  This study is testing a wearable device to track movement in people with muscle disorders like Duchenne Muscular Dystrophy and Myotonic Dystrophy to see how it can help improve their care.

  Recruiting at 2 sites Centre Hospitalier Universitaire de Liege · Liège and 7 other locations · NCT05982119

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.