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

Launched by PEPGEN INC · Oct 29, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new medicine called PGN-EDODM1 to see how safe it is and how well it works for people with Myotonic Dystrophy Type 1 (DM1). Myotonic dystrophy is a genetic condition that affects muscle function and can cause weakness and stiffness. The study will compare the effects of PGN-EDODM1 to a placebo, which is a treatment that looks like the medicine but has no active ingredients. The trial is currently looking for participants aged 18 to 75 who have been diagnosed with DM1 and meet certain health criteria.

Participants in the study can expect to receive the investigational medicine multiple times while being closely monitored by healthcare professionals. To be eligible, they must have a confirmed diagnosis of DM1, show some ability to move their muscles, and have a specific body mass index. However, individuals with certain other health conditions or who are currently taking specific medications may not be able to join. This trial aims to gather important information about how this new treatment might help those living with myotonic dystrophy.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
• • Medical Research Council (MRC) score of ≥ Grade 4 in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
• • Presence of myotonia
• • Body Mass Index (BMI) of \< 32.0 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.