Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1

Launched by PEPGEN INC · Jan 3, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called PGN-EDODM1 for people with Myotonic Dystrophy Type 1 (DM1). The main goal is to see how safe the treatment is and how well people tolerate it when given as a single intravenous (IV) dose. Participants will go through a screening period of up to 30 days to determine their eligibility, followed by a treatment and observation period lasting 16 weeks.

To be eligible for this trial, participants must have a confirmed diagnosis of DM1, which means they have a specific genetic change in their DMPK gene, and they need to be able to move their ankle muscles with a certain level of strength. People with congenital DM1 or certain other medical conditions may not qualify. Throughout the trial, participants will closely monitor their health and any side effects from the treatment. This study is currently recruiting participants aged 18 to 62, regardless of gender, and aims to better understand how PGN-EDODM1 can help manage the symptoms of DM1.

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
• 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
• • Medications specific for the treatment of myotonia within 2 weeks prior to screening
• • Percent predicted forced vital capacity (FVC) \<40%
• • Note: Other inclusion and exclusion criteria may apply.