Join us at Health Research Day — June 6th at Canton Waterfront Park, Baltimore!   Learn More →
← Back to all trials
Recruiting NCT06667453

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

Conditions: Myotonic Dystrophy 1

Sex: All
Ages: 16 Years – 65 Years
Healthy volunteers: No
Phase: PHASE2
Enrollment: 24
Sponsor: PepGen Inc

Location: University of Calgary Calgary Alberta

Summary

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.

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 * 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) \

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT06667453). StuddyBuddy aggregates publicly available trial information.