RecruitingPhase 2NCT06667453

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

A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)

Sponsor

PepGen Inc

Enrollment

24 participants

Start Date

Dec 10, 2024

Study Type

INTERVENTIONAL

Conditions

Myotonic Dystrophy 1

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

Min Age: 16 YearsMax Age: 60 Years

Inclusion Criteria4

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 Criteria6

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

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