A Clinical Study of PGN-EDODM1 in People with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)

PepGen

Status and phase: Enrolling; Phase 2

Conditions: Myotonic Dystrophy 1

Treatments: Other: Placebo; Drug: PGN-EDODM1

Study type: Interventional

Funder types: Industry

Identifiers

NCT06667453

PGN-EDODM1-102

Details and patient eligibility

About

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.

Enrollment: 24 estimated patients

Sex: All

Ages: 16 to 60 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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24 participants in 2 patient groups, including a placebo group

PGN-EDODM1

Experimental group

Description:

Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks

Treatment:

Drug: PGN-EDODM1

Placebo

Placebo Comparator group

Description:

Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks

Treatment:

Other: Placebo