A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)

PepGen Inc12 sites in 3 countries24 target enrollmentDecember 12, 2023

ConditionsMyotonic Dystrophy 1

InterventionsPGN-EDODM1 for infusion Placebo

Overview

Phase: Phase 1
Intervention: PGN-EDODM1 for infusion
Conditions: Myotonic Dystrophy 1
Sponsor: PepGen Inc
Enrollment: 24
Locations: 12
Primary Endpoint: Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
Status: Completed
Last Updated: 2 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).

The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).

Registry

clinicaltrials.gov

Start Date

December 12, 2023

End Date

October 28, 2025

Last Updated

2 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

PepGen Inc

Responsible Party

Sponsor

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.

Arms & Interventions

PGN-EDODM1

PGN-EDODM1 for infusion

Intervention: PGN-EDODM1 for infusion

Placebo

0.9% NaCl

Intervention: Placebo

Outcomes

Primary Outcomes

Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs

Time Frame: Baseline to Week 16

Secondary Outcomes

Area Under the Concentration-time Curve of PGN-EDODM1(Baseline up to Day 3)
Apparent Terminal Half-Life (t½) of PGN-EDODM1(Baseline up to Day 3)
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1(Baseline up to Day 3)
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1(Baseline up to Day 3)