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

Phase 1CompletedNCT06204809

PepGen Inc24 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Myotonic Dystrophy 1

Interventions

PGN-EDODM1 for infusionDRUG

Single dose of PGN-EDODM1 by intravenous (IV) infusion

PlaceboOTHER

Administered by IV infusion

Eligibility

Sex: ALLMin age: 18 YearsMax age: 60 Years

Medical Language ↔ Plain English

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.

Locations (12)

UCI Center for Clinical Research

Irvine, California, United States

Stanford University

Palo Alto, California, United States

Rare Disease Research

Atlanta, Georgia, United States

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

Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1

Time frame: Baseline up to Day 3

Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1

Time frame: Baseline up to Day 3

Apparent Terminal Half-Life (t½) of PGN-EDODM1

Time frame: Baseline up to Day 3

Area Under the Concentration-time Curve of PGN-EDODM1

Time frame: Baseline up to Day 3

Data from ClinicalTrials.gov

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