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OpenTrials
Completed

NCT Number: NCT06204809

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

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).

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Key information

Conditions

Myotonic Dystrophy 1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Myotonic Disorders Myotonic Dystrophy Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases

Age range

18 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Calgary, Calgary, Alberta, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

PGN-EDODM1 for infusion

Drug

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

Placebo

Other

Administered by IV infusion

Primary outcomes

  1. 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

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

    Time frame: Baseline up to Day 3

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

    Time frame: Baseline up to Day 3

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

    Time frame: Baseline up to Day 3

  4. Area Under the Concentration-time Curve of PGN-EDODM1

    Time frame: Baseline up to Day 3

Sponsors and collaborators

Lead sponsor

PepGen Inc

Industry

Registry information

Official study title

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)

Acronym: FREEDOM-DM1

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Jan 12, 2024
Registry last updated
Feb 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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