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NCT Number: NCT06667453

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

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.

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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

16 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

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
  • Presence of myotonia
  • Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
  • Body Mass Index (BMI) of < 35.0 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.

Treatment and study plan

PGN-EDODM1

Drug

Administered by intravenous (IV) infusion

Placebo

Other

Administered by intravenous (IV) infusion

Primary outcomes

  1. Safety and tolerability as assessed by number of participants with Adverse Events (AEs)

    Time frame: Baseline through Day 112

Secondary outcomes

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

    Time frame: Day 1 through Day 84

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

    Time frame: Day 1 through Day 84

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

    Time frame: Day 1 through Day 84

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

    Time frame: Day 1 through Day 84

  5. Change in splicing index in skeletal muscle tissue

    Time frame: Baseline through Day 91

  6. Change in myotonia as measured by video Hand Opening Time (vHOT)

    Time frame: Baseline through Day 112

  7. Hand grip strength

    Time frame: Baseline through Day 112

    by dynamometer

  8. Change in mobility as measured by 10 meter walk/run time

    Time frame: Baseline through Day 112

Study contacts

Contact information is provided by the study sponsor or research team.

PepGen

CONTACT

[email protected]

781-797-0979

Sponsors and collaborators

Lead sponsor

PepGen Inc

Industry

Registry information

Official study title

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)

Acronym: FREEDOM2-DM1

Important dates

Study start
2024
Primary completion
2027
Study completion
2027
First posted
Oct 31, 2024
Registry last updated
Apr 28, 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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