This study is in progress, not accepting new patients

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

a study on Myotonic Dystrophy Atrophic Muscular Disorders Neurodegenerative Disease Myopathy

Summary

Eligibility
for people ages 18-60 (full criteria)
Location
at UC Irvine
Dates
study started
study ends around

Description

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).

Official 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)

Keywords

Myotonic Dystrophy 1, Myotonic Dystrophy, Muscular Dystrophies, Genetic Diseases, Inborn, Neuromuscular Diseases, Nervous System Diseases, Musculoskeletal Diseases, Myotonic Disorders, Muscular Disorders, Atrophic, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases, Muscular Diseases, Steinert Disease, Inborn Genetic Diseases, Atrophic Muscular Disorders, Nervous System Heredodegenerative Disorders, PGN-EDODM1 for infusion, PGN-EDODM1

Eligibility

You can join if…

Open to people ages 18-60

  • 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

You CAN'T join if...

  • 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

  • UCI Center for Clinical Research
    Irvine 5359777 California 5332921 92697 United States
  • Stanford University
    Palo Alto 5380748 California 5332921 94304 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
PepGen Inc
ID
NCT06204809
Phase
Phase 1 research study
Study Type
Interventional
Participants
About 24 people participating
Last Updated