ACTIVE_NOT_RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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)

Quick Facts

Study Start:2023-12-12
Study Completion:2025-10
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT06204809

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 60 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT
Inclusion CriteriaExclusion Criteria
  1. * Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
  2. * 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)
  3. * Presence of myotonia
  1. * Congenital DM1
  2. * Known history or presence of any clinically significant conditions that may interfere with study safety assessments
  3. * Abnormal laboratory tests at screening
  4. * Medications specific for the treatment of myotonia within 2 weeks prior to screening
  5. * Percent predicted forced vital capacity (FVC) \<40%

Contacts and Locations

Study Locations (Sites)

UCI Center for Clinical Research
Irvine, California, 92697
United States
Stanford University
Palo Alto, California, 94304
United States
Rare Disease Research
Atlanta, Georgia, 30329
United States
University of Kansas Medical Center
Fairway, Kansas, 66205
United States
Massachusetts General Hospital
Boston, Massachusetts, 02114
United States
University of Rochester Medical Center
Rochester, New York, 14642
United States
Virginia Commonwealth University
Richmond, Virginia, 23298
United States

Collaborators and Investigators

Sponsor: PepGen Inc

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-12-12
Study Completion Date2025-10

Study Record Updates

Study Start Date2023-12-12
Study Completion Date2025-10

Terms related to this study

Keywords Provided by Researchers

  • 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

Additional Relevant MeSH Terms

  • Myotonic Dystrophy 1