Clinical Trials Logo
  1. Home
  2. Myotonic Dystrophy 1
  3. NCT06204809

Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 — FREEDOM-DM1  

Myotonic Dystrophy 1 Clinical Trial

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)

Clinical Trial 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).

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06204809
Study type Interventional
Source PepGen Inc
Contact PepGen
Phone 781-797-0979
Email clinicaltrials@pepgen.com
Status Recruiting
Phase Phase 1
Start date December 12, 2023
Completion date April 2025
View Details
See also
  Status Clinical Trial Phase
Recruiting NCT02880735 - Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy N/A
Recruiting NCT06378216 - Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]
Active, not recruiting NCT06089018 - Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy
Completed NCT05662150 - Low-frequency Repetitive Nerve Stimulation in Myotonic Dystrophy Type 1 N/A
Active, not recruiting NCT04698551 - NIPD on cffDNA for Triplet Repeat Diseases
Completed NCT05027269 - Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients Phase 1/Phase 2
Completed NCT04712422 - Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
Recruiting NCT05006924 - Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
Recruiting NCT04656210 - Myotonic Dystrophy - Vascular and Cognition
Completed NCT04634682 - Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1 N/A
Withdrawn NCT06270186 - Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach N/A
Not yet recruiting NCT06300307 - Study of ATX-01 in Participants With DM1 Phase 1/Phase 2
Completed NCT02729597 - Tracking the Brain in Myotonic Dystrophies: a 5-year Longitudinal Follow-up Study N/A
Recruiting NCT06138743 - Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Phase 1
Not yet recruiting NCT05532813 - Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) Phase 3
Active, not recruiting NCT04886518 - Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 Phase 2
Completed NCT05036447 - Myotonic Dystrophy Type 1 and Resistance Exercise N/A
Recruiting NCT03424460 - Venous Thromboembolism in Myotonic Dystrophy Type 1 N/A
Recruiting NCT05865483 - Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)
Completed NCT04001920 - Effects of a Multiple Component Training Program on Muscles in Adults With Myotonic Dystrophy Type 1 N/A