PGN-EDODM1 for Myotonic Dystrophy
(FREEDOM-DM1 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing the safety and tolerability of a drug called PGN-EDODM1, given through an IV, in people with Myotonic Dystrophy Type 1 (DM1). The drug aims to address the underlying issues causing muscle problems in these patients. The study includes initial assessments followed by a period of receiving the drug and monitoring.
Will I have to stop taking my current medications?
The trial requires that you stop taking medications specifically for the treatment of myotonia at least 2 weeks before the screening period.
How is the drug PGN-EDODM1 different from other drugs for myotonic dystrophy type 1?
What data supports the effectiveness of the drug PGN-EDODM1 for Myotonic Dystrophy?
Research on similar treatments, like antisense oligonucleotides (ASOs), shows they can improve muscle issues in myotonic dystrophy by targeting specific genetic problems. Combining ASO treatment with exercise has been shown to reduce fatigue in mice with myotonic dystrophy, suggesting potential benefits for similar therapies.46789
Are You a Good Fit for This Trial?
This trial is for individuals with Myotonic Dystrophy Type 1. Participants must be adults who meet specific health criteria during the screening period, which lasts up to 30 days before starting treatment.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment and Observation
Participants receive single intravenous (IV) doses of PGN-EDODM1 or placebo and are observed for safety and tolerability
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- PGN-EDODM1
- Placebo
Find a Clinic Near You
Who Is Running the Clinical Trial?
PepGen Inc
Lead Sponsor