PGN-EDODM1 for Muscular Dystrophy
(FREEDOM2-DM1 Trial)
Trial Summary
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 screening. For other medications, the protocol does not specify, so it's best to discuss with the study team.
What data supports the effectiveness of the drug PGN-EDODM1 for Muscular Dystrophy?
Research on similar treatments, like eteplirsen, shows that increasing dystrophin production can help slow down muscle decline in Duchenne muscular dystrophy. Additionally, studies on utrophin modulation suggest that enhancing related proteins can improve muscle function and stability, which might be relevant for PGN-EDODM1.12345
How is the drug PGN-EDODM1 different from other treatments for muscular dystrophy?
PGN-EDODM1 is unique because it targets the prostaglandin D2 (PGD2) pathway, which is involved in muscle necrosis in Duchenne muscular dystrophy (DMD). This approach is different from other treatments that focus on gene therapy or exon skipping, as it aims to reduce inflammation and muscle damage by inhibiting the enzyme responsible for PGD2 production.678910
What is the purpose of this trial?
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.
Eligibility Criteria
This trial is for people with myotonic dystrophy type 1, who have myotonia and a confirmed genetic diagnosis. Participants should be able to move their tibialis anterior muscles against moderate pressure and have a BMI of less than 32 kg/m2.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive either PGN-EDODM1 or placebo once every 4 weeks for 12 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- PGN-EDODM1
Find a Clinic Near You
Who Is Running the Clinical Trial?
PepGen Inc
Lead Sponsor