PGN-EDODM1 for Myotonic Dystrophy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial focuses on the safety and tolerability of the treatment PGN-EDODM1 for people with myotonic dystrophy type 1 (DM1). The study examines long-term outcomes for individuals receiving this treatment. Participants will receive doses of PGN-EDODM1 once every four weeks. Suitable candidates for this trial are those who have completed a previous study with PGN-EDODM1. As a Phase 2 trial, the research aims to measure the treatment's effectiveness in an initial, smaller group of people.
Is there any evidence suggesting that PGN-EDODM1 is likely to be safe for humans?
Research has shown that PGN-EDODM1 is usually well-tolerated. In earlier studies, most side effects were mild or moderate, indicating they weren't serious. No major safety problems emerged, suggesting the treatment is safe for most people. However, reactions can vary, so discussing any concerns with a doctor is important.12345
Why do researchers think this study treatment might be promising?
PGN-EDODM1 is unique because it targets the underlying genetic cause of Myotonic Dystrophy, offering a new approach compared to current treatments that primarily manage symptoms. While most existing treatments focus on alleviating muscle weakness and pain, PGN-EDODM1 utilizes a novel mechanism targeting the faulty RNA responsible for the disease. Researchers are excited because this could potentially alter the disease course, offering hope for more effective management of Myotonic Dystrophy in the future.
What evidence suggests that PGN-EDODM1 might be an effective treatment for myotonic dystrophy?
Research shows that PGN-EDODM1, the treatment under study in this trial, may help treat myotonic dystrophy type 1 (DM1). A previous study demonstrated a 53.7% improvement in correcting gene activity after just one dose of the drug. This is significant because it targets the genetic cause of DM1. All patients in that study experienced some improvement. The treatment was generally well-tolerated, with no serious side effects reported. These findings suggest that PGN-EDODM1 could help manage DM1 symptoms.23678
Are You a Good Fit for This Trial?
This trial is for individuals with myotonic dystrophy type 1 who have already completed a previous study involving PGN-EDODM1. It's designed to gather more information on the long-term effects of this treatment.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Open-label extension
Participants receive doses of PGN-EDODM1 once every 4 weeks to evaluate long-term 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
Trial Overview
PGN-EDODM1, a potential treatment for myotonic dystrophy type 1, is being tested for its safety and tolerability over an extended period in patients who've used it before.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W)
Find a Clinic Near You
Who Is Running the Clinical Trial?
PepGen Inc
Lead Sponsor
Citations
1.
investors.pepgen.com
investors.pepgen.com/news-releases/news-release-details/pepgen-announces-highest-mean-splicing-correction-reported-dm1PepGen Announces Highest Mean Splicing Correction ...
53.7% mean splicing correction observed following a single 15 mg/kg dose of PGN-EDODM1, with all patients showing an improvement in splicing ...
PepGen's stock up 120% on DM1 trial success
PGN-EDODM1 was generally well-tolerated at 15mg/kg, with no serious treatment-related adverse events (AEs). All treatment-related AEs at 15mg/kg ...
NCT06667453 | A Clinical Study of PGN-EDODM1 in ...
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 ...
4.
neurologylive.com
neurologylive.com/view/early-phase-1-trial-data-suggests-therapeutic-potential-pgn-edodm1-myotonic-dystrophy-type-1Early Phase 1 Trial Data Suggests Therapeutic Potential ...
Initial data reported from the FREEDOM-DM1 trial suggest PGN-EDODM1 has dose-dependent effects in patients with DM1, with further results from study cohorts ...
EX-99.1
All six patients (100%) receiving a 15 mg/kg dose of PGN-EDODM1 responded to treatment by showing improved splicing correction. · In addition, ...
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in ...
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 ...
23OResults from 15 mg/kg single dose PGN-EDODM1 ...
The emerging safety profile for PGN-EDODM1 is favorable. We expect to report data from Cohort 3 at 15 mg/kg in this presentation. In pharmacological studies ...
PepGen Inc. Announces FDA has Lifted the Clinical Hold ...
Our novel PGN-. EDODM1 approach targets the toxic RNA species responsible for the disease, and the strength of our Enhanced Delivery ...
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.