Actively Recruiting
An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
Led by PepGen Inc · Updated on 2026-03-30
48
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to learn about the long-term safety and tolerability of PGN-EDODM1 in people with myotonic dystrophy type 1 (DM1) who have previously participated in a study involving PGN-EDODM1. The study is a phase 2 open-label extension designed to gather more information on how participants respond to ongoing treatment with this drug. Participants will receive PGN-EDODM1 through intravenous (IV) infusion once every 4 weeks. This dosing schedule continues for up to 108 weeks, allowing researchers to monitor the effects of the medication over a long period. The study does not include a placebo group and is not blinded, meaning both participants and researchers know the treatment being given. During the study, participants will be closely monitored for any adverse events and other safety concerns. Evaluations will include tracking side effects and tolerability from the start of the study through week 108. Researchers will also assess how the drug behaves in the body over time. Participants may undergo laboratory tests and other assessments to support these evaluations, with a focus on maintaining participant safety throughout the study period.
CONDITIONS
Brief Title
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant has completed a prior study with PGN-EDODM1
You will not qualify if you...
- Abnormal laboratory tests at screening considered clinically significant by the Investigator
- Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-lives of the study drug (whichever is longer) prior to study entry
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 108 weeks
Participants receive doses of PGN-EDODM1 administered by intravenous infusion once every 4 weeks.
Dosing visits every 4 weeks
Trial Site Locations
Total: 3 locations
1
University of Calgary
Calgary, Alberta, Canada, T2N 4Z6
Actively Recruiting
2
Ottawa Hospital Research Institute
Ottawa, Ontario, Canada
Actively Recruiting
3
CIUSSS du Saguenay-Lac-Saint-Jean
Saguenay, Canada
Actively Recruiting
Research Team
P
PepGen Patient Advocacy
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here