Myotonic Dystrophy 1Phase 2NCT07220603
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
A Phase 2 study testing PGN-EDODM1 in people with Myotonic Dystrophy 1, run by PepGen Inc at 3 sites in Canada.
Trial summary
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Myotonic Dystrophy 1
- What is being tested?
- PGN-EDODM1
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 3 sitesCanada
- Study dates
- 23 Dec 2025to Jan 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participant has completed a prior study with PGN-EDODM1
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Canada3 sites, 3 recruiting
- Recruiting
University of Calgary
Calgary, Alberta, T2N 4Z6
Patient Advocacy, 781-797-0979, clinicaltrials@pepgen.com
- Recruiting
Ottawa Hospital Research Institute
Ottawa, Ontario
Patient Advocacy, clinicaltrials@pepgen.com
- Recruiting
CIUSSS du Saguenay-Lac-Saint-Jean
Saguenay
Patient Advocacy, clinicaltrials@pepgen.com
About this study
Official title: An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.
- Conditions
- Myotonic Dystrophy 1
- Being tested
- PGN-EDODM1
- Keywords
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, PepGen, PGN-EDODM1, Myotonic Muscular Dystrophy, Steinhert's Disease, Myotonic Dystrophies, Genetic Diseases, Inborn, Neuromuscular Diseases, Nervous System Diseases, Musculoskeletal Diseases, Myotonic Disorders, Muscular Disorders, Atrophic, Heredodegenerative Disorders, Nervous System, Muscular Diseases
- Medical subject headings
- Myotonic Dystrophy, Genetic Diseases, Inborn, Neuromuscular Diseases, Nervous System Diseases, Musculoskeletal Diseases, Myotonic Disorders, Muscular Disorders, Atrophic, Heredodegenerative Disorders, Nervous System
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PGN-EDODM1Drug
Administered by intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| PGN-EDODM1 | Experimental | Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W) Receives: Drug: PGN-EDODM1 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
Measured: Baseline through Week 108
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2025
- Study start23 Dec 2025
- Primary completionJan 2029 (estimated)
- Study completionJan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PepGen Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07220603
- Sponsor's protocol ID
- PGN-EDODM1-103
- Status last verified
- Mar 2026
- First submitted
- 22 Oct 2025
- Last updated
- 30 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
- ClinicalTrials.gov ID
- NCT07220603
- Acronym / sponsor ID
- PGN-EDODM1-103
- Conditions
- Myotonic Dystrophy 1
- Keywords
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, PepGen, PGN-EDODM1, Myotonic Muscular Dystrophy, Steinhert's Disease, Myotonic Dystrophies
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- PepGen IncIndustry (pharma, biotech, devices)
- Study start
- 23 Dec 2025 (actual)
- Primary completion
- Jan 2029 (estimated)
- Study completion
- Jan 2029 (estimated)
- First posted
- 24 Oct 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PGN-EDODM1 | Experimental | Drug: PGN-EDODM1Participants will receive doses of PGN-EDODM1 once every 4 weeks (Q4W) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Participant has completed a prior study with PGN-EDODM1
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PGN-EDODM1Administered by intravenous (IV) infusion | Drug | PGN-EDODM1 |
Primary outcome
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
Time frame: Baseline through Week 108
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PGN-EDODM1 in people with Myotonic Dystrophy 1, run by PepGen Inc at 3 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 30 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact PepGen Patient Advocacy (781-797-0979, clinicaltrials@pepgen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability as assessed by number of participants with Adverse Events (AEs) (time frame: Baseline through Week 108). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2026.