Myotonic Dystrophy 1Phase 2FREEDOM2-DM1NCT06667453
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
A Phase 2 study testing PGN-EDODM1 in people with Myotonic Dystrophy 1, run by PepGen Inc at 12 sites in 4 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 16 to 65 years
- 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?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 10 Dec 2024to Mar 2027
- 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 16 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
- Presence of myotonia
- Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
- Body Mass Index (BMI) of < 35.0 kg/m^2
Who cannot join
- Congenital DM1
- Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- Abnormal laboratory tests at screening considered clinically significant by the Investigator
- Medications specific for the treatment of myotonia within 2 weeks prior to screening
- Percent predicted forced vital capacity (FVC) <40%
- Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening
- Note: Other inclusion and exclusion criteria may apply.
Trial locations and contacts
12 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 4 countries
Canada4 sites, 4 recruiting
- Recruiting
University of Calgary
Calgary, Alberta, T2N 4Z6
Patient Advocacy, 781-797-0979, clinicaltrials@pepgen.com
- Recruiting
CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec
Jessica Tremblay, 418-541-1000 ext. 2824, jessica.tremblay.agt@ssss.gouv.qc.ca
- Recruiting
Montreal Neurological Institute
Montreal, Quebec, H3A 2B4
Patient Advocacy, 781-797-0979, clinicaltrials@pepgen.com
- Recruiting
Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario
Jessica MacGregor, 613-798-5555 ext. 19627, jemacgregor@ohri.ca
United Kingdom4 sites, 4 recruiting
- Recruiting
Cardiff and Vale Hospital
Cardiff
Patient Advocacy, 781-797-0979, clinicaltrials@pepgen.com
- Recruiting
University College London Hospitals NHS Foundation Trust
London, UK
Patient Advocacy, 781-797-0979, clinicaltrials@pepgen.com
- Recruiting
Newcastle Upon Tyne Hospitals
Newcastle upon Tyne, NE7 7DN
Patient Advocacy, 781-797-0979, clinicaltrials@pepgen.com
- Recruiting
Salford Royal Hospital
Salford, England
Patient Advocacy, 781-797-0979, clinicaltrials@pepgen.com
South Korea3 sites, 3 recruiting
- Recruiting
Kyungpook National University Chilgok Hospital
Daegu
Minji Kim, ehclalswl8869@knuh-ext.kr
- Recruiting
Pusan National University Yangsan Hospital
Pusan, Gyeongsangnam-do
Patient Advocacy, 781-797-0979, clinicaltrials@pepgen.com
- Recruiting
Seoul National University Hospital
Seoul
Hohyung Lee, 8x013@snuh.org
New Zealand1 site, 1 recruiting
- Recruiting
Pacific Clinical Research Network Auckland
Takapuna, Auckland, 0622
Miriam Rodrigues, Neurogenetics@adhb.govt.nz
About this study
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
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.
- Conditions
- Myotonic Dystrophy 1
- Being tested
- PGN-EDODM1, Placebo
- Keywords
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, PepGen, PGN-EDODM1, Myotonic Muscular Dystrophy, Steinert's Disease
- Medical subject headings
- Myotonic Dystrophy
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
PlaceboOther
Administered by intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| PGN-EDODM1 | Experimental | Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks Receives: Drug: PGN-EDODM1 |
| Placebo | Placebo comparator | Participants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks Receives: Other: Placebo |
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 Day 112
Secondary outcomes (8)
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Measured: Day 1 through Day 84
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Measured: Day 1 through Day 84
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Measured: Day 1 through Day 84
Area Under the Concentration-time Curve of PGN-EDODM1
Measured: Day 1 through Day 84
Change in splicing index in skeletal muscle tissue
Measured: Baseline through Day 91
Change in myotonia as measured by video Hand Opening Time (vHOT)
Measured: Baseline through Day 112
Hand grip strength
by dynamometer
Measured: Baseline through Day 112
Change in mobility as measured by 10 meter walk/run time
Measured: Baseline through Day 112
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2024
- Study start10 Dec 2024
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 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
- NCT06667453
- Sponsor's protocol ID
- PGN-EDODM1-102
- Acronym
- FREEDOM2-DM1
- Status last verified
- Jul 2026
- First submitted
- 22 Oct 2024
- Last updated
- 29 Jul 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 29 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
- ClinicalTrials.gov ID
- NCT06667453
- Acronym / sponsor ID
- FREEDOM2-DM1 / PGN-EDODM1-102
- Conditions
- Myotonic Dystrophy 1
- Keywords
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, PepGen, PGN-EDODM1, Myotonic Muscular Dystrophy, Steinert's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- PepGen IncIndustry (pharma, biotech, devices)
- Study start
- 10 Dec 2024 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 31 Oct 2024 (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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PGN-EDODM1 | Experimental | Drug: PGN-EDODM1Participants will be randomized to receive ascending doses of PGN-EDODM1, once every 4 weeks (Q4W) for 12 weeks |
| Placebo | Placebo comparator | Other: PlaceboParticipants randomized to the placebo arm will receive doses of saline (0.9% NaCl), once every 4 weeks (Q4W) for 12 weeks |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
- Presence of myotonia
- Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed
- Body Mass Index (BMI) of < 35.0 kg/m^2
Key exclusion criteria (7 listed)
- Congenital DM1
- Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- Abnormal laboratory tests at screening considered clinically significant by the Investigator
- Medications specific for the treatment of myotonia within 2 weeks prior to screening
- Percent predicted forced vital capacity (FVC) <40%
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 |
| PlaceboAdministered by intravenous (IV) infusion | Other | Placebo |
Primary outcome
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
Time frame: Baseline through Day 112
8 secondary outcomes
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Time frame: Day 1 through Day 84
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Time frame: Day 1 through Day 84
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Time frame: Day 1 through Day 84
Area Under the Concentration-time Curve of PGN-EDODM1
Time frame: Day 1 through Day 84
Change in splicing index in skeletal muscle tissue
Time frame: Baseline through Day 91
Change in myotonia as measured by video Hand Opening Time (vHOT)
Time frame: Baseline through Day 112
Hand grip strength
Time frame: Baseline through Day 112
Change in mobility as measured by 10 meter walk/run time
Time frame: Baseline through Day 112
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 29 Jul 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.
12 sites in 4 countries: 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 12 sites in 4 countries.
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 4 inclusion and 7 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 29 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Canada, United Kingdom, South Korea, New Zealand. 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. Some participants in this study may receive a placebo. 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 (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 Day 112). 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 29 Jul 2026.