Myotonic Dystrophy 1Phase 1FREEDOM-DM1NCT06204809
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
A Phase 1 study testing PGN-EDODM1 for infusion in people with Myotonic Dystrophy 1, run by PepGen Inc at 12 sites in 3 countries.
Trial summary
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 Myotonic Dystrophy Type 1 (DM1). The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Myotonic Dystrophy 1
- What is being tested?
- PGN-EDODM1 for infusion
- 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 3 countries
- Study dates
- 12 Dec 2023to 28 Oct 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 18 to 60 years
- All sexes
- No healthy volunteers
- 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
- Medical Research Council (MRC) score of ≥ Grade 4- in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
- Presence of myotonia
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
- Medications specific for the treatment of myotonia within 2 weeks prior to screening
- Percent predicted forced vital capacity (FVC) <40%
- Note: Other inclusion and exclusion criteria may apply.
Trial locations and contacts
12 sites in 3 countries.
12 sites in 3 countries
United States7 sites
Rare Disease Research
Atlanta, Georgia, 30329
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of Kansas Medical Center
Fairway, Kansas, 66205
UCI Center for Clinical Research
Irvine, California, 92697
Stanford University
Palo Alto, California, 94304
Virginia Commonwealth University
Richmond, Virginia, 23298
University of Rochester Medical Center
Rochester, New York, 14642
Canada3 sites
University of Calgary
Calgary, Alberta, T3M 1M4
CIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec
Ottawa Hospital Research Institute (OHRI)
Ottawa, Ontario
United Kingdom2 sites
University College London Hospital
London, UK, NW1 2PG
Salford Royal Hospital
Salford
About this study
Official title: A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)
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 Myotonic Dystrophy Type 1 (DM1).
The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
- Conditions
- Myotonic Dystrophy 1
- Being tested
- PGN-EDODM1 for infusion, Placebo
- Keywords
- Myotonic Dystrophy, Muscular Dystrophies, Genetic Diseases, Inborn, Neuromuscular Diseases, Nervous System Diseases, Musculoskeletal Diseases, Myotonic Disorders, Muscular Disorders, Atrophic, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases, Muscular Diseases, Steinert Disease
- Medical subject headings
- Myotonic Dystrophy, Muscular Dystrophies, Genetic Diseases, Inborn, Neuromuscular Diseases, Nervous System Diseases, Musculoskeletal Diseases, Myotonic Disorders, Muscular Disorders, Atrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PGN-EDODM1 for infusionDrug
Single dose of PGN-EDODM1 by intravenous (IV) infusion
PlaceboOther
Administered by IV infusion
| Study group | Role | What they receive |
|---|---|---|
| PGN-EDODM1 | Experimental | PGN-EDODM1 for infusion Receives: Drug: PGN-EDODM1 for infusion |
| Placebo | Placebo comparator | 0.9% NaCl Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
Measured: Baseline to Week 16
Secondary outcomes (4)
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Measured: Baseline up to Day 3
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Measured: Baseline up to Day 3
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Measured: Baseline up to Day 3
Area Under the Concentration-time Curve of PGN-EDODM1
Measured: Baseline up to Day 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2024
- Study start12 Dec 2023
- Primary completion28 Oct 2025
- Study completion28 Oct 2025
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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- PepGen Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06204809
- Sponsor's protocol ID
- PGN-EDODM1-101
- Acronym
- FREEDOM-DM1
- Status last verified
- Feb 2026
- First submitted
- 5 Dec 2023
- Last updated
- 13 Feb 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 13 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)
- ClinicalTrials.gov ID
- NCT06204809
- Acronym / sponsor ID
- FREEDOM-DM1 / PGN-EDODM1-101
- Conditions
- Myotonic Dystrophy 1
- Keywords
- Myotonic Dystrophy, Muscular Dystrophies, Genetic Diseases, Inborn, Neuromuscular Diseases, Nervous System Diseases, Musculoskeletal Diseases, Myotonic Disorders, Muscular Disorders, Atrophic
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- PepGen IncIndustry (pharma, biotech, devices)
- Study start
- 12 Dec 2023 (actual)
- Primary completion
- 28 Oct 2025 (actual)
- Study completion
- 28 Oct 2025 (actual)
- First posted
- 12 Jan 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 primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
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-EDODM1 for infusionPGN-EDODM1 for infusion |
| Placebo | Placebo comparator | Other: Placebo0.9% NaCl |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
- Medical Research Council (MRC) score of ≥ Grade 4- in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
- Presence of myotonia
Key exclusion criteria (6 listed)
- Congenital DM1
- Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- Abnormal laboratory tests at screening
- 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-EDODM1 for infusionSingle dose of PGN-EDODM1 by intravenous (IV) infusion | Drug | PGN-EDODM1 |
| PlaceboAdministered by IV infusion | Other | Placebo |
Primary outcome
Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
Time frame: Baseline to Week 16
4 secondary outcomes
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Time frame: Baseline up to Day 3
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Time frame: Baseline up to Day 3
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Time frame: Baseline up to Day 3
Area Under the Concentration-time Curve of PGN-EDODM1
Time frame: Baseline up to Day 3
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Feb 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PGN-EDODM1 for infusion in people with Myotonic Dystrophy 1, run by PepGen Inc at 12 sites in 3 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 3 inclusion and 6 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: finished, as of the sponsor's last update on 13 Feb 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 United States, Canada, United Kingdom. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs (time frame: Baseline to Week 16). 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 13 Feb 2026; AzTrials last refreshed its copy on 7 Nov 2026.