Duchenne Muscular DystrophyPhase 2CONNECT1-EDO51NCT06079736
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
A Phase 2 study testing PGN-EDO51 in people with Duchenne Muscular Dystrophy, run by PepGen Inc at 5 sites in Canada.
Trial summary
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks). The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51… Read more about this study
- Who is it for?
- Ages 6 to 16 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- PGN-EDO51
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 5 sitesCanada
- Study dates
- 3 Jan 2024to 28 Aug 2025
- Phase
- Phase 2
- 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 6 to 16 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Confirmed diagnosis of DMD able to be corrected by skipping Exon 51
- Body weight at least 18kg at Screening
- Performance of Upper Limb (PUL) 2.0 entry score of at least 4 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD)
Who cannot join
- Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- Treatment with any gene replacement therapy for the treatment of DMD at any time
- Current or recent systemic infection within 2 weeks prior to Screening or infection requiring IV antibiotics within 4 weeks prior to Screening
- Recent surgery requiring anesthesia within 3 months prior to Screening or expected surgery requiring general anesthesia during the study
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Canada5 sites
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick, E3B0C7
Children's Hospital of Eastern Ontario (CHEO)
Ottawa, Ontario, K1H8L1
CHU de Québec
Québec, Quebec, G1V4G2
The Hospital for Sick Children (SickKids)
Toronto, Ontario, M5G0A4
British Columbia Children's Hospital
Vancouver, British Columbia, V6H1G9
About this study
Official title: A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne Muscular Dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- PGN-EDO51
- Keywords
- Enhanced Delivery Oligonucleotide, Peptide-conjugated phosphorodiamidate, Morpholino oligomer, Oligonucleotide, Exon 51, Next-generation oligonucleotide, Cell-penetrating peptide, Muscular Dystrophies, Neuromuscular Disease, Dystrophin production, Splice correcting oligonucleotide, Endosomal Escape, Delivery to the cell nucleus, Antisense oligonucleotide, phosphorodiamidate morpholino oligomer (PPMO)
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PGN-EDO51Drug
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| PGN-EDO51 at Dose Level 1 every 4 weeks | Experimental | Not described Receives: Drug: PGN-EDO51 |
| PGN-EDO51 at Dose Level 2 every 4 weeks | Experimental | Not described Receives: Drug: PGN-EDO51 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)
Adverse events and serious adverse events
Measured: Baseline to Week 16
Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period)
Adverse events and serious adverse events
Measured: Baseline to Week 108
Secondary outcomes (7)
Plasma pharmacokinetic (PK) parameters (MAD period)
Maximum observed plasma concentration of PGN-EDO51
Measured: Baseline to Week 12
Plasma pharmacokinetic (PK) parameters (MAD period)
Time to maximum observed plasma concentration of PGN-EDO51
Measured: Baseline to Week 12
Plasma pharmacokinetic (PK) parameters (MAD period)
Apparent terminal half-life of PGN-EDO51
Measured: Baseline to Week 12
Plasma pharmacokinetic (PK) parameters (MAD period)
Area under the curve for concentration time of PGN-EDO51
Measured: Baseline to Week 12
PK Plasma levels (LTE period)
PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels
Measured: Baseline to Week 104
Skeletal muscle concentration of PGN-EDO51 (MAD period)
Change from baseline in skeletal muscle concentration of PGN-EDO51 after multiple doses
Measured: Baseline to Week 16
Dystrophin Levels (MAD period)
Change from baseline in dystrophin levels measured after multiple doses
Measured: Baseline to Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2023
- Study start3 Jan 2024
- Primary completion28 Aug 2025
- Study completion28 Aug 2025
Why it stopped: PGN-EDO51 development terminated by Sponsor
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 7 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
- NCT06079736
- Sponsor's protocol ID
- PGN-EDO51-102
- Acronym
- CONNECT1-EDO51
- Status last verified
- Dec 2025
- First submitted
- 29 Sep 2023
- Last updated
- 2 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
- ClinicalTrials.gov ID
- NCT06079736
- Acronym / sponsor ID
- CONNECT1-EDO51 / PGN-EDO51-102
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Enhanced Delivery Oligonucleotide, Peptide-conjugated phosphorodiamidate, Morpholino oligomer, Oligonucleotide, Exon 51, Next-generation oligonucleotide, Cell-penetrating peptide, Muscular Dystrophies
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: PGN-EDO51 development terminated by Sponsor
- Enrolment
- 7 participants (actual)
- Lead sponsor
- PepGen IncIndustry (pharma, biotech, devices)
- Study start
- 3 Jan 2024 (actual)
- Primary completion
- 28 Aug 2025 (actual)
- Study completion
- 28 Aug 2025 (actual)
- First posted
- 12 Oct 2023 (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 study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne Muscular Dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PGN-EDO51 at Dose Level 1 every 4 weeks | Experimental | Drug: PGN-EDO51 |
| PGN-EDO51 at Dose Level 2 every 4 weeks | Experimental | Drug: PGN-EDO51 |
Who may qualify
- Ages
- 6 to 16 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Confirmed diagnosis of DMD able to be corrected by skipping Exon 51
- Body weight at least 18kg at Screening
- Performance of Upper Limb (PUL) 2.0 entry score of at least 4 at Screening (assessing upper limb function in ambulant and non-ambulant individuals with DMD)
Key exclusion criteria (4 listed)
- Known history or presence of any clinically significant conditions that may interfere with study safety assessments
- Treatment with any gene replacement therapy for the treatment of DMD at any time
- Current or recent systemic infection within 2 weeks prior to Screening or infection requiring IV antibiotics within 4 weeks prior to Screening
- Recent surgery requiring anesthesia within 3 months prior to Screening or expected surgery requiring general anesthesia during the study
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-EDO51IV infusion | Drug | PGN-EDO51 at Dose Level 1 every 4 weeks, PGN-EDO51 at Dose Level 2 every 4 weeks |
Primary outcome
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)
Time frame: Baseline to Week 16
Adverse events and serious adverse events
Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period)
Time frame: Baseline to Week 108
Adverse events and serious adverse events
7 secondary outcomes
Plasma pharmacokinetic (PK) parameters (MAD period)
Time frame: Baseline to Week 12
Plasma pharmacokinetic (PK) parameters (MAD period)
Time frame: Baseline to Week 12
Plasma pharmacokinetic (PK) parameters (MAD period)
Time frame: Baseline to Week 12
Plasma pharmacokinetic (PK) parameters (MAD period)
Time frame: Baseline to Week 12
PK Plasma levels (LTE period)
Time frame: Baseline to Week 104
Skeletal muscle concentration of PGN-EDO51 (MAD period)
Time frame: Baseline to Week 16
Dystrophin Levels (MAD period)
Time frame: Baseline to Week 16
Results and safety
The study was stopped early (reason given: PGN-EDO51 development terminated by Sponsor). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 2025.
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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.
5 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-EDO51 in people with Duchenne Muscular Dystrophy, run by PepGen Inc at 5 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 3 inclusion and 4 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: stopped early, as of the sponsor's last update on 2 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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?
The study was stopped early (reason given: PGN-EDO51 development terminated by Sponsor). Results are not available in the source record.
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?
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period) (time frame: Baseline to Week 16). The study lists 2 primary outcomes in all. 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 2 Dec 2025.