Duchenne Muscular Dystrophy (DMD)Phase 2CONNECT2-EDO51NCT06833931
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
A Phase 2 study testing IV infusion in people with Duchenne Muscular Dystrophy (DMD), run by PepGen Inc.
Trial summary
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks). Read more about this study
- Who is it for?
- Ages 6 to 16 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- IV infusion
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 17 Dec 2024to 28 May 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 with a genetic alteration that can be treated by skipping exon 51
- Body weight at least 25kg (55lbs)
- Performance of Upper Limb (PUL) 2.0 entry score of at least 4
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
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- IV infusion, Placebo
- 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.
IV infusionDrug
IV infusion
PlaceboOther
IV infusion
| Study group | Role | What they receive |
|---|---|---|
| PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks | Experimental | Not described Receives: Drug: IV infusion; Other: Placebo |
| PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks | Experimental | Not described Receives: Drug: IV infusion; Other: Placebo |
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 during the MAD period)
Measured: Signing of informed consent to Week 28
Dystrophin levels (MAD period)
Measured: Baseline to Week 28
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the LTE period)
Measured: Signing of informed consent to Week 108
Secondary outcomes (6)
Plasma pharmacokinetic (PK) parameters (MAD period)
Maximum observed plasma concentration of PGN-EDO51
Measured: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Time to maximum observed plasma concentration of PGN-EDO51
Measured: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Apparent terminal half-life of PGN-EDO51
Measured: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Area under the curve for concentration time of PGN-EDO51
Measured: Baseline to Week 28
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 28
Plasma pharmacokinetic (PK) parameters (LTE period)
PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels
Measured: Baseline to Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2025
- Study start17 Dec 2024
- Primary completion28 May 2025
- Study completion28 May 2025
Why it stopped: Global PGN-EDO51 development voluntarily discontinued 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 Dystrophy (DMD)Studies 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
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 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
- NCT06833931
- Sponsor's protocol ID
- PGN-EDO51-103
- Other IDs
- U1111-1303-9370, 2023-508383-29-00, 2023
- Acronym
- CONNECT2-EDO51
- Status last verified
- Jun 2025
- First submitted
- 13 Feb 2025
- Last updated
- 24 Jun 2025
- 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 24 Jun 2025. 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-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)
- ClinicalTrials.gov ID
- NCT06833931
- Acronym / sponsor ID
- CONNECT2-EDO51 / PGN-EDO51-103
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- 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
- WithdrawnReason stopped: Global PGN-EDO51 development voluntarily discontinued by Sponsor
- Enrolment
- 0 participants (actual)
- Lead sponsor
- PepGen IncIndustry (pharma, biotech, devices)
- Study start
- 17 Dec 2024 (actual)
- Primary completion
- 28 May 2025 (actual)
- Study completion
- 28 May 2025 (actual)
- First posted
- 19 Feb 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 study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
The primary purpose of the MAD period is to evaluate the safety and tolerability and levels of dystrophin after multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). During the MAD period, participants will be randomized to either receive PGN-EDO51 or placebo in a 3:1 fashion, meaning that participants have a 75% chance of receiving PGN-EDO51 and a 25% chance of receiving placebo during this period. The primary purpose of the open-label LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. All participants who roll-over into the LTE will receive PGN-EDO51 (no placebo in the LTE).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks | Experimental | Drug: IV infusion, Other: Placebo |
| PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks | Experimental | Drug: IV infusion, Other: Placebo |
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 with a genetic alteration that can be treated by skipping exon 51
- Body weight at least 25kg (55lbs)
- Performance of Upper Limb (PUL) 2.0 entry score of at least 4
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 |
|---|---|---|
| IV infusionIV infusion | Drug | PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks, PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks |
| PlaceboIV infusion | Other | PGN-EDO51 at Dose Level 1 or Placebo every 4 weeks, PGN-EDO51 at Dose Level 2 or Placebo every 4 weeks |
Primary outcome
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the MAD period)
Time frame: Signing of informed consent to Week 28
Dystrophin levels (MAD period)
Time frame: Baseline to Week 28
Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 during the LTE period)
Time frame: Signing of informed consent to Week 108
6 secondary outcomes
Plasma pharmacokinetic (PK) parameters (MAD period)
Time frame: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Time frame: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Time frame: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (MAD period)
Time frame: Baseline to Week 28
Skeletal muscle concentration of PGN-EDO51 (MAD period)
Time frame: Baseline to Week 28
Plasma pharmacokinetic (PK) parameters (LTE period)
Time frame: Baseline to Week 104
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IV infusion in people with Duchenne Muscular Dystrophy (DMD), run by PepGen Inc.
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: withdrawn, as of the sponsor's last update on 24 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
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 withdrawn before enrolling participants, so there are no results.
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 during the MAD period) (time frame: Signing of informed consent to Week 28). The study lists 3 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 24 Jun 2025.