A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment

Study Overview
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Brief Summary
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The primary purpose of the study 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 study consists of 2 periods: A Screening Period (up to 45 days) and a Treatment and Observation Period (16 weeks).

Official Title
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A Phase 2, Open-Label, Multiple Ascending Dose Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: PGN-EDO51


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males by birth, age at least 10 years at the time of consent/assent provided Confirmed diagnosis of DMD able to be corrected by skipping Exon 51 Body weight at least 25kg at Screening Performance of Upper Limb (PUL) 2.0 entry score of at least 3 at Screening Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 10 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PGN-EDO51 at Dose Level 1<br> | Drug: PGN-EDO51<br>* IV infusion<br>|
| Experimental: PGN-EDO51 at Dose Level 2<br> | Drug: PGN-EDO51<br>* IV infusion<br>|
| Experimental: PGN-EDO51 at Dose Level 3<br> | Drug: PGN-EDO51<br>* IV infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events and serious adverse events (safety and tolerability of PGN-EDO51) | Adverse events and serious adverse events | Baseline to Week 16 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Plasma pharmacokinetic (PK) parameters | Maximum observed plasma concentration of PGN-EDO51 | Baseline to Week 12 | 
| Plasma pharmacokinetic (PK) parameters | Time to maximum observed plasma concentration of PGN-ED051 | Baseline to Week 12 | 
| Plasma pharmacokinetic (PK) parameters | Apparent terminal half-life of PGN-EDO51 | Baseline to Week 12 | 
| Plasma pharmacokinetic (PK) parameters | Area under the curve for concentration time of PGN-EDO51 | Baseline to Week 12 |