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A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)

Primary Purpose

Duchenne Muscular Dystrophy

Status
Not yet recruiting
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
PGN-EDO51
Sponsored by
PepGen Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Experimental

    Experimental

    Experimental

    Arm Label

    PGN-EDO51 at Dose Level 1

    PGN-EDO51 at Dose Level 2

    PGN-EDO51 at Dose Level 3

    Arm Description

    Outcomes

    Primary Outcome Measures

    Adverse events and serious adverse events (safety and tolerability of PGN-EDO51)
    Adverse events and serious adverse events

    Secondary Outcome Measures

    Plasma pharmacokinetic (PK) parameters
    Maximum observed plasma concentration of PGN-EDO51
    Plasma pharmacokinetic (PK) parameters
    Time to maximum observed plasma concentration of PGN-ED051
    Plasma pharmacokinetic (PK) parameters
    Apparent terminal half-life of PGN-EDO51
    Plasma pharmacokinetic (PK) parameters
    Area under the curve for concentration time of PGN-EDO51

    Full Information

    First Posted
    September 29, 2023
    Last Updated
    October 5, 2023
    Sponsor
    PepGen Inc
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    1. Study Identification

    Unique Protocol Identification Number
    NCT06079736
    Brief Title
    A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
    Acronym
    CONNECT1-EDO51
    Official Title
    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)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    October 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    October 2023 (Anticipated)
    Primary Completion Date
    March 2025 (Anticipated)
    Study Completion Date
    March 2025 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    PepGen Inc

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    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).

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Duchenne Muscular Dystrophy

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Sequential Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    10 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    PGN-EDO51 at Dose Level 1
    Arm Type
    Experimental
    Arm Title
    PGN-EDO51 at Dose Level 2
    Arm Type
    Experimental
    Arm Title
    PGN-EDO51 at Dose Level 3
    Arm Type
    Experimental
    Intervention Type
    Drug
    Intervention Name(s)
    PGN-EDO51
    Intervention Description
    IV infusion
    Primary Outcome Measure Information:
    Title
    Adverse events and serious adverse events (safety and tolerability of PGN-EDO51)
    Description
    Adverse events and serious adverse events
    Time Frame
    Baseline to Week 16
    Secondary Outcome Measure Information:
    Title
    Plasma pharmacokinetic (PK) parameters
    Description
    Maximum observed plasma concentration of PGN-EDO51
    Time Frame
    Baseline to Week 12
    Title
    Plasma pharmacokinetic (PK) parameters
    Description
    Time to maximum observed plasma concentration of PGN-ED051
    Time Frame
    Baseline to Week 12
    Title
    Plasma pharmacokinetic (PK) parameters
    Description
    Apparent terminal half-life of PGN-EDO51
    Time Frame
    Baseline to Week 12
    Title
    Plasma pharmacokinetic (PK) parameters
    Description
    Area under the curve for concentration time of PGN-EDO51
    Time Frame
    Baseline to Week 12

    10. Eligibility

    Sex
    Male
    Minimum Age & Unit of Time
    10 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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

    12. IPD Sharing Statement

    Learn more about this trial

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

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