A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
NCT ID: NCT06079736
Last Updated: 2025-12-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE2
7 participants
INTERVENTIONAL
2024-01-03
2025-08-28
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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PGN-EDO51 at Dose Level 1 every 4 weeks
PGN-EDO51
IV infusion
PGN-EDO51 at Dose Level 2 every 4 weeks
PGN-EDO51
IV infusion
Interventions
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PGN-EDO51
IV infusion
Eligibility Criteria
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Inclusion Criteria
* 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)
Exclusion Criteria
* 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
6 Years
16 Years
MALE
No
Sponsors
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PepGen Inc
INDUSTRY
Responsible Party
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Locations
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British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick, Canada
Children's Hospital of Eastern Ontario (CHEO)
Ottawa, Ontario, Canada
The Hospital for Sick Children (SickKids)
Toronto, Ontario, Canada
CHU de Québec
Québec, Quebec, Canada
Countries
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Other Identifiers
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PGN-EDO51-102
Identifier Type: -
Identifier Source: org_study_id
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