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

Duchenne Muscular Dystrophy

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.

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Conditions de participation

  • Sexe:

    MALE

  • Âges admissibles:

    6 to 16

Critères de participation

Inclusion Criteria:

* 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)

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

Lieu de l'étude

Children's Hospital of Eastern Ontario (CHEO)
Children's Hospital of Eastern Ontario (CHEO)
Ottawa, Ontario
Canada

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Primary Contact

Emilie Hill-Smith

[email protected]
Stan Cassidy Centre for Rehabilitation
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick
Canada

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Backup Contact

Susan McCully

[email protected]
Primary Contact

Shane McCullum

[email protected]
The Hospital for Sick Children (SickKids)
The Hospital for Sick Children (SickKids)
Toronto, Ontario
Canada

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Primary Contact

Ana Stosic

[email protected]
British Columbia Children's Hospital
British Columbia Children's Hospital
Vancouver, British Columbia
Canada

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Primary Contact

Nela Martic

[email protected]
CHU de Québec
CHU de Québec
Québec, Quebec
Canada

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Primary Contact

Patient Advocacy

[email protected]
Étude parrainée par
PepGen Inc
Participants recherchés
Plus d'informations
ID de l'étude: NCT06079736