Duchenne Muscular Dystrophy
Conditions
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)
Brief summary
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.
Interventions
DRUGPGN-EDO51
IV infusion
Sponsors
PepGen Inc
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
6 Years to 16 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period) | Baseline to Week 16 | Adverse events and serious adverse events |
| Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period) | Baseline to Week 108 | Adverse events and serious adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma pharmacokinetic (PK) parameters (MAD period) | Baseline to Week 12 | Maximum observed plasma concentration of PGN-EDO51 |
| PK Plasma levels (LTE period) | Baseline to Week 104 | PK sampling for PGN-EDO51 and PGN-PMO51 plasma levels |
| Skeletal muscle concentration of PGN-EDO51 (MAD period) | Baseline to Week 16 | Change from baseline in skeletal muscle concentration of PGN-EDO51 after multiple doses |
| Dystrophin Levels (MAD period) | Baseline to Week 16 | Change from baseline in dystrophin levels measured after multiple doses |
Countries
Canada
Outcome results
None listed