Clinical trial NCT06079736: A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
Official title: A Phase 2, Open-Label, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT1-EDO51)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
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)
Lead sponsor: PepGen Inc
Sponsor class: INDUSTRY
First posted: 2023-10-12
Enrollment: 7
Sex: MALE
Minimum age (years): 6
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Adverse events and serious adverse events (safety and tolerability of PGN-EDO51 in MAD period)
timeFrame: Baseline to Week 16
measure: Adverse events and serious adverse events (long-term safety and tolerability of PGN-EDO51 in LTE period)
timeFrame: Baseline to Week 108
Secondary outcomes:
measure: Plasma pharmacokinetic (PK) parameters (MAD period)
timeFrame: Baseline to Week 12
measure: Plasma pharmacokinetic (PK) parameters (MAD period)
timeFrame: Baseline to Week 12
measure: Plasma pharmacokinetic (PK) parameters (MAD period)
timeFrame: Baseline to Week 12
measure: Plasma pharmacokinetic (PK) parameters (MAD period)
timeFrame: Baseline to Week 12
measure: PK Plasma levels (LTE period)
timeFrame: Baseline to Week 104
measure: Skeletal muscle concentration of PGN-EDO51 (MAD period)
timeFrame: Baseline to Week 16
measure: Dystrophin Levels (MAD period)
timeFrame: Baseline to Week 16
Other outcomes:
Locations:
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
state: British Columbia
city: Fredericton
country: Canada
facility: Stan Cassidy Centre for Rehabilitation
state: New Brunswick
city: Ottawa
country: Canada
facility: Children's Hospital of Eastern Ontario (CHEO)
state: Ontario
city: Toronto
country: Canada
facility: The Hospital for Sick Children (SickKids)
state: Ontario
city: Québec
country: Canada
facility: CHU de Québec
state: Quebec
Study officials:
References: