Clinical trial NCT06667453: A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Myotonic Dystrophy 1
Keywords: DM1
Myotonic Dystrophy 1
Myotonic Dystrophy
PepGen
PGN-EDODM1
Myotonic Muscular Dystrophy
Steinert&#39;s Disease
Lead sponsor: PepGen Inc
Sponsor class: INDUSTRY
First posted: 2024-10-31
Enrollment: 24
Sex: ALL
Minimum age (years): 16
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.


Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats
* Medical Research Council (MRC) score of ≥ Grade 4- in bilateral tibialis anterior (TA) muscles (the ability to move through full range of motion and hold against at least moderate pressure from the examiner)
* Presence of myotonia
* Body Mass Index (BMI) of \< 32.0 kg/m\^2

Exclusion Criteria:

* Congenital DM1
* Known history or presence of any clinically significant conditions that may interfere with study safety assessments
* Abnormal laboratory tests at screening considered clinically significant by the Investigator
* Medications specific for the treatment of myotonia within 2 weeks prior to screening
* Percent predicted forced vital capacity (FVC) \<40%
* Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening

Note: Other inclusion and exclusion criteria may apply.

Primary outcomes:
measure: Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
timeFrame: Baseline through Day 112
Secondary outcomes:
measure: Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
timeFrame: Day 1 through Day 84
measure: Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
timeFrame: Day 1 through Day 84
measure: Apparent Terminal Half-Life (t½) of PGN-EDODM1
timeFrame: Day 1 through Day 84
measure: Area Under the Concentration-time Curve of PGN-EDODM1
timeFrame: Day 1 through Day 84
measure: Change in splicing index in skeletal muscle tissue
timeFrame: Baseline through Day 91
measure: Change in myotonia as measured by video Hand Opening Time (vHOT)
timeFrame: Baseline through Day 112
measure: Hand grip strength
timeFrame: Baseline through Day 112
measure: Change in mobility as measured by 10 meter walk/run time
timeFrame: Baseline through Day 112
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Ottawa
country: Canada
facility: Ottawa Hospital Research Institute (OHRI)
state: Ontario
city: Chicoutimi
country: Canada
facility: CIUSSS du Saguenay-Lac-Saint-Jean
state: Quebec
city: London
country: United Kingdom
facility: University College London Hospitals NHS Foundation Trust
state: UK
Study officials:
References: