Clinical trial NCT06204809: Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
Official title: A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Myotonic Dystrophy 1
Keywords: Myotonic Dystrophy
Muscular Dystrophies
Genetic Diseases, Inborn
Neuromuscular Diseases
Nervous System Diseases
Musculoskeletal Diseases
Myotonic Disorders
Muscular Disorders, Atrophic
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Muscular Diseases
Steinert Disease
Lead sponsor: PepGen Inc
Sponsor class: INDUSTRY
First posted: 2024-01-12
Enrollment: 32
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).

The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).


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

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
* Medications specific for the treatment of myotonia within 2 weeks prior to screening
* Percent predicted forced vital capacity (FVC) \<40%

Note: Other inclusion and exclusion criteria may apply.

Primary outcomes:
measure: Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs
timeFrame: Baseline to Week 16
Secondary outcomes:
measure: Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
timeFrame: Baseline up to Day 3
measure: Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
timeFrame: Baseline up to Day 3
measure: Apparent Terminal Half-Life (t½) of PGN-EDODM1
timeFrame: Baseline up to Day 3
measure: Area Under the Concentration-time Curve of PGN-EDODM1
timeFrame: Baseline up to Day 3
Other outcomes:
Locations:
city: Irvine
country: United States
facility: UCI Center for Clinical Research
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Atlanta
country: United States
facility: Rare Disease Research
state: Georgia
city: Fairway
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
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 Hospital
state: UK
city: Salford
country: United Kingdom
facility: Salford Royal Hospital
Study officials:
References: