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Recruiting Phase 2 NCT06667453

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1

Phase 2 – studying effectiveness and dosage
Conditions: Myotonic Dystrophy 1

Sponsor: PepGen Inc

trial.available_in: БГ
Overview
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.
Who can participate
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.
Locations 3
Canada (1)
University of Calgary
Calgary , Alberta
New Zealand (1)
Pacific Clinical Research Network Auckland
Takapuna , Auckland
ACTIVE_NOT_RECRUITING
United Kingdom (1)
Salford Royal Hospital
Salford , England
Technical details
Status
Recruiting
Phase
Phase 2
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
16 Years
Maximum age
60 Years
Healthy volunteers
No
Start date
10.12.2024
Completion date
01.08.2026
Registry ID
NCT06667453
Source
anzctr
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