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Набира участници Фаза 2 NCT06667453

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

Фаза 2 – изследване на ефективността и дозировката
Заболявания: Myotonic Dystrophy 1

Спонсор: PepGen Inc

Налично на: БГ
Обобщение
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.
Кой може да участва
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.
Места на провеждане 3
Канада (1)
University of Calgary
Calgary , Alberta
New Zealand (1)
Pacific Clinical Research Network Auckland
Takapuna , Auckland
ACTIVE_NOT_RECRUITING
Великобритания (1)
Salford Royal Hospital
Salford , England
Технически детайли
Статус
Набира участници
Фаза
Фаза 2
Вид изследване
INTERVENTIONAL
Пол
Мъже и жени
Минимална възраст
16 Years
Максимална възраст
60 Years
Здрави доброволци
Не
Начална дата
10.12.2024
Крайна дата
01.08.2026
Регистрационен номер
NCT06667453
Източник
anzctr
Запитване за медицински туризъм

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