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Набира участници Фаза 3 NCT07486934

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

Фаза 3 – широко изпитване преди одобрение
Заболявания: Myotonic Dystrophy Type 1 (DM1) DM1 Myotonic Dystrophy Steinert Disease Steinert

Спонсор: Dyne Therapeutics

Налично на: БГ
Обобщение
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).
Описание
The study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks). An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.
Кой може да участва
Inclusion Criteria: * Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\>) 100. Historical results from clinical testing are acceptable. * Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the ankle are allowed). * Body mass index (BMI) less than (\<) 35 kilograms per meter square (kg/m\^2). Exclusion Criteria: * A known diagnosis of congenital DM1. * History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator. * Use of glucagon-like peptide 1 (GLP-1) agonist/incretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments. Note: Other inclusion and exclusion criteria may apply.
Интервенции
zeleciment basivarsen (DYNE-101)
DRUG
Placebo
DRUG
Места на провеждане 4
Япония (1)
Yamaguchi University Hospital
Ube-Shi , Yamaguchi
Masayuki Nakamori, MD, PhD, Professor
САЩ (3)
Rare Disease Research, LLC
Atlanta , Georgia
Rare Disease Research, LLC
Hillsborough , North Carolina
The University of Texas Health Science Center at San Antonio
San Antonio , Texas
Marlon Humbert Tamayo Muradas
Технически детайли
Статус
Набира участници
Фаза
Фаза 3
Вид изследване
INTERVENTIONAL
Пол
Мъже и жени
Минимална възраст
16 Years
Здрави доброволци
Не
Начална дата
01.04.2026
Крайна дата
01.01.2029
Регистрационен номер
NCT07486934
Източник
clinicaltrials.gov
Запитване за медицински туризъм

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