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

Официальное название

A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1

Обзор

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).

Подробное 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.

Вмешательства

• Препарат zeleciment basivarsen (DYNE-101)
  Administered by IV infusion
• Препарат Placebo
  Administered by IV infusion

Первичные конечные точки

• 5 Times Sit-To-Stand (5×STS) Time [Срок оценки: Baseline, Week 49]

Вторичные конечные точки (12)

• Video Hand Opening Time (vHOT) [Middle Finger] [Срок оценки: Baseline, Week 49]
• Quantitative Muscle Testing (QMT) Total [Срок оценки: Baseline, Week 49]
• Clinician Global Impression of Change (CGI-C) [Срок оценки: Week 49]
• 10-Meter Walk/Run Test (10-MWRT) Velocity (m/s) [Срок оценки: Baseline, Week 49]
• Patient Global Impression of Change (PGI-C) [Срок оценки: Week 49]
• DM1-ACTIV^C Total Score [Срок оценки: Baseline, Week 49]
• Myotonic Dystrophy Health Index (MDHI) Total [Срок оценки: Baseline, Week 49]
• Patient Global Impression of Severity (PGI-S) [Срок оценки: Baseline, Week 49]
• Clinician Global Impression of Severity (CGI-S) [Срок оценки: Baseline, Week 49]
• 9-Hole Peg Test (9-HPT) Time [Срок оценки: Baseline, Week 49]
• Myotonic Dystrophy Health Index (MDHI) Subscale Scores [Срок оценки: Baseline, Week 49]
• Maximum Observed Plasma Drug Concentration (Cmax) of DYNE-101 [Срок оценки: Pre-dose, and at multiple time points up to Week 73]

Критерии участия

Критерии включения

• 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).

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 8 центров

• UCSD - Altman Clinical and Translational Research Institute — La Jolla
• Rare Disease Research, LLC — Atlanta
• IU Health Neuroscience Center — Indianapolis
• Roy Blunt NextGen Precision Health Institute — Columbia
• Rare Disease Research, LLC — Hillsborough
• The University of Texas Health Science Center at San Antonio — San Antonio
• University of Utah Clinical Neurosciences Center — Salt Lake City
• Virginia Commonwealth University - Biotech One — Richmond

Франция · 4 центра

• CHU Marseille - Hôpital de la Timone (APHM) — Marseille
• CHU de Nantes - Hôtel-Dieu — Nantes
• Pitié-Salpêtrière Hospital — Paris
• CHU de Toulouse - Hôpital Pierre-Paul Riquet — Toulouse

Германия · 3 центра

• LMU Klinikum der Universität München Neurologische Klinik und Poliklinik Friedrich-Baur-In — Munich
• Universitätsklinikum Bonn AöR Zentrum für Neurologie Klinik für Neuroimmunologie und Neuro — Bonn
• Charité Universitätsmedizin Berlin Campus-Buch Muscle Research Unit — Berlin

Италия · 3 центра

• Centro Clinico Nemo Milano, Fondazione Serena ETS — Milan
• Fondazione Policlinico Universitario A. Gemelli IRCCS - Department of Women's, Child and P — Roma
• Azienda Ospedaliero-Universitaria Sant'Andrea - UOC Neurologia — Roma

Япония · 2 центра

• National Hospital Organization Osaka Toneyama Medical Center — Toyonaka-Shi
• Yamaguchi University Hospital — Ube-Shi

Великобритания · 2 центра

• University College London Hospitals NHS Foundation Trust National Hospital for Neurology a — London
• Northern Care Alliance NHS Foundation Trust — Salford

Бельгия · 1 центр

• UZ Leuven — Leuven

Дания · 1 центр

• Rigshospitalet, (Neuromuscular Clinic and Research Unit, Department 8077) — Copenhagen

Испания · 1 центр

• Hospital Universitario Infanta Sofía — San Sebastián de los Reyes

Идентификаторы

NCT: NCT07486934 · DYNE101-DM1-301 · 2025-522957-20-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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