Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Официальное название

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

Обзор

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Подробное описание

The study consists of a Screening Period of up to either 4-weeks or 8-weeks depending on the prior parent trial, and up to a 4-year Treatment Period. The anticipated duration is 50 months and 2 weeks (4 years and 2.5 months).

Participants will receive an intravenous infusion of del-desiran at the clinical study site every 8 weeks for a total of 7 doses per year. The final dose will occur at Year 4, Visit 7, followed by a final assessment 8 weeks after the last dose.

An additional subgroup of de novo participants will also be included in a Fixed-Dose PK cohort.

An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Вмешательства

• Препарат Del-desiran (AOC 1001)
  Del-desiran will be administered by intravenous (IV) infusion.

Первичные конечные точки

• TEAEs [Срок оценки: From enrollment to the end of treatment at 4 years]

Критерии участия

FROM A PARENT STUDY

Критерии включения

• Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator.

Критерии исключения

• Breastfeeding, pregnancy, or intent to become pregnant during the study.
• Unwilling to comply with contraceptive requirements.
• Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.

FIXED-DOSE PK COHORT

Критерии включения

• Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.

Критерии исключения

• Breastfeeding, pregnancy, or intent to become pregnant during the study
• Unwilling to comply with contraceptive requirements
• Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
• Diabetes that is not adequately controlled
• History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded
• Body Mass Index > 35 kg/m2 at Screening
• Recently treated with an investigational drug or biological agent

Note: Additional protocol defined Inclusion and Exclusion criteria apply

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 19 центров

• University of California, Los Angeles (UCLA) — Los Angeles
• Stanford University — Stanford
• University of Colorado — Denver
• University of Florida — Gainesville
• University Research Center of South Florida — Tampa
• Indiana University (IU) — Indianapolis
• Kansas University Medical Center — Kansas City
• Kennedy Krieger Institute — Baltimore
• … и ещё 11 центров

Япония · 4 центра

• Aomori Hospital — Aomori
• National Hospital Organization Osaka Toneyama Medical Center — Osaka
• Osaka University Hospital — Osaka
• National Center of Neurology and Psychiatry — Tokyo

Канада · 2 центра

• The Ottawa Hospital — Ottawa
• Montreal Neurological Institute — Montreal

Дания · 2 центра

• Aarhus University Hospital — Aarhus
• Rigshospitalet — Copenhagen

Нидерланды · 2 центра

• Maastricht University Medical Center — Maastricht
• Stichting Radboud Universitair Medisch Centrum — Nijmegen

Великобритания · 2 центра

• University College London Hospital — London
• St. Georges University Hospitals NHS Foundation Trust — London

Франция · 1 центр

• AP-HP Hopital Pitie-Salpetriere — Paris

Германия · 1 центр

• Klinikum der Ludwig-Maximilians-Universitaet Muenchen — München

Италия · 1 центр

• Fondazione Serena Onlus - Centro Clinico NeMO Milano — Milan

Испания · 1 центр

• Hospital Universitario Donostia — San Sebastián

Идентификаторы

NCT: NCT07008469 · AOC 1001-CS4

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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