Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies

Официальное название

An Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20 or ORION-19 Studies (VICTORION-PEDS-OLE)

Обзор

The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 (CKJX839C12301), ORION-13 (CKJX839C12302), ORION-20 (CKJX839C12303) or ORION-19 (CKJX839C12304) studies.

Подробное описание

This is an open-label, single arm, multicenter study designed to evaluate long-term safety and tolerability of inclisiran. In addition, the study will provide participants the opportunity to have continued access to treatment with inclisiran.

Вмешательства

• Препарат Inclisiran
  Solution for injection

Первичные конечные точки

• Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs) [Срок оценки: From Day 1 in the study up to the end of study visit; up to 1080 days]

Вторичные конечные точки (1)

• Percentage and absolute change in LDL-C from baseline in the feeder study to end of study [Срок оценки: Baseline (of feeder study) and Day 1080]

Критерии участия

Key inclusion:

• Male and female participants with a diagnosis of HeFH or HoFH who completed the ORION-16, ORION-13, ORION-20 or ORION-19 studies
• Per investigator's clinical judgment, participant derived benefit from treatment with inclisiran in the ORION-16, ORION-13, ORION-20 or ORION-19 studies

Key exclusion:

• Participants who in the feeder ORION-16, ORION-13, ORION-20, or ORION-19 studies either screen failed or permanently discontinued from the treatment/study for any reason or had serious safety or tolerability issues related to inclisiran treatment
• Any uncontrolled or serious disease, or any medical, physical, or surgical condition, that may either interfere with participation in the clinical study or interpretation of clinical study results, and/or put the participant at significant risk

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 · 5 центров

• Excel Medical Clinical Trials LLC — Boca Raton
• Icahn School of Med at Mt Sinai — New York
• Cincinnati Childrens Hospital MC — Cincinnati
• Childrens Hosp Pittsburgh UPMC — Pittsburgh
• Primary Childrens Medical Center — Salt Lake City

Италия · 4 центра

• Novartis Investigative Site — Milan
• Novartis Investigative Site — Modena
• Novartis Investigative Site — Roma
• Novartis Investigative Site — Roma

Испания · 4 центра

• Novartis Investigative Site — Pamplona
• Novartis Investigative Site — Oviedo
• Novartis Investigative Site — A Coruña
• Novartis Investigative Site — Córdoba

Turkey (Türkiye) · 4 центра

• Novartis Investigative Site — Istanbul
• Novartis Investigative Site — Adana
• Novartis Investigative Site — Ankara
• Novartis Investigative Site — Izmir

Бразилия · 3 центра

• Novartis Investigative Site — Fortaleza
• Novartis Investigative Site — São Paulo
• Novartis Investigative Site — São Paulo

Чехия · 2 центра

• Novartis Investigative Site — Prague
• Novartis Investigative Site — Prague

Франция · 2 центра

• Novartis Investigative Site — Besançon
• Novartis Investigative Site — Toulouse

Германия · 2 центра

• Novartis Investigative Site — Frankfurt am Main
• Novartis Investigative Site — Freiburg im Breisgau

Греция · 2 центра

• Novartis Investigative Site — Athens
• Novartis Investigative Site — Athens

Израиль · 2 центра

• Novartis Investigative Site — Jerusalem
• Novartis Investigative Site — Ramat Gan

Ливан · 2 центра

• Novartis Investigative Site — Beirut
• Novartis Investigative Site — Beirut

Малайзия · 2 центра

• Novartis Investigative Site — Sungai Buloh
• Novartis Investigative Site — Kuala Lumpur

Нидерланды · 2 центра

• Novartis Investigative Site — Amsterdam
• Novartis Investigative Site — Rotterdam

Польша · 2 центра

• Novartis Investigative Site — Gdansk
• Novartis Investigative Site — Lodz

Россия · 2 центра

• Novartis Investigative Site — Kemerovo
• Novartis Investigative Site — Moscow

ЮАР · 2 центра

• Novartis Investigative Site — Bloemfontein
• Novartis Investigative Site — Cape Town

Аргентина · 1 центр

• Novartis Investigative Site — Formosa

Канада · 1 центр

• Novartis Investigative Site — Québec

Венгрия · 1 центр

• Novartis Investigative Site — Pécs

Иордания · 1 центр

• Novartis Investigative Site — Irbid

Норвегия · 1 центр

• Novartis Investigative Site — Oslo

Словакия · 1 центр

• Novartis Investigative Site — Poprad

Словения · 1 центр

• Novartis Investigative Site — Ljubljana

Швейцария · 1 центр

• Novartis Investigative Site — Geneva

Тайвань · 1 центр

• Novartis Investigative Site — Taipei

Великобритания · 1 центр

• Novartis Investigative Site — Middlesex

Идентификаторы

NCT: NCT05682378 · CKJX839C12001B · 2022-002316-23

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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