Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: sepofarsen, Placebo IVT.
- Кому может быть актуально
- Состояния в реестре: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders. Базовые параметры: от 6 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- США, Бельгия, Бразилия, Канада, Франция +4
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
Не всё понятно в терминах? Прочитайте наш гид для пациентов →
Официальное название
A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene
Обзор
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.
Подробное описание
This is a double-masked, randomized, placebo-controlled, paired-eye study in which one eye of each subject will serve as a control.
At the start of the study the two eyes of each subject will be randomized such that one eye receives sepofarsen and the other eye receives placebo for the first year. In the second year, for all subjects, the eye that was randomized to receive sepofarsen will continue to receive sepofarsen. For the eye that was randomized to placebo in the first year, treatment in the second year will be allocated, as follows: 50% of the eyes will continue to receive placebo, and 50% of the eyes will receive sepofarsen.
Sepofarsen and placebo will be administered via intravitreal injection every 6 months.
Вмешательства
- Препарат sepofarsen
RNA antisense oligonucleotide for intravitreal injection - Другое Placebo IVT
Placebo with identical appearance to sepofarsen
Первичные конечные точки
- Change from baseline in Best-Corrected Visual Acuity (BCVA) [Срок оценки: 12 Months]
Вторичные конечные точки (12)
- Eye-specific Patient Global Impression of Change (PGI-C) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) [Срок оценки: Month 12]
- Change from baseline in Low Luminance Visual Acuity (LLVA) based on FrACT between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) [Срок оценки: Month 12]
- Change from baseline in retinal sensitivity as measured by dark-adapted Full-Field Stimulus Test (FST) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) [Срок оценки: Month 12]
- Change from baseline in Contrast Sensitivity (CS) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) based on qCSF [Срок оценки: Month 12]
- Change from baseline in retinal sensitivity as measured by light-adapted Full-Field Stimulus Test (FST) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) [Срок оценки: Month 12]
- Response in BCVA (FrACT), defined as an improvement of at least 0.2 logMAR from baseline at 12 months and compared between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs), evaluated by the percentage of eyes that achieve this improvement. [Срок оценки: Month 12]
- Response in BCVA (FrACT), defined as an improvement of at least 0.3 logMAR from baseline at 12 months and compared between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs), evaluated by the percentage of eyes that achieve this improvement. [Срок оценки: Month 12]
- Response in Low Luminance Visual Acuity (LLVA) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement. [Срок оценки: Month 12]
- Response in Full-Field Stimulus Test (FST) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement. [Срок оценки: Month 12]
- Response in Patient Global Impression of Change (PGI-C) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement. [Срок оценки: Month 12]
- Response in Contrast Sensitivity (CS) defined as clinically relevant improvement between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) at 12 months, evaluated by the percentage of eyes that achieve this improvement. [Срок оценки: Month 12]
- Response defined as a clinically relevant improvement according to a multimodal assessment in TEs compared to the corresponding response in PCEs at 12 months [Срок оценки: Month 12]
Критерии участия
Критерии включения
- Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A>G mutation in CEP290.
- Adults: >=18 years / Minors: 6 to <18 years.
- BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equivalent 20/50) to +2.9 logMAR based on quantifiable, reliable FrACT. LP subjects with documented evidence of prior better vision eligible.
- Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline.
- Detectable ONL in the macular area as determined by the CRC at Screening.
Критерии исключения
- Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes.
- Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible.
- Presence of unstable concurrent CME, or subject started on (or changed dose of) topical or systemic carbonic anhydrase inhibitor treatment in the 3 months prior to enrollment. CME is allowed if stable for 3 months (with or without treatment).
- Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale.
- Any prior receipt of genetic (RNA or DNA therapy) or stem-cell therapy for ocular or non-ocular disease, including sepofarsen.
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 · 5 центров
- UCSF Wayne and Gladys Valley Center for Vision — San Francisco
- University of Miami - Bascom Palmer Eye Institute — Miami
- University of Iowa — Iowa City
- University of Minnesota Medical School — Minneapolis
- University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics — Philadelphia
Германия · 3 центра
- Justus-Liebig Universität - Department of Ophthalmology — Giessen
- Klinikum der Ludwig-Maximilian Universität München — München
- University of Tuebingen - Inst. for Ophthalmic Research — Tübingen
Бразилия · 2 центра
- INRET Clínica/ Santa Casa de Misericórdia de Belo Horizonte — Belo Horizonte
- Federal University of São Paulo - Hospital São Paulo (UNIFESP-HSP) — São Paulo
Канада · 2 центра
- University of Alberta — Edmonton
- The Hospital for Sick Children - SickKids — Toronto
Бельгия · 1 центр
- Universitair Ziekenhuis Gent (UZ) — Ghent
Франция · 1 центр
- Centre de maladies rares CHNO des Quinze Vingt — Paris
Нидерланды · 1 центр
- Radboud Universitair Medisch Centrum — Nijmegen
Испания · 1 центр
- Hospital Sant Joan de Déu (SJD Barcelona Children's Hospital) — Barcelona
Великобритания · 1 центр
- Moorfields Eye Hospital NHS Foundation Trust — London
Идентификаторы
NCT: NCT06891443 · SB-110-007 · 2024-518378-14-00