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Идёт набор NCT06789445

A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)

Фаза I / Фаза II С лечением Primary Photoreceptor Disease Retinitis Pigmentosa (RP) Usher Syndrome Inherited Retinal Disease (IRD)

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: OpCT-001, OpCT-001.
Кому может быть актуально
Состояния в реестре: Primary Photoreceptor Disease, Retinitis Pigmentosa (RP), Usher Syndrome, Inherited Retinal Disease (IRD). Базовые параметры: от 18 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
Следующий шаг
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

A Phase 1/2a Study of Subretinal Administration of OpCT-001 Photoreceptor Precursor Cells Derived From iPSCs in Patients With Primary Photoreceptor Disease

Обзор

Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 focuses on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups.

Подробное описание

Phase 1 of the study includes up to 4 planned dose levels to be administered across up to 4 cohorts. Participants are assigned to receive OpCT-001 in an open-label manner.

Dose escalation in Phase 1 is being conducted using a standard 3+3 scheme in which a total of up to 24 legally blind participants (\~3 to 6 per cohort) will receive OpCT-001. Phase 2 is planned to enroll a maximum of 15 participants per cohort in 2 cohorts to evaluate 2 dose levels of OpCT-001 that will be selected based on Phase 1 safety and tolerability data. Phase 2 participants will be randomized 1:1 to either dose-level cohort. Phase 2 participants and investigators and study site personnel outside of the surgical team will be masked to the Phase 2 OpCT-001 dose-level assignments.

Вмешательства

  • Биопрепарат OpCT-001
    Cell therapy product composed of photoreceptor precursor cells derived from human induced pluripotent stem cells (iPSCs).
  • Биопрепарат OpCT-001
    Cell therapy product composed of photoreceptor precursor cells derived from human iPSCs.

Первичные конечные точки

  • Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) through the Week 52 visit [Срок оценки: From OpCT-001 administration through the Week 52 visit]
Вторичные конечные точки (1)
  • Change from baseline over time through the Week 52 visit in the treated portion of the study eye (SE) retina in outer retinal layer thickness as measured by spectral domain optical coherence tomography (SD-OCT) [Срок оценки: From baseline through the Week 52 visit]

Критерии участия

Критерии включения

  • Confirmed genetic diagnosis of primary photoreceptor (PR) disease
  • Best corrected visual acuity (BCVA) in the study eye at Screening for Phase 1: Logmarithm of the minimum angle of resolution (LogMAR) 3.9 to LogMAR 1.3. BCVA at Screening for Phase 2: ETDRS letter score between 20 to 60, inclusive.
  • Retinal structure examination in the study eye demonstrating regions suitable for cell administration.

Критерии исключения

  • Clinically relevant, active ocular inflammation or infection
  • Glaucoma or other significant optic neuropathy
  • Diabetic macular edema or diabetic retinopathy
  • Clinically significant cystoid macular edema
  • In phakic participants: Spherical equivalent refractive error of greater than 8.00 diopters myopia
  • Ocular surgery ≤3 months before Screening
  • Monocular vision (ie, no light perception in the fellow eye)
  • Currently active malignancy, or history of malignancy within 5 years before OpCT-001 administration. Exception: Basal cell carcinoma that has been definitively treated.
  • Any current and active infection (bacterial/viral/fungal) that could put the participant at risk from immunosuppression
  • History of any cell therapy, gene therapy, or retinal implant at any time
  • Previously received a bone marrow or solid organ transplant

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Нерандомизированное
Модель
Последовательный дизайн
Маскирование
Открытое
Основная цель
Лечение

Центры проведения

США · 5 центров
  • University of Southern California /Children's Hospital of Los Angeles — Los Angeles
  • University of Miami, Bascom Palmer Eye Institute — Miami
  • Mid Atlantic Retina/ Wills Eye — Philadelphia
  • Retina Foundation of the Southwest — Dallas
  • University of Wisconsin — Madison

Идентификаторы

NCT: NCT06789445 · OpCT-001-101

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗