Study to Evaluate the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency

Официальное название

An Open-Label Study Evaluating the Safety and Efficacy of CSB-001 Ophthalmic Solution 0.1% in Subjects With Limbal Stem Cell Deficiency

Обзор

This study will enroll subjects with qualifying limbal stem cell deficiency (LSCD). All subjects will receive CSB-001 investigational drug in either one or both study eyes. The study is comprised of two identical phases (Dosing Phase I and II) of test article dosing separated by a 31- to 40-day period, the Dosing Holiday, where no test article is administered. Dosing Phase II is followed by an observational, noninterventional phase (Observation Phase).

Вмешательства

• Биопрепарат CSB-001 Ophthalmic Solution 0.1%
  CSB-001: human recombinant dHGF (5-amino acid deleted hepatocyte growth factor)

Первичные конечные точки

• Safety as Assessed by Adverse Event Reporting [Срок оценки: Day 0 through Week 144]
• Safety as Assessed by Slit-lamp Biomicroscopy [Срок оценки: Day 0 through Week 144]
• Safety as Assessed by Best-corrected Distance LogMAR Visual Acuity [Срок оценки: Day 0 through Week 144]

Вторичные конечные точки (2)

• Efficacy as Assessed by Change in Stage of LSCD [Срок оценки: Day 0 through Week 144]
• Efficacy as Assessed by Change in Best-corrected Distance LogMAR Visual Acuity [Срок оценки: Day 0 through Week 144]

Критерии участия

Критерии включения

• Have an area of the central 5 mm zone of cornea affected, presence of at least one clinical sign observed by slit-lamp examination with white light and/or cobalt blue light, fluorescein, and a Wratten filter, and at least one clinical sign observed with AS-OCT.
• If dosing with Acthar at time of Screening Visit, must have a history of stable dosing for approximately 8 weeks, plan to maintain the same Acthar treatment dose and regimen, and have demonstrated little to no improvement in severity of LSCD.
• Sponsor written confirmation of qualifying LSCD diagnosis.
• Inflammation associated with LSCD or other non-infectious inflammation that will not interfere with subject's participation.

Критерии исключения

• Any active ocular infection (bacterial, viral, fungal, or protozoan) in either eye.
• Ocular surgery within 30 days of Day 0 where investigator assesses that healing is not considered complete/stable.
• Planned ocular surgery on or before Week 20 visit.
• Presence or history of any ocular or systemic disorder, condition, or procedure that might hinder the efficacy of the study treatment or its evaluation, or could interfere with the interpretation of study results, in the opinion of the investigator.

Note: Other inclusion/exclusion criteria apply.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

США · 6 центров

• Loma Linda University Eye Institute — Loma Linda
• Midwest Cornea Associates, LLC — Carmel
• Francis Price Jr, MD — Indianapolis
• Minnesota Eye Consultants — Minnetonka
• Legacy Devers Eye Institute — Portland
• Stuart A. Terry, MD PA — San Antonio

Идентификаторы

NCT: NCT06452316 · CSB-C23-001

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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