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Идёт набор NCT07016750

A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa

Фаза III С лечением Dystrophic Epidermolysis Bullosa DEB - Dystrophic Epidermolysis Bullosa Recessive Dystrophic Epidermolysis Bullosa Dominant Dystrophic Epidermolysis Bullosa

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: KB803, Placebo.
Кому может быть актуально
Состояния в реестре: Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa. Базовые параметры: от 6 мес. · 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 Double-Blind Crossover of KB803 and Matched Placebo for the Treatment and Prevention of Corneal Abrasions in Dystrophic Epidermolysis Bullosa

Обзор

KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).

Подробное описание

Subjects participating in the Sponsor's natural history study (protocol NHS) for at least 12 weeks and that meet all other eligibility criteria will be randomized 1:1 to receive ophthalmic administration of B-VEC three times a week or placebo three times a week for the first 12 week intervention period. During the second 12-week intervention period subjects will be crossed-over to receive the alternative Investigational Product (IP) for an additional 12 weeks. IP will be administered in the home setting by a trained designee. The Investigator (or designee) will contact subjects (or their parent/legal guardian) to assess for any adverse events or changes in medications, treatments, or procedures. Subjects (or their parent/legal guardian) will complete weekly symptom diaries and monthly questionnaires to document corneal abrasion symptoms and frequency and gauge overall subject (or parent/legal guardian) impressions of disease severity and disease/symptom changes during the study.

Вмешательства

  • Биопрепарат KB803
    Ophthalmic suspension of replication-defective, non-integrating herpes simplex virus (HSV-1) expressing the human collagen VII protein
  • Препарат Placebo
    Vehicle

Первичные конечные точки

  • To evaluate the safety and tolerability of KB803. [Срок оценки: 24 weeks]
  • To evaluate the effect of ophthalmic KB803 on frequency of corneal abrasion symptoms. [Срок оценки: 24 weeks]
Вторичные конечные точки (1)
  • To evaluate the effect of ophthalmic KB803 on eye pain. [Срок оценки: 24 weeks]

Критерии участия

Критерии включения

  • The subject and/or their parent/legal guardian must provide informed consent/assent and must be able to and willing to follow study procedures and instructions.
  • Age 6 months or older at time of informed consent/assent.
  • Confirmed diagnosis of DEB with a mutation in the COL7A1 gene.
  • Meets minimum corneal abrasion symptom frequency in the NHS study.

Критерии исключения

  • Initiation of any new treatment regimen or change in treatment for ocular disease during the run-in period except for preservative free topical antibiotics or artificial tears/lubricants associated with standard of care treatment of corneal abrasions.
  • Treatment with an investigational agent or off-label ophthalmic use of an approved product during the run-in period or planned use during the study (Exceptions may be approved by the medical monitor on a case-by-case basis).
  • Any condition that, in the opinion of the Investigator, would impact the completion of all study-related assessments, interfere with the administration of study drug, and/or poses an additional risk to the subject.
  • Women who are pregnant or nursing.
  • Subject who is unwilling to comply with contraception requirements per protocol.
  • Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение
Рандомизированное
Модель
Перекрёстный дизайн
Маскирование
Четверное слепое
Основная цель
Лечение

Центры проведения

США · 5 центров
  • Mission Dermatology Center — Rancho Santa Margarita
  • Skin Research Institute — Coral Gables
  • UMass Memorial Medical Center — Worcester
  • Cincinnati Children's Hospital Medical Center — Cincinnati
  • Texas Dermatology and Laser Specialists — San Antonio

Идентификаторы

NCT: NCT07016750 · KB803-EYE-01

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗