A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting
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: Prademagene Zamikeracel.
- Кому может быть актуально
- Состояния в реестре: Recessive Dystrophic Epidermolysis Bullosa. Базовые параметры: Без ограничений · 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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Обзор
The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who were treated with pz-cel. The main questions it aims to answer are: * The long-term safety profile of pz-cel. * The wound healing durability of pz-cel. * The occurrence of all malignancies after treatment with pz-cel, regardless of type or location, in individuals who received pz-cel treatment for RDEB in the post-marketing setting. Participants will then be followed annually up to 15 years post-treatment. Assessments will occur via remote data collection. If the participants provider determines an office visit is necessary after the first year, some of this data can be collected in person as well. Annual assessments will include, but are not limited to: * Review of Adverse Events (AEs), Serious Adverse Events (SAEs), hospitalizations, and any evidence of malignancies and potential retroviral infection * Wound-healing durability data collection via the modified Caregiver Global Impression of Change for Wound Healing * Any changes in concomitant medications and procedures.
Вмешательства
- Комбинированный продукт Prademagene Zamikeracel
ZEVASKYN® (prademagene zamikeracel) is a type of gene therapy made from your own skin cells that is surgically applied to treat wounds in both children and adults who have recessive dystrophic epidermolysis bullosa (RDEB).
Первичные конечные точки
- The number and incidence of treatment-related malignancies. [Срок оценки: From enrollment to 15 years post-treatment]
- The number and incidence of treatment-emergent SAEs, including systematic and wound specific SAEs [Срок оценки: From enrollment to 15 years post-treatment]
- The number and incidences of treatment-related SAEs related to pz-cel, including systematic and wound-specific SAEs. [Срок оценки: From enrollment to 15 years post-treatment]
- The number and incidence of positive RCR testing results required for AEs and SAEs where retroviral infection is a consideration [Срок оценки: From enrollment to 15 years post-treatment]
Критерии участия
Критерии включения
- Willing and able to give consent/assent; if under the age of 18, guardian(s) is(are) willing and able to give consent.
- Patients who received treatment with prademagene zamikeracel in the post-marketing setting.
Критерии исключения
- Inability to properly follow the protocol as determined by the Principal Investigator (PI).
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Только случаи
Центры проведения
США · 4 центра
- Lucile Packard Children's Hospital — Palo Alto
- Children's Hospital Colorado — Aurora
- Ann & Robert H. Lurie Children's Hospital of Chicago - Department of Dermatology — Chicago
- University of Texas Medical Branch — Galveston
Идентификаторы
NCT: NCT07700966 · Pz-cel-RY-401