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Идёт набор NCT05329935

Congenital Athymia Patient Registry

— Наблюдательное Complete DiGeorge Anomaly Complete DiGeorge Syndrome Congenital Athymia

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: Cultured Thymus Tissue.
Кому может быть актуально
Состояния в реестре: Complete DiGeorge Anomaly, Complete DiGeorge Syndrome, Congenital Athymia. Базовые параметры: 0 лет — 21 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

Congenital Athymia Patient Registry of RETHYMIC

Обзор

This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC. Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).

Подробное описание

This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC.

Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).

Currently, there is a single site where patients are being treated with RETHYMIC. The Investigator will be responsible for recruiting patients who have recently had (i.e., within the past 60 days) or are scheduled to have this treatment within 30 days and obtaining signed informed consent (and assent as applicable). The Investigator will collect baseline medical history and clinical data information for each enrolled subject. The start of follow-up (day 1) begins on the day following treatment surgery. Clinical data and flow cytometry test results (when conducted) will be abstracted from the medical records by Clinical Staff at baseline and during follow-up at predefined intervals.

Вмешательства

  • Биопрепарат Cultured Thymus Tissue
    Product will be surgically administered into the quadriceps

Первичные конечные точки

  • Vital Status [Срок оценки: 12 months post treatment with RETHYMIC.]
  • Flow cytometry including total and naïve CD3, CD4, and CD8 counts [Срок оценки: Immediately after the intervention/procedure/surgery]
Вторичные конечные точки (1)
  • AESI classified by MedDRA coding, severity and grade [Срок оценки: Immediately after the intervention/procedure/surgery]

Критерии участия

Критерии включения

  • Pediatric patients diagnosed with Congenital Athymia:
  • Who are scheduled for implantation with RETHYMIC within the next 30 days, or who were treated with RETHYMIC within the previous 60 days.
  • Who have provided written informed consent directly, or written informed consent has been provided by the patient, the parent, or legal guardian.

Критерии исключения

  • Written informed consent cannot be obtained.

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

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

США · 1 центр
  • Duke University School of Medicine — Durham

Публикации

  • RETHYMIC US Prescribing Information, 2021.

Идентификаторы

NCT: NCT05329935 · RVT-802-4001

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗