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Идёт набор NCT06224413

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

— Наблюдательное Cerebral Adrenoleukodystrophy (CALD)

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: No Intervention.
Кому может быть актуально
Состояния в реестре: Cerebral Adrenoleukodystrophy (CALD). Базовые параметры: Без ограничений · Мужчины.
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 Postmarketing, Prospective, Multicenter, Observational, Long-Term Safety and Effectiveness Registry Study of Patients With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel (Stargazer)

Обзор

The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major functional disability (MFD)-free survival over time in participants with more advanced early active CALD. All enrolled participants with CALD treated with eli-cel in the post-marketing setting will be followed in this study for 15 years. No investigational drug product will be administered in this study. This study will enroll 120 participants with CALD treated with eli-cel in the post-marketing setting. A subpopulation of 24 participants with more advanced early active CALD will be specifically enrolled as required by the US FDA as a condition of accelerated approval and will be considered as a separate cohort for effectiveness outcomes.

Вмешательства

  • Другое No Intervention
    This is non-interventional study.

Первичные конечные точки

  • Number of Participants who Experience Each Individual Adverse Events of Interest (AEIs) [Срок оценки: Through 15 years postinfusion]
  • Number of Participants with Newly Diagnosed Malignancies [Срок оценки: Through 15 years postinfusion]
  • Number of Participants with Insertional Oncogenesis [Срок оценки: Through 15 years postinfusion]
  • Major Functional Disability (MFD)-Free Survival [Срок оценки: Through 15 years postinfusion]
Вторичные конечные точки (9)
  • Overall Survival (OS) [Срок оценки: Through 15 years postinfusion]
  • Number of Participants with Serious Adverse Events (SAEs) [Срок оценки: Through 15 years postinfusion]
  • Number of Participants with Eli-cel Related AEs [Срок оценки: Through 15 years postinfusion]
  • Number of Participants with Presence of Insertional Oncogenesis in Subpopulation with Newly Diagnosed Hematologic Malignancy [Срок оценки: Through 15 years postinfusion]
  • Number of Participants with Complete Remission in Subpopulation with Newly Diagnosed Hematologic Malignancy [Срок оценки: Through 15 years postinfusion]
  • Relapse-free Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy [Срок оценки: Through 15 years postinfusion]
  • Overall Survival in Subpopulation with Newly Diagnosed Hematologic Malignancy [Срок оценки: Through 15 years postinfusion]
  • Number of Participants with Clinically Significant Abnormalities by Karyotyping in Bone Marrow Aspirate in Subpopulation with Newly Diagnosed Hematologic Malignancy [Срок оценки: Through 15 years postinfusion]
  • Number of Participants with Clinically Significant Abnormalities on Fluorescence In-situ Hybridization (FISH) and Next Generation Sequencing (NGS) in Subpopulation with Newly Diagnosed Hematologic Malignancy [Срок оценки: Through 15 years postinfusion]

Критерии участия

Критерии включения

Participants who fulfill the following criteria will be eligible for inclusion in this Registry Study.

  • Participant must be treated with eli-cel in the post marketing setting at a center in the United States (US) that participates in the Registry Study.
  • Participant must have provided an informed consent and/or assent to participate in Center for International Blood and Marrow Transplant Research (CIBMTR) registry.
  • Participant must have provided an informed consent and/or assent to participate in the Registry Study.
  • Participant must receive follow up care by a US-based physician with the ability to submit REG-502 data.

Registry Study Subpopulation inclusion:

Twenty-four of the 120 patients in the Registry Study must meet the following inclusion criteria which will be used to create the more advanced early active CALD subpopulation:

  • Participant must meet the above inclusion criteria and have a Loes score of 4.5 through 9.0 with GdE+ (gadolinium enhancement positivity) from an MRI performed before treatment with eli-cel and with NFS of 0 or 1 at baseline.

Критерии исключения

There are no exclusion criteria for this Registry Study.

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

США · 3 центра
  • Boston Children's Hospital — Boston
  • Children's Hospital of Philadelphia — Philadelphia
  • UT Southwestern Medical Center — Dallas

Идентификаторы

NCT: NCT06224413 · REG-502

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗