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Идёт набор NCT05991336

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

— Наблюдательное Transfusion-dependent Beta-Thalassemia Gene Therapy

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: Gene therapy.
Кому может быть актуально
Состояния в реестре: Transfusion-dependent Beta-Thalassemia, Gene Therapy. Базовые параметры: 3 лет — 14 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
Китай
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

Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Обзор

The investigate will conduct a cohort study to compare the growth and development, metabolism, lifestyle behavior, and health-related quality of life among three groups: children with transfusion-dependent β-thalassemia (TDT) who have received gene therapy, TDT children with lifelong supportive therapy and healthy children.

Вмешательства

  • Генная терапия Gene therapy
    Autologous edited hematopoietic stem cell transplantation

Первичные конечные точки

  • Growth in pediatric subjects will be assessed by height. [Срок оценки: Up to 5 years.]
  • Growth in pediatric subjects will be assessed by weight. [Срок оценки: Up to 5 years.]
  • Growth in pediatric subjects will be assessed by the head and chest circumference. [Срок оценки: Up to 5 years.]
  • Growth in pediatric subjects will be assessed by interocular distance. [Срок оценки: Up to 5 years.]
  • Growth in pediatric subjects will be assessed by bone density. [Срок оценки: Up to 5 years.]
  • The pubertal status will be measured using by Tanner scale. [Срок оценки: Up to 5 years.]
  • Hormonal levels will be measured by clinical examination. [Срок оценки: Up to 5 years.]
  • Quality of life will be measured using by Pediatric Quality of Life Inventory (PedsQL) at baseline and the end of the study. [Срок оценки: Up to 5 years.]
  • Lifestyle behaviors will be assessed by general questioning. [Срок оценки: Up to 5 years.]

Критерии участия

Критерии включения

Gene therapy group-inclusion

  • Male or female age of 3-14 years
  • TDT Children who have received gene therapy.
  • Subjects who are willing and able to provide written informed consent.

Supportive therapy group-inclusion

  • Gender same as the matched case
  • Age similar to the matched case
  • Children with β-TDT
  • No history of gene therapy or allogeneic hematopoietic stem cell transplantation
  • Subjects who are willing and able to provide written informed consent.

Healthy children group-inclusion

  • Gender same as the matched case
  • Age similar to the matched case
  • Subjects who are willing and able to provide written informed consent.

Критерии исключения

  • Diagnosis of compound α-thalassemia
  • Uncontrolled systemic fungal, bacterial, or viral infection
  • History of malignant solid tumors, myeloproliferative or immunodeficiency diseases
  • Diagnosed with mental illness
  • Patients considered to be ineligible for the study by the investigator for reasons other than the above

Discontinuation of Study :

  • Subjects who are unwilling or unable to continue participating in the study (withdrawal of informed consent) may withdraw from the study
  • Subjects who received gene therapy or allogeneic hematopoietic stem cell transplantation during the study
  • The subject is seriously non-compliant with the study requirements, such as missing 2 consecutive visits
  • Subject lost to follow-up

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

Здоровые добровольцы: Да

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

Китай · 1 центр
  • Regenerative Medicine Center — Тяньцзинь

Идентификаторы

NCT: NCT05991336 · GDH-001

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗