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Набор по приглашению NCT04628585

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

— Наблюдательное Sickle Cell Disease

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: Safety and efficacy assessments.
Кому может быть актуально
Состояния в реестре: Sickle Cell Disease. Базовые параметры: 2 лет — 53 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, Франция
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

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector

Обзор

This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.

Вмешательства

  • Другое Safety and efficacy assessments
    Safety evaluations, disease-specific assessments, and assessments to monitor for long-term complications of autologous transplant

Первичные конечные точки

  • Number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) [Срок оценки: Through 15 years post-drug product infusion]
  • Number of subjects with new or worsening hematologic disorders [Срок оценки: Through 15 years post-drug product infusion]
  • Number of subjects with new or worsening neurologic disorders [Срок оценки: Through 15 years post-drug product infusion]
  • Number of subjects with malignancies [Срок оценки: Through 15 years post-drug product infusion]
Вторичные конечные точки (12)
  • Proportion of subjects with complete resolution of severe VOEs (sVOE-CR) over time through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Proportion of subjects with complete resolution of VOEs (VOE-CR) over time through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Annualized number of severe VOEs over time through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Annualized number of VOEs over time through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Change from parent study baseline in annualized number of severe VOEs over time through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Assessment of total Hb over time post-drug product infusion through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Assessment of non-transfused total Hb over time post-drug product infusion through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Assessment of HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Assessment of HbAT87Q percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Assessment of non-HbS percentage of non-transfused total Hb over time post-drug product infusion through Year 15 [Срок оценки: Through 15 years post-drug product infusion]
  • Change from parent study baseline through Year 15 in hemolysis markers [Срок оценки: Through 15 years post-drug product infusion]
  • Change from parent study baseline through Year 15 in markers of iron stores [Срок оценки: 15 years post-drug product infusion]

Критерии участия

Критерии включения

  • Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s)
  • Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study

Критерии исключения

  • There are no exclusion criteria for this study

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 · 15 центров
  • University of Alabama — Birmingham
  • UCSF Benioff Children's Hospital Oakland — Oakland
  • Children's Healthcare of Atlanta — Atlanta
  • Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
  • Warren Grant Magnuson Clinical Center — Bethesda
  • University of Minnesota Masonic Children's Hospital — Minneapolis
  • Hackensack University Medical Center — Hackensack
  • Cohen Children's Medical Center — New Hyde Park
  • … и ещё 7 центров
Франция · 1 центр
  • Hospital Necker — Paris

Публикации

  • Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Treluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poiree M, Monpoux F, Touraine P, Brouzes C, de Monta PMID 35075288

Идентификаторы

NCT: NCT04628585 · LTF-307 · 2019-004266-18 · 2024-513901-30-00

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗