Меню
Набор скоро начнётся NCT07752121

First-Line Luspatercept in Transfusion-Dependent Lower-Risk Myelodysplastic Neoplasms

— Наблюдательное Myelodysplastic Neoplasms

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: Luspatercept.
Кому может быть актуально
Состояния в реестре: Myelodysplastic Neoplasms. Базовые параметры: от 18 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
Германия
Следующий шаг
Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

Не всё понятно в терминах? Прочитайте наш гид для пациентов →

Официальное название

A Prospective, Multicenter, Non-Interventional Study Assessing First-line Luspatercept in Anemic Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Neoplasms Requiring Red Blood Cell Transfusions

Обзор

This study will observe adults with lower-risk myelodysplastic neoplasms (MDS) who have anemia requiring regular red blood cell transfusions and who are prescribed first-line luspatercept as part of routine medical care. The study will follow participants for up to 2 years to understand how often treatment leads to periods without transfusions, changes in hemoglobin levels, health-related quality of life, and safety outcomes. Information on treatment use and outcomes in routine clinical practice in Germany will also be collected.

Вмешательства

  • Препарат Luspatercept
    As per product label

Первичные конечные точки

  • Percentage of participants achieving red blood cell transfusion independence (RBC-TI) for at least 8 consecutive weeks [Срок оценки: Up to Week 24]
Вторичные конечные точки (12)
  • Percentage of participants achieving red blood cell transfusion independence for at least 12 consecutive weeks [Срок оценки: Up to Week 48]
  • Percentage of participants achieving red blood cell transfusion independence for at least 16 consecutive weeks [Срок оценки: Up to Week 48]
  • Mean change from baseline in hemoglobin concentration [Срок оценки: Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years)]
  • Percentage of participants with hemoglobin increase of at least 1.5 g/dl from baseline [Срок оценки: Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years)]
  • Percentage of participants achieving >50% reduction in transfusion burden compared with baseline [Срок оценки: Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years)]
  • Time from first luspatercept administration to first on-treatment red blood cell transfusion. [Срок оценки: Up to 2-years]
  • Time to red blood cell transfusion independence for at least 8 consecutive weeks [Срок оценки: Up to Week 24]
  • Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 56 consecutive days. [Срок оценки: Up to 2-years]
  • Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 84 consecutive days. [Срок оценки: Up to 2-years]
  • Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 112 consecutive days. [Срок оценки: Up to 2-years]
  • Percentage of participants achieving hematologic improvement-erythroid response according to International Working Group (IWG) 2006 criteria [Срок оценки: Up to Week 48]
  • Number of participants with adverse events [Срок оценки: Up to 2-years]

Критерии участия

Критерии включения

  • Males and females ≥18 years of age at enrollment.
  • Documented diagnosis of myelodysplastic neoplasms according to World Health Organisation (WHO) 2022 or WHO 2016 classification meeting International Prognostic Scoring System-Revised (IPSS-R) criteria for very low-, low-, or intermediate-risk disease.
  • Documented red blood cell transfusion dependence of ≥2 units of red blood cells within the 8 weeks preceding Day 1 treatment initiation.
  • First-line treatment based on the approved luspatercept label and decision for treatment with luspatercept as assessed by the treating physician prior to study participation
  • Provision of written informed consent.

Критерии исключения

  • Contraindication according to the Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC).
  • Parallel participation in an interventional clinical trial (except follow-up phase as specified in protocol).

Patients who have completed their participation in an interventional clinical trial or who are not receiving any study drug anymore and who are only in the follow-up phase can be enrolled. For blinded studies, the study drug administered needs to be known at the time of enrolment.

  • Concurrent malignancy requiring treatment.

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Когортное

Центры проведения

Германия · 1 центр
  • Universitätsmedizin der Johannes Gutenberg-Univ. III. Med. Klinik Hämatologie/Onkologie — Mainz

Идентификаторы

NCT: NCT07752121 · CA056-1175

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗