Идёт набор NCT07215975

A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East

— Наблюдательное β-thalassemia

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: β-thalassemia. 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: Oman, Саудовская Аравия
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

REal-World Application of Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East (RELATE): A Non-interventional Retrospective and Prospective Observational Study

Обзор

The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East

Вмешательства

Препарат Luspatercept
According to the product label

Первичные конечные точки

Change in transfusion burden [Срок оценки: Baseline and up to week 144]
Change in mean pre-transfusion hemoglobin level [Срок оценки: Baseline and up to week 144]

Вторичные конечные точки (12)

Change in transfusion-related visits [Срок оценки: Baseline and up to week 144]
Proportion of participants achieving ≥33% reduction in red blood cell (RBC) transfusion burden (number of RBC units transfused) plus a reduction of ≥2 units [Срок оценки: Baseline and up to week 144]
Proportion of participants achieving ≥50% reduction in red blood cell transfusion burden plus a reduction of ≥2 units [Срок оценки: Baseline and up to week 144]
Time from first luspatercept dosing date to the first erythroid response [Срок оценки: Baseline and up to week 144]
Time from the date the erythroid response is first observed until the last day of response [Срок оценки: Baseline and up to week 144]
Participant pretransfusion hemoglobin level [Срок оценки: Baseline and up to week 144]
Participant baseline transfusion burden [Срок оценки: Baseline]
Proportion of participants without red blood cell transfusion during any consecutive 12-week or 24-week treatment period [Срок оценки: Baseline and up to week 144]
Time from first luspatercept dosing date to first onset of red blood cell-transfusion independence ≥12 weeks and ≥24 weeks [Срок оценки: Baseline and up to week 144]
Time from the date a 12-week and 24-week red blood cell-transfusion independence is first observed until the date the participant has a subsequently documented red blood cell transfusion [Срок оценки: Baseline and up to week 144]
Change in mean serum ferritin (SF) level [Срок оценки: Baseline and up to week 144]
Change in proportion of participants with mean serum ferritin <1,000, 1000-2500, and >2,500 μg/L [Срок оценки: Baseline and up to week 144]

Критерии участия

Критерии включения

Male or female participants of any race aged at least 18 years at time of initiation of luspatercept treatment
Participants with documented diagnosis of transfusion-dependent β-thalassemia (TDT).
Participants who have been initiated on treatment with luspatercept as per the product's Summary of Product Characteristics (SmPC) no longer than 12 months prior to informed consent signature, and for whom therapy is ongoing.
Participants for whom the decision to prescribe luspatercept treatment is clearly separated from the physician's decision to include the participant in the current study.
Participants who have provided signed informed consent for participating in the study and for collecting and analyzing medical data pertinent to the objectives of this study

Критерии исключения

Participants that meet any of the contraindications to the administration of luspatercept as outlined in the latest version of the locally approved SmPC.
Participants who are currently receiving or are planned to receive treatment with any investigational drug/device/intervention or who have received any investigational product within 1 month or 5 half-lives of the investigational agent (whichever is longer) prior to luspatercept therapy initiation.
Participants who are currently pregnant, breastfeeding, or planning a pregnancy during the study observation period.
Participants who have not provided signed informed consent for participating in the study and for collecting and analysing medical data pertinent to the objectives of this study.

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

Здоровые добровольцы: Нет