Идёт набор NCT06612268

A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

Фаза III С лечением 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: Etavopivat, Placebo.
Кому может быть актуально: Состояния в реестре: Sickle Cell Disease. Базовые параметры: от 12 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, Австралия, Бельгия, Бразилия, Канада +16
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 Global Phase 3, Randomised, Double-blind and Placebo-controlled Study Evaluating the Efficacy and Safety of Etavopivat in Adolescents and Adults With Sickle Cell Disease

Обзор

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.

Вмешательства

Препарат Etavopivat
Etavopivat will be administered orally.
Препарат Placebo
Placebo matching Etavopivat will be administered orally.

Первичные конечные точки

Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact [Срок оценки: Baseline (week 0) to week 52]

Вторичные конечные точки (10)

Change in Haemoglobin (Hb) greater than 1 grams per decilitre (g/dL) [Срок оценки: Baseline (week 0) to week 24]
Time to onset of first adjudicated Vaso-occlusive crisis (VOC) [Срок оценки: Baseline (week 0) to week 52]
Change in standardised T-score on the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 7a Scale [Срок оценки: Baseline (week 0) to week 52]
Change in haemoglobin (Hb) [Срок оценки: Baseline (week 0) to week 52]
Change in lactate dehydrogenase (LDH) [Срок оценки: Baseline (week 0) to week 52]
Change in absolute reticulocyte count [Срок оценки: Baseline (week 0) to week 52]
Change in indirect bilirubin [Срок оценки: Baseline (week 0) to week 52]
Change in distance travelled during the 6-minute walking test (6MWT) [Срок оценки: Baseline (week 0) to week 52]
Participants achieving the threshold for clinically meaningful change (yes/no) in PROMIS fatigue scale 7a [Срок оценки: Baseline (week 0) to week 52]
Participants achieving the threshold for clinically meaningful change (yes/no) in 6MWT [Срок оценки: Baseline (week 0) to week 52]

Критерии участия

Критерии включения

Male or female.
Age 12 years or above at the time of signing the informed consent.
Confirmed diagnosis of sickle cell disease: Documentation of sickle cell disease (SCD) genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing or screening test results from central laboratory. Molecular genotyping is not required. SCD genotype may be determined from the results of haemoglobin (Hb) electrophoresis, high-performance liquid chromatography (HPLC) or similar testing. Note that Hb electrophoresis is performed by the central laboratory at screening.
Have 1-15 episodes of documented vaso occlusive crises (VOC) within the 12 months prior to screening. Documentation must exist in the participant's medical record prior to randomisation. Events based solely on participant recall without supporting documentation should not be counted towards eligibility.
Hb greater than or equal to (≥) 5.0 and less than or equal to (≤) 10.0 g/dL (greater than or equal to (≥) 50 and less than or equal to (≤) 100 g/L) at screening.

Критерии исключения

More than 15 VOCs within the past 12 months prior to screening documented in the participant's medical record. Events based solely on participant recall without supporting documentation should not be counted towards eligibility.
Use of voxelotor or similar agent within 28 days prior to starting study treatment or anticipated need for this agent during the study.
Use of a selectin antagonist (e.g., crizanlizumab, monoclonal antibody or small molecule) within 28 days or 5 half-lives (whichever is longer) prior to starting study treatment or anticipated need for such agents during the study.
Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or greater than or equal to 6 transfusion events in the previous 12 months (i.e., an average of 1 transfusion event every 60 days).
Participants who have received an RBC transfusion for any reason within 60 days of the screening period or 60 days of the randomisation day are only eligible if HbA (adult haemoglobin) less than 10% by Hb electrophoresis is documented prior to starting study treatment.
Receiving or use of concomitant medications that are strong inducers of CYP3A4 (cytochrome p450 3a4) within 2 weeks of starting study treatment or anticipated need for such agents during the study.
Use of erythropoietin or other haematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study.
Receipt of prior cellular-based therapy (e.g., haematopoietic cell transplant, gene modification therapy).
Hepatic dysfunction characterized by:

Alanine aminotransferase (ALT) greater than 4.0 × upper limit of normal (ULN) or
Direct bilirubin greater than 3.0 × ULN.
Participants who are not taking or are unable to take antimalarial prophylaxis at the time of consent and during the study if they live in areas of endemic malaria where prophylaxis is recommended.
Severe renal dysfunction (estimated glomerular filtration rate \[eGFR\] at screening, calculated by the central laboratory greater than 30 mL/min/1.73 m\^ 2) or on chronic dialysis.
Travelled distance on standardized 6MWT below 100m at screening.

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение: Рандомизированное
Модель: Параллельные группы
Маскирование: Четверное слепое
Основная цель: Лечение