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Идёт набор NCT05285917

Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa

Фаза III С лечением Sickle Cell Anemia in Children 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: Hydroxyurea.
Кому может быть актуально
Состояния в реестре: Sickle Cell Anemia in Children, Sickle Cell Disease. Базовые параметры: 6 мес. — 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
Angola
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

BrUOG 419 - Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa (PUSHUP)

Обзор

Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year. Most infants with SCA are born in the low-resource settings of sub- Saharan Africa, where an estimated 50-90% will die before 5 years of age due to lack of early diagnosis and appropriate care. Hydroxyurea is a safe and effective once-daily oral medication that has become the standard of care for the treatment of children with SCA in high-resource settings. There is now a growing body of evidence to support the safety and clinical benefits of hydroxyurea for the treatment of SCA in sub-Saharan Africa. The requirement for frequent laboratory monitoring, uncertainties about appropriate, most effective dosing, and the concern for hematologic laboratory toxicities, however, will continue to limit widespread hydroxyurea utilization and real-world effectiveness. The investigators have recently developed and prospectively evaluated an individualized, pharmacokinetics-guided hydroxyurea dosing strategy for children with SCA that has demonstrated optimal clinical and laboratory benefits with minimal toxicity. In this research study, the investigators aim to extend this precision medicine approach to Africa.

Подробное описание

The Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa (PUSHUP) trial is a prospective, randomized clinical trial of hydroxyurea for 400 children with SCA in Luanda, Angola. The study will prospectively evaluate the safety and efficacy of hydroxyurea with limited laboratory monitoring and will bring precision medicine to children with SCA using several novel features including measurement of hydroxyurea using a battery-powered HPLC machine and individualized dose calculations using an automated computer-based algorithm. The objective of this study is to establish evidence-based guidelines for hydroxyurea in sub-Saharan Africa, including appropriate dosing and laboratory monitoring strategy with the goal of allowing for widespread use of hydroxyurea across sub-Saharan Africa, regardless of clinical or laboratory resources.

Вмешательства

  • Препарат Hydroxyurea
    Hydroxyurea has a narrow therapeutic window such that selection of the correct dose is essential to optimize benefits and avoid toxicity.

Первичные конечные точки

  • Rate of clinical, sickle cell adverse events (grade ≥ 3) as assessed by CTCAE v5.0 [Срок оценки: From start of study treatment through first 12 months of treatment.]
  • Number of non-SCA related adverse events (grade ≥3), including death as assessed by CTCAE v5.0 [Срок оценки: From start of study treatment through treatment completion, approximately 24 months.]
Вторичные конечные точки (1)
  • Hematologic response at 12 months [Срок оценки: From start of study treatment through first 12 months of treatment.]

Критерии участия

Критерии включения

  • Diagnosis of sickle cell anemia (HbSS or HbS/B0-thalassemia)
  • Age 6 months- 12 years of age at enrollment
  • Parent or guardian willing and able to provide written or informed consent
  • Weight ≥ 7.5 kg (temporary exclusion)

Критерии исключения

  • Splenomegaly with evidence of hypersplenism as defined by platelet count <150,000, hemoglobin <5 g/dL or absolute neutrophil count <1.0 x10\^9/L
  • Hydroxyurea use within the past 6 months
  • Blood transfusion within the past 6 months (temporary exclusion)
  • Pregnancy
  • Pre-existing severe hematologic toxicity, as defined by platelet count <80,000, hemoglobin <4 regardless of ANC; hemoglobin <6 AND ARC <100; hemoglobin <7 AND ARC <80 x10\^9/L (temporary exclusion)

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
Открытое
Основная цель
Лечение

Центры проведения

Angola · 1 центр
  • Hospital Geral dos Cajueiros — Luanda

Идентификаторы

NCT: NCT05285917 · BrUOG 419 · U01HL157872

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗