Integrating Point of Care Testing (POCT) For Newborn Screening and Early Care for Sickle Cell Disease in Yopougon, Côte d'Ivoire

Обзор

The goal of this clinical trial is to learn if a multifaceted intervention composed of Gazelle-Multispectral sickle cell disease (SCD) point of care testing (POCT) and early initiation of comprehensive SCD care in children with SCD disease aged 0 - 6 months can improve their clinical outcomes. The main questions it aims to answer are: * Does the intervention lower the number of SCD-related illnesses? * Does the intervention lower the illness incidence defined as seeking health care at any health facility - with or without treatment - for any episode related to a SCD related illness? * Does the intervention lower all-cause death rate (at the end of 1, 2, 3 and 3.5 years of follow-up)? Researchers will compare the multifaceted intervention results with those of historical data (based on erratic SCD testing and treatment) from the region. Participants will undergo a SCD screening test using the Gazelle Multispectral platform and if positive they will undergo confirmatory testing with HemoTypeSC™. Participants with SCD will receive early comprehensive clinical interventions i.e. standard administration of antibacterial and antimalarial prophylaxis, vaccinations for pneumococcal and Haemophilus influenzae type b (Hib), hydroxyurea, parental education about the need for regular and, if necessary, urgent medical care.

Подробное описание

Sickle cell disease (SCD) is an inherited life-threatening hematological disorder. Globally, every year, an estimated 300,000 children are born with sickle cell anemia (SCD - SS, homozygous form), the most severe form of SCD. About 75% of these births occur in Sub-Saharan Africa (SSA), of whom 50-90% die before their 5th birthday. In high income countries, it has been shown that newborn SCD screening and early comprehensive clinical interventions such as penicillin prophylaxis, safe blood transfusion, hydroxyurea and acute medical care, substantially reduces SCD morbidity and mortality. In low and middle-income countries (LMICs), the implementation of newborn SCD screening and early clinical interventions are largely poor.

The most commonly used newborn SCD screening techniques are isoelectric focusing (IEF), capillary electrophoresis (CE) and cation-exchange high performance liquid chromatography (HPLC). These techniques have a high cost, require skilled staff and a reliable electricity supply, and are therefore not suitable for remote settings in LMICs. In order to overcome these challenges, several point of care tests (POCTs) have been recently developed. The Gazelle Multispectral platform is the first SCD POCT that can be used to identify and quantify hemoglobin variants in newborns and people of any age.

In Côte d'Ivoire, as in many other African countries, the implementation of newborn SCD screening and early clinical interventions are lagging behind, and subsequently SCD morbidity and mortality are high. In this project, the investigators will implement a novel multifaceted intervention composed of: 1) integration of Gazelle-Multispectral POCT for newborn SCD screening; and 2) initiation of early comprehensive clinical interventions i.e. standard administration of antibacterial and antimalarial prophylaxis, vaccinations for pneumococcal and Haemophilus influenzae type b (Hib), hydroxyurea, parental education about the need for regular and, if necessary, urgent medical care. The investigators will assess the effectiveness and cost of the multifaceted intervention. The effectiveness will be assessed based on the 2.5 - 3.5 years follow-up clinical records of SCD morbidity and mortality (clinical outcomes). The effectiveness data will be compared descriptively with historical data from the region, and used to inform SCD policy in Côte d'Ivoire. Historical data will be obtained from published literature in Cote d'Ivoire or nearby Countries such as Ghana. After three and a half years, the investigators aim to hand over the project to the Ministry of Health of Côte d'Ivoire for integration into its routine health care activities and potential nation-wide scale up.

Вмешательства

• Комбинированный продукт Gazelle-Multispectral point of care testing for newborn sickle cell disease screening; and initiation of early comprehensive clinical interventions
  A novel multifaceted intervention composed of: 1) integration of Gazelle-Multispectral point of care testing (POCT) for newborn sickle cell disease (SCD) screening; and 2) initiation of early comprehensive clinical interventions i.e. standard administration of antibacterial and antimalarial prophylaxis, vaccinations for pneumococcal and Haemophilus influenzae type b (Hib), hydroxyurea and parental education about SCD medical care.

Первичные конечные точки

• The clinical outcomes of implementing a novel multifaceted intervention composed of a Gazelle SCD POCT, and early initiation of comprehensive SCD care are: [Срок оценки: Baseline, 1 year, 2 years, 3 years, and 3.5 years]
• The clinical outcomes of implementing a novel multifaceted intervention composed of a Gazelle SCD POCT, and early initiation of comprehensive SCD care are: [Срок оценки: Baseline, 1 year, 2 years, 3 years, and 3.5 years]

Вторичные конечные точки (2)

• The implementation costs and the cost-effectiveness of a novel multifaceted intervention composed of a Gazelle SCD POCT, and early initiation of comprehensive SCD care are: [Срок оценки: Baseline, 1 year, 2 years, 3 years, and 3.5 years]
• The budget impact (per year) associated with early comprehensive SCD care for all children who test positive in Côte d'Ivoire [Срок оценки: Baseline, 1 year, 2 years, 3 years, and 3.5 years]

Критерии участия

Критерии включения

• Children aged 0 - 6 months old identified at selected health facilities within the study area
• Babies whose age plus the gestation period reaches 37 weeks or more
• Children who did not receive a blood transfusion within the past 90 days
• Children whose parents or guardians have consented to participate in the study by signing a consent form

Критерии исключения

• Children already diagnosed with SCD
• Children that are participating in another clinical research project
• Children that are critically ill and require emergency treatment

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Организация здравоохранения

Центры проведения

Côte d’Ivoire · 1 центр

• Yopougon SCD Center of Excellence — Yopougon

Идентификаторы

NCT: NCT07488520 · AO_2025-00105

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗