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Universal Newborn Screening For Sickle Cell Disease In Mozambique

17 de julho de 2026 atualizado por: St. Jude Children's Research Hospital
The overarching goal of this study is to evaluate the feasibility of a new methodology that combines three multi-level implementation strategies to optimize the population-level uptake of essential evidence-based, standard of care treatments for infants with sickle cell disease (SCD) in low-resource settings. The study will be done in Mozambique.

Visão geral do estudo

Status

Ainda não está recrutando

Condições

Intervenção / Tratamento

Descrição detalhada

This prospective mixed-methods hybrid effectiveness-implementation feasibility study will evaluate the feasibility and effectiveness of an implementation strategy package designed to improve early diagnosis and care entry for children born with sickle cell disease (SCD) in rural, low-resource settings. The implementation strategy package includes three components: 1) integration of essential products into national supply chain systems, 2) integration of systematic newborn screening using point-of-care-testing into clinical site workflows and 3) linkage of children who screen positive to a PEN-Plus Non-Communicable Disease (NCD) clinic for longitudinal care. Participants will be tracked longitudinally to evaluate protocol adoption over time and clinical outcomes among participating children at 2 years of age.

Primary Objective

  • To test if combining three multi-level implementation strategies can facilitate systematic birth diagnosis and timely linkage to evidence-based care interventions for infants with SCD in under-resourced settings.

Secondary Objectives (Micro level):

  • To test the clinical effectiveness of implementing the BB-SCD through the three combined implementation strategies to prevent excess mortality of children with SCD under the age of 2 years.
  • To evaluate the effectiveness of combining the three implementation strategies to promote care retention among infants with SCD by 2 years of age.

Secondary Objectives (Meso level)

  • To evaluate the institutional coverage and health facility staff adoption of the treatment for infants through the combined implementation strategies.
  • To evaluate the need for a confirmatory test among positive and negative screening results using POCT.
  • To conduct a cost-effectiveness analysis of the combined implementation strategies.

Secondary Objective (Macro level)

  • To evaluate the operational effectiveness of integrating the procurement of SCD consumables into national supply chain systems to achieve sustainable availability, effective last mile delivery to relevant clinics and stockout avoidance.

Tipo de estudo

Intervencional

Inscrição (Estimado)

6750

Estágio

  • Não aplicável

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Contato de estudo

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

  • Filho
  • Adulto
  • Adulto mais velho

Aceita Voluntários Saudáveis

Sim

Descrição

Inclusion Criteria:

  • Children participants: All infants between birth and 6.0 months of age who are born or receive care at secondary-level facilities involved in the UNIQUE study.

  • Children participants will fall into one of two categories:

    • Patient participants: All infants between birth and 6.0 months of age who were screened through the UNIQUE study and tested positive for SCD (HbSS, HbSC, or other form of SCD) or had indeterminate results.
    • Healthy control participants: Infants between birth and 6.0 months of age who screen negative for SCD (HbAA) through the UNIQUE study or tested positive for sickle cell trait (HbAS).
  • Health facility staff participants: Healthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study.
  • Supply chain expert participants: Administrative professionals with experience working in or around the national supply chain systems in Mozambique to support procurement, importation, customs clearance, storage, and in-country distribution of medical products.
  • National public health system expert participants: Administrative professionals with experience working in the national public health system (e.g., Ministry of Health, MISAU) who oversee the delivery of health services to infants in-country, such as neonatal testing and vaccination programs.

Exclusion Criteria:

  • Children participants:

    • Stillbirths.
    • Children who received an erythrocyte (blood) transfusion within 3 months of testing. Exogenous (transfused) HbA could artificially lower the sickle hemoglobin concentration, thus causing false-negative results.
  • Patient participants: none

  • Healthy control participants:

    • Stillbirths.
    • Children who received an erythrocyte (blood) transfusion within 3 months of testing. Exogenous (transfused) HbA could artificially lower the sickle hemoglobin concentration, thus causing false-negative results.
  • Health facility staff participants: none.
  • Supply chain expert participants: none.
  • National public health system expert participants: none.

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Outro
  • Alocação: Não randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Outro: Patient participants
All infants between birth and 6.0 months of age who were screened through the UNIQUE study and tested positive for SCD (HbSS, HbSC, or other form of SCD) or had indeterminate results.
Outro: Point-of-care testing (POCT) -Patient participants
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT).
Outro: Healthy control participants
Infants between birth and 6.0 months of age who screen negative for SCD (HbAA) through the UNIQUE study or tested positive for sickle cell trait (HbAS).
Outro: Point-of-care testing (POCT) - Healthy control participants
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT) and prospectively monitored for survival.
Outro: Health facility staff participants
Healthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study.
Outro: Context Assessment for Community Health (COACH) survey
The COACH survey will collect quantitative data on eight contextual factors that impact a site's ability to implement evidence-based interventions.
Outro: Semi-structured interview
Interview questions will revolve primarily around the current process for SCD screening and care referrals, factors impacting newborn care delivery and screening, and challenges to integrating SCD newborn screening and care referral into standard care delivery.
Outro: Supply chain expert participants
Administrative professionals with experience working in or around the national supply chain systems in Mozambique to support procurement, importation, customs clearance, storage, and in-country distribution of medical products.
Outro: Semi-structured interview
Interview questions will revolve primarily around the current process for SCD screening and care referrals, factors impacting newborn care delivery and screening, and challenges to integrating SCD newborn screening and care referral into standard care delivery.
Outro: National public health system expert participants
Administrative professionals with experience working in the national public health system (e.g., Ministry of Health, MISAU) who oversee the delivery of health services to infants in-country, such as neonatal testing and vaccination programs.
Outro: Semi-structured interview
Interview questions will revolve primarily around the current process for SCD screening and care referrals, factors impacting newborn care delivery and screening, and challenges to integrating SCD newborn screening and care referral into standard care delivery.

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Prazo
Percentage of eligible population screened for Sickle Cell Disease
Prazo: 3 years
3 years
Percentage of new SCD cases linked to care
Prazo: 3 years
3 years

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Comparison of under-2 mortality between participants with SCD whom received longitudinal care and participants who screened negative for SCD
Prazo: 2 years post-screening and therapy
The primary outcome measure will be 'Alive and in care: Yes or No'.
2 years post-screening and therapy
Percentage of SCD cases retained in care by 2 years of age
Prazo: 2 years post-screening and therapy
2 years post-screening and therapy
Percentage of eligible secondary-level clinical wards implementing systematic screening and linkage to longitudinal care for new SCD cases
Prazo: 3 years
3 years
Percentage of eligible health facility staff participants in secondary-level clinical wards implementing systematic screening and linkage to longitudinal care for new SCD cases
Prazo: 3 years
3 years
Comparison of SCD Screening Performance Results from Initial point-of-care (POCT), Gazelle, and Hemoglobin Fractionation
Prazo: 3 years
Investigators will estimate the sensitivity (proportion of diseased subjects that yield a positive test result) and specificity (the proportion of non-diseased subjects that yield a negative test result) of the POCT for detecting SCD compared to the gold standard with basic proportions.
3 years
Cost-Effectiveness of Integrating SCD Consumables into National Supply Chains
Prazo: 2 to 5 years
The standard formula known as incremental cost-effectiveness ratios (ICERs) will be used. ICER is a summary statistic used in economic evaluations to compare the relative value of different healthcare interventions. It is scored by comparing the resulting cost-per-QALY against a predefined threshold, where lower ICERs indicate better value.
2 to 5 years
Cost-Effectiveness of POCT for Early SCD Diagnosis
Prazo: 2 to 5 years
The standard formula known as incremental cost-effectiveness ratios (ICERs) will be used. ICER is a summary statistic used in economic evaluations to compare the relative value of different healthcare interventions. It is scored by comparing the resulting cost-per-QALY against a predefined threshold, where lower ICERs indicate better value.
2 to 5 years
Cost-Effectiveness of Decentralized PEN-Plus SCD Management
Prazo: 2 to 5 years
The standard formula known as incremental cost-effectiveness ratios (ICERs) will be used. ICER is a summary statistic used in economic evaluations to compare the relative value of different healthcare interventions. It is scored by comparing the resulting cost-per-QALY against a predefined threshold, where lower ICERs indicate better value.
2 to 5 years
Frequency of SCD Supply Stockouts at Study Sites
Prazo: 5 years
5 years

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

St. Jude Children's Research Hospital

Investigadores

  • Investigador principal: Jane Hankins, MD, St. Jude Children's Research Hospital
  • Investigador principal: Ana O. Mocumbi, MD PhD FESC, Eduardo Mondlane University, National Institute of Health, Chronic Disease Determinants Program

Publicações e links úteis

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Links úteis

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Estimado)

1 de agosto de 2026

Conclusão Primária (Estimado)

1 de agosto de 2031

Conclusão do estudo (Estimado)

1 de agosto de 2031

Datas de inscrição no estudo

Enviado pela primeira vez

17 de julho de 2026

Enviado pela primeira vez que atendeu aos critérios de CQ

17 de julho de 2026

Primeira postagem (Real)

22 de julho de 2026

Atualizações de registro de estudo

Última Atualização Postada (Real)

22 de julho de 2026

Última atualização enviada que atendeu aos critérios de controle de qualidade

17 de julho de 2026

Última verificação

1 de julho de 2026

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • UNIQUE
  • U1111-1335-3817 (Outro identificador: World Health Organization (Universal Trial Number))

Plano para dados de participantes individuais (IPD)

Planeja compartilhar dados de participantes individuais (IPD)?

NÃO

Informações sobre medicamentos e dispositivos, documentos de estudo

Estuda um medicamento regulamentado pela FDA dos EUA

Não

Estuda um produto de dispositivo regulamentado pela FDA dos EUA

Não

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