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Universal Newborn Screening For Sickle Cell Disease In Mozambique

17. juli 2026 opdateret af: 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.

Studieoversigt

Status

Ikke rekrutterer endnu

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

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.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

6750

Fase

  • Ikke anvendelig

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ja

Beskrivelse

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.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Andet
  • Tildeling: Ikke-randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Andet: 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.
Andet: Point-of-care testing (POCT) -Patient participants
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT).
Andet: 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).
Andet: 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.
Andet: Health facility staff participants
Healthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study.
Andet: 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.
Andet: 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.
Andet: 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.
Andet: 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.
Andet: 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.
Andet: 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.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Tidsramme
Percentage of eligible population screened for Sickle Cell Disease
Tidsramme: 3 years
3 years
Percentage of new SCD cases linked to care
Tidsramme: 3 years
3 years

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Comparison of under-2 mortality between participants with SCD whom received longitudinal care and participants who screened negative for SCD
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 3 years
3 years
Comparison of SCD Screening Performance Results from Initial point-of-care (POCT), Gazelle, and Hemoglobin Fractionation
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 5 years
5 years

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

St. Jude Children's Research Hospital

Efterforskere

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

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Hjælpsomme links

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

1. august 2026

Primær færdiggørelse (Anslået)

1. august 2031

Studieafslutning (Anslået)

1. august 2031

Datoer for studieregistrering

Først indsendt

17. juli 2026

Først indsendt, der opfyldte QC-kriterier

17. juli 2026

Først opslået (Faktiske)

22. juli 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

22. juli 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

17. juli 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • UNIQUE
  • U1111-1335-3817 (Anden identifikator: World Health Organization (Universal Trial Number))

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

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Ingen

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Kliniske forsøg med Seglcellesygdom

Kliniske forsøg med Point-of-care testing (POCT) -Patient participants

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