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Universal Newborn Screening For Sickle Cell Disease In Mozambique

2026년 7월 17일 업데이트: 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.

연구 개요

상태

아직 모집하지 않음

정황

개입 / 치료

상세 설명

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.

연구 유형

중재적

등록 (추정된)

6750

단계

  • 해당 없음

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

  • 어린이
  • 성인
  • 고령자

건강한 자원 봉사자를 받아들입니다

예

설명

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.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 다른
  • 할당: 무작위화되지 않음
  • 중재 모델: 병렬 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
다른: 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.
다른: Point-of-care testing (POCT) -Patient participants
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT).
다른: 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).
다른: 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.
다른: Health facility staff participants
Healthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study.
다른: 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.
다른: 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.
다른: 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.
다른: 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.
다른: 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.
다른: 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.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
기간
Percentage of eligible population screened for Sickle Cell Disease
기간: 3 years
3 years
Percentage of new SCD cases linked to care
기간: 3 years
3 years

2차 결과 측정

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

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

St. Jude Children's Research Hospital

수사관

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

간행물 및 유용한 링크

연구에 대한 정보 입력을 담당하는 사람이 자발적으로 이러한 간행물을 제공합니다. 이것은 연구와 관련된 모든 것에 관한 것일 수 있습니다.

유용한 링크

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (추정된)

2026년 8월 1일

기본 완료 (추정된)

2031년 8월 1일

연구 완료 (추정된)

2031년 8월 1일

연구 등록 날짜

최초 제출

2026년 7월 17일

QC 기준을 충족하는 최초 제출

2026년 7월 17일

처음 게시됨 (실제)

2026년 7월 22일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 7월 22일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 7월 17일

마지막으로 확인됨

2026년 7월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • UNIQUE
  • U1111-1335-3817 (기타 식별자: World Health Organization (Universal Trial Number))

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

아니

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

겸상 적혈구 질환에 대한 임상 시험

Point-of-care testing (POCT) -Patient participants에 대한 임상 시험

유사한 임상시험 검색

스폰서 및 공동 작업자

건강 상태

약물 개입

CROs by country

CROs in Cambodia

정황

희귀 질병

약물 개입

식이 보충제

스폰서 / 협력자

위치

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