Universal Newborn Screening For Sickle Cell Disease In Mozambique
2026年7月17日 更新者：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.
調査の概要
状態
まだ募集していません
条件
詳細な説明
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.
研究の種類
介入
入学 (推定)
6750
段階
- 適用できない
連絡先と場所
このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。
研究連絡先
- 名前：Jane Hankins, MD
- 電話番号：888-226-4343
- メール：referralinfo@stjude.org
参加基準
研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。
適格基準
就学可能な年齢
- 子
- 大人
- 高齢者
健康ボランティアの受け入れ
はい
説明
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.
研究計画
このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：他の
- 割り当て：非ランダム化
- 介入モデル：並列代入
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
他の：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.
|
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT).
|
|
他の：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).
|
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT) and prospectively monitored for survival.
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他の：Health facility staff participants
Healthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study.
|
The COACH survey will collect quantitative data on eight contextual factors that impact a site's ability to implement evidence-based interventions.
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.
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他の：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.
|
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.
|
|
他の：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.
|
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.
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
時間枠 |
|---|---|
|
Percentage of eligible population screened for Sickle Cell Disease
時間枠：3 years
|
3 years
|
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Percentage of new SCD cases linked to care
時間枠：3 years
|
3 years
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Comparison of under-2 mortality between participants with SCD whom received longitudinal care and participants who screened negative for SCD
時間枠：2 years post-screening and therapy
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The primary outcome measure will be 'Alive and in care: Yes or No'.
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2 years post-screening and therapy
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Percentage of SCD cases retained in care by 2 years of age
時間枠：2 years post-screening and therapy
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2 years post-screening and therapy
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Percentage of eligible secondary-level clinical wards implementing systematic screening and linkage to longitudinal care for new SCD cases
時間枠：3 years
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3 years
|
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Percentage of eligible health facility staff participants in secondary-level clinical wards implementing systematic screening and linkage to longitudinal care for new SCD cases
時間枠：3 years
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3 years
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Comparison of SCD Screening Performance Results from Initial point-of-care (POCT), Gazelle, and Hemoglobin Fractionation
時間枠：3 years
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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.
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3 years
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Cost-Effectiveness of Integrating SCD Consumables into National Supply Chains
時間枠：2 to 5 years
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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.
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2 to 5 years
|
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Cost-Effectiveness of POCT for Early SCD Diagnosis
時間枠：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
|
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Cost-Effectiveness of Decentralized PEN-Plus SCD Management
時間枠：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
時間枠：5 years
|
5 years
|
協力者と研究者
ここでは、この調査に関係する人々や組織を見つけることができます。
捜査官
- 主任研究者：Jane Hankins, MD、St. Jude Children's Research Hospital
- 主任研究者：Ana O. Mocumbi, MD PhD FESC、Eduardo Mondlane University, National Institute of Health, Chronic Disease Determinants Program
出版物と役立つリンク
研究に関する情報を入力する責任者は、自発的にこれらの出版物を提供します。これらは、研究に関連するあらゆるものに関するものである可能性があります。
研究記録日
これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。
主要日程の研究
研究開始 (推定)
2026年8月1日
一次修了 (推定)
2031年8月1日
研究の完了 (推定)
2031年8月1日
試験登録日
最初に提出
2026年7月17日
QC基準を満たした最初の提出物
2026年7月17日
最初の投稿 (実際)
2026年7月22日
学習記録の更新
投稿された最後の更新 (実際)
2026年7月22日
QC基準を満たした最後の更新が送信されました
2026年7月17日
最終確認日
2026年7月1日
詳しくは
本研究に関する用語
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- UNIQUE
- U1111-1335-3817 (その他の識別子：World Health Organization (Universal Trial Number))
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
いいえ
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
いいえ
米国FDA規制機器製品の研究
いいえ
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