Sickle Cell DiseaseNCT07719972
Universal Newborn Screening For Sickle Cell Disease In Mozambique
A study testing Point-of-care testing (POCT) -Patient participants, Context Assessment for Community Health (COACH) survey and Semi-structured interview in people with Sickle Cell Disease, run by St. Jude Children's Research Hospital.
Trial summary
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. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Point-of-care testing (POCT) -Patient participants, Context Assessment for Community Health (COACH) survey and Semi-structured interview
- Type of treatment
- Vaccines
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6,750 participants
- Where
- No sites listed yet
- Study dates
- Aug 2026to Aug 2031
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 10Show fewer
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
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.
Read the detailed description
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.
- Conditions
- Sickle Cell Disease
- Being tested
- Point-of-care testing (POCT) -Patient participants, Point-of-care testing (POCT) - Healthy control participants, Context Assessment for Community Health (COACH) survey, Semi-structured interview
- Keywords
- Mozambique, Patient participants, Healthy control participants, Health facility staff participants, Supply chain expert participants, National public health system expert participants
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Point-of-care testing (POCT) -Patient participantsOther
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT).
Point-of-care testing (POCT) - Healthy control participantsOther
Infants will be screened for Sickle Cell Disease using with point-of-care testing (POCT) and prospectively monitored for survival.
Context Assessment for Community Health (COACH) surveyOther
The COACH survey will collect quantitative data on eight contextual factors that impact a site's ability to implement evidence-based interventions.
Semi-structured interviewOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Patient participants | Other | 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. Receives: Other: Point-of-care testing (POCT) -Patient participants |
| Healthy control participants | Other | 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). Receives: Other: Point-of-care testing (POCT) - Healthy control participants |
| Health facility staff participants | Other | Healthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study. Receives: Other: Context Assessment for Community Health (COACH) survey; Other: Semi-structured interview |
| Supply chain expert participants | Other | 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. Receives: Other: Semi-structured interview |
| National public health system expert participants | Other | 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. Receives: Other: Semi-structured interview |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of eligible population screened for Sickle Cell Disease
Measured: 3 years
Percentage of new SCD cases linked to care
Measured: 3 years
Secondary outcomes (9)
Comparison of under-2 mortality between participants with SCD whom received longitudinal care and participants who screened negative for SCD
The primary outcome measure will be 'Alive and in care: Yes or No'.
Measured: 2 years post-screening and therapy
Percentage of SCD cases retained in care by 2 years of age
Measured: 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
Measured: 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
Measured: 3 years
Comparison of SCD Screening Performance Results from Initial point-of-care (POCT), Gazelle, and Hemoglobin Fractionation
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.
Measured: 3 years
Cost-Effectiveness of Integrating SCD Consumables into National Supply Chains
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.
Measured: 2 to 5 years
Cost-Effectiveness of POCT for Early SCD Diagnosis
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.
Measured: 2 to 5 years
Cost-Effectiveness of Decentralized PEN-Plus SCD Management
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.
Measured: 2 to 5 years
Frequency of SCD Supply Stockouts at Study Sites
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2026
- Study startAug 2026 (estimated)
- Primary completionAug 2031 (estimated)
- Study completionAug 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6,750 participants (estimated)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jane Hankins, MD, Principal investigator, St. Jude Children's Research HospitalAna O. Mocumbi, MD PhD FESC, Principal investigator, Eduardo Mondlane University, National Institute of Health, Chronic Disease Determinants Program
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07719972
- Sponsor's protocol ID
- UNIQUE
- Other IDs
- U1111-1335-3817
- Status last verified
- Jul 2026
- First submitted
- 17 Jul 2026
- Last updated
- 22 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Universal Newborn Screening For Sickle Cell Disease In Mozambique
- ClinicalTrials.gov ID
- NCT07719972
- Acronym / sponsor ID
- UNIQUE
- Conditions
- Sickle Cell Disease
- Keywords
- Mozambique, Patient participants, Healthy control participants, Health facility staff participants, Supply chain expert participants, National public health system expert participants
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6,750 participants (estimated)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- Aug 2026 (estimated)
- Primary completion
- Aug 2031 (estimated)
- Study completion
- Aug 2031 (estimated)
- First posted
- 22 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Patient participants | Other | Other: Point-of-care testing (POCT) -Patient participantsAll 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 | Other | Other: Point-of-care testing (POCT) - Healthy control participantsInfants 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 | Other | Other: Context Assessment for Community Health (COACH) survey, Other: Semi-structured interviewHealthcare staff ages ≥18 years working at secondary-level facilities involved in the UNIQUE study. |
| Supply chain expert participants | Other | Other: Semi-structured interviewAdministrative 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 | Other | Other: Semi-structured interviewAdministrative 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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:
- 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.
Key exclusion criteria (10 listed)
- Children participants:
- Patient participants: none
- Healthy control participants:
- Health facility staff participants: none.
- Supply chain expert participants: none.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Point-of-care testing (POCT) -Patient participantsInfants will be screened for Sickle Cell Disease using with point-of-care testing (POCT). | Other | Patient participants |
| Point-of-care testing (POCT) - Healthy control participantsInfants will be screened for Sickle Cell Disease using with point-of-care testing (POCT) and prospectively monitored for survival. | Other | Healthy control participants |
| Context Assessment for Community Health (COACH) surveyThe COACH survey will collect quantitative data on eight contextual factors that impact a site's ability to implement evidence-based interventions. | Other | Health facility staff participants |
| Semi-structured interviewInterview 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. | Other | Health facility staff participants, National public health system expert participants, Supply chain expert participants |
Primary outcome
Percentage of eligible population screened for Sickle Cell Disease
Time frame: 3 years
Percentage of new SCD cases linked to care
Time frame: 3 years
9 secondary outcomes
Comparison of under-2 mortality between participants with SCD whom received longitudinal care and participants who screened negative for SCD
Time frame: 2 years post-screening and therapy
Percentage of SCD cases retained in care by 2 years of age
Time frame: 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
Time frame: 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
Time frame: 3 years
Comparison of SCD Screening Performance Results from Initial point-of-care (POCT), Gazelle, and Hemoglobin Fractionation
Time frame: 3 years
Cost-Effectiveness of Integrating SCD Consumables into National Supply Chains
Time frame: 2 to 5 years
Cost-Effectiveness of POCT for Early SCD Diagnosis
Time frame: 2 to 5 years
Cost-Effectiveness of Decentralized PEN-Plus SCD Management
Time frame: 2 to 5 years
Frequency of SCD Supply Stockouts at Study Sites
Time frame: 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2026.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Sickle Cell Disease
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jane Hankins, MD principal investigator
St. Jude Children's Research Hospital
Ana O. Mocumbi, MD PhD FESC principal investigator
Eduardo Mondlane University, National Institute of Health, Chronic Disease Determinants Program
Questions about this trial
What is this clinical trial studying?
A study testing Point-of-care testing (POCT) -Patient participants, Context Assessment for Community Health (COACH) survey and Semi-structured interview in people with Sickle Cell Disease, run by St. Jude Children's Research Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 22 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jane Hankins, MD (888-226-4343, referralinfo@stjude.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of eligible population screened for Sickle Cell Disease (time frame: 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2026.