Sickle Cell Anemia in ChildrenPhase 3BRAINSAFEIINCT04750707
Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda ( BRAINSAFE-II )
A Phase 3 study testing Hydroxyurea in people with Sickle Cell Anemia in Children, run by Global Health Uganda LTD at 1 site in Uganda.
Trial summary
Worldwide, an estimated 200,000 babies are born with Sickle Cell Anemia (SCA) annually. Affected children suffer chronic ill health with some having frequent hospitalization. The patients are also at a high risk of brain injury arising from small and large cerebral blood vessel damage in SCA, also called sickle cell… Read more about this study
- Who is it for?
- Ages 3 to 9 years
- Conditions the study is for
- Sickle Cell Anemia in Children
- What is being tested?
- Hydroxyurea
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 270 participants
- Where
- 1 siteUganda
- Study dates
- 9 Mar 2021to 10 Mar 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 3 to 9 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Documented laboratory diagnosis of HbSS or HbS-B 0 thalassemia (both types are treated equally in SCA studies)
- Ages 3 through 9 years (inclusive) at enrolment
- To ensure clinic follow-up, child has attended Mulago Hospital Sickle Cell Anaemia (SCA)clinic 2 times in the prior 4 years, or at least once in the past 2 years if younger than < 4 years
- No history of hydroxyurea use for longer than 6 months
- Parent/legal guardian has provided a written consent (and if child is ≥8 years of age, has provided assent)
Who cannot join
- History of neurological abnormality known before age 4 months (to avoid those with non SCA brain injury)
- Child is currently enrolled in another clinical intervention trial
- Prior stroke as detected by standard Pediatric NIH Stroke Scale (PedNIHSS) examination
- Temporary exclusion criteria
- Pre-existing hematological toxicity
- Hemoglobin <4.0 gm/dL
Show all 12Show fewer
- Hemoglobin <6.0 gm/dL with Absolute Reticulocyte Count <100 x 10 9/L
- Absolute Reticulocyte Count <80 x 109/L with Hemoglobin <7.0 gm/dL
- Platelets <80 x 109/L
- Absolute Neutrophil Count <1.0 x 109/L
- Found to be with acute illness at enrolment with fever in last 1 week, respiratory infection, etc.
- History of a sickle crisis within the prior 2 weeks, or blood transfusion within the past 90 days Participants with temporary exclusion can be enrolled later when stable or the excluding criteria has resolved
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda: A Single Arm Open Label Trial, "BRAIN SAFE II"
Worldwide, an estimated 200,000 babies are born with Sickle Cell Anemia (SCA) annually. Affected children suffer chronic ill health with some having frequent hospitalization. The patients are also at a high risk of brain injury arising from small and large cerebral blood vessel damage in SCA, also called sickle cell vasculopathy (SCV). SCV is associated with the high risk of stroke. Such injury may manifest with neurological and cognitive impairment. An abnormal blood flow to the brain, as measured by a Doppler Ultrasound scan is a known risk factor for stroke.
The hypothesis is that hydroxyurea therapy will prevent, stabilize or improve SCV and its effects.
Read more
The study is an open label, single arm clinical trial to test the impact of hydroxyurea treatment in 270 children with SCA starting at ages 3-9 years.
Following baseline assessments, all participants will begin hydroxyurea therapy starting at about 20mg/kg/day. Changes in the frequency and severity of each test (neurological and cognitive tests and cerebral blood flow velocity) will be compared with their baseline tests (prior to hydroxyurea) by repeating these tests at 18 and 36 months. In a randomly selected subset of 90 participants, an evaluation of the impact of hydroxyurea on structural brain vascular injury using magnetic resonance brain imaging (MRI) and magnetic vessel imaging ,also called angiography (MRA) at baseline and at 36 months. Lastly, an assessment of changes to biomarkers of anemia, inflammation and malnutrition from before and during hydroxyurea therapy and determine their relationship to the outcomes. The proposed intervention with hydroxyurea is the first Africa-based trial to broadly prevent or ameliorate manifestations of SCV.
Read the detailed description
Annually, an estimated 200,000 babies are born with Sickle Cell Anemia (SCA) worldwide. Affected children often suffer repeated acute illnesses and chronic ill health from vascular occlusive phenomenon. In particular, they are at a high risk of stroke due to the development of sickle cell cerebral vascular (SCV) injury. SCV can also cause neurocognitive impairment. Stroke risk may be predicted by abnormal cerebral arterial flow detected by transcutaneous Doppler ultrasound (TCD).
The specific aims are:
Aim 1: Determine the impact of hydroxyurea therapy on the frequency and severity of SCV in a cohort of children treated for three years, as measured by 3 distinct outcomes: abnormal TCD, neurocognitive impairment and primary stroke, compared with baseline.
Aim 2: Evaluate the impact of hydroxyurea on structural SCV using MRI and MRA in a randomly selected subset of this cohort.
Aim 3: Assess changes to anemia, malnutrition status, C-reactive protein and other inflammatory markers during hydroxyurea therapy, over time, compared with baseline levels.
Design: This will an open label, single arm trial of hydroxyurea to test the three year impact of hydroxyurea treatment in children with SCA on each of three clinically important outcomes: abnormal cerebral arterial flow, neurocognitive impairment and primary stroke.
In aim 1, there will be enrollment of a new longitudinal cohort of 270 children ages 3-9 years selected randomly from among eligible patients attending Mulago hospital SCA clinic (MHSCC) roster, without evidence of prior stroke by examination. The hypothesis is that hydroxyurea therapy will prevent, stabilize or improve Sickle Cell Vasculopathy(SCV) and its effects. Eligible subjects will be evaluated by TCD, stroke-focused neurological examination and neurocognitive testing at baseline. Following initial assessments, study subjects will begin standardized hydroxyurea therapy. Overtime, changes in the frequency and severity of each distinct measure of SCV with baseline by repeating these three tests at 18 and 36 months and against the age specific prevalence of SCV in children who did not receive any intervention in the earlier BRAIN SAFE I observational study (our historical cohort).
In Aim 2, a subset of the study subjects, a randomly selected subset of 90 subjects, will undergo brain imaging at baseline and at 36 months to assess structural findings over time. The hypothesis is that prospective brain imaging by MRI and MRA provides critical structural definitions [1,2] of SCV changes or stabilization over three years compared to baseline, while on hydroxyurea. Results of brain imaging and biomarkers will be correlated with these outcomes over time.
In aim 3, there will be prospective examination for risk factors of SCV overtime. Biomarkers for risk of SCV may include severe anemia, the inflammatory marker C- reactive protein (CRP) and malnutrition status. The hypothesis is that a higher burden of anemia, inflammation and malnutrition predispose African children with SCA to greater burden of SCV than in the West. Changes in the values of these biomarkers will be assessed over time while on hydroxyurea
Site: Participants will be recruited from Mulago hospital Sickle Cell Anemia Clinic in Kampala, Uganda.
Follow up: Participants will be on study medication and follow-up for 36 months.
Data Analysis: The investigators will estimate the effects of hydroxyurea for each distinct endpoint, (stroke, abnormal TCD or impaired cognition) by comparing against the age specific prevalence in children who did not receive any intervention in the earlier BRAIN SAFE I study. To assess the risk of stabilization or reduction in SCV, time to event analyses using Cox proportional hazards modelling will be used after Kaplan Meier curves are generated. Multilevel regression models will be used to asses within and between subject changes over time for continuous measures of TCD velocity and cognitive z-scores.
- Conditions
- Sickle Cell Anemia in Children
- Being tested
- Hydroxyurea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
Also called Siklos
The study intervention will be daily oral Hydroxyurea given in single doses starting at approximately 20mg/kg/day and escalated to a maximum 30mg/kg/day depending on clinical need and according to standard guidelines.
| Study group | Role | What they receive |
|---|---|---|
| single arm | Other | Single arm, open label of hydroxyurea starting at 20mg/kg and increased to 30mg/kg depending on clinical need and according to standard guidelines Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
The frequency, age specific prevalence and severity of Sickle Cell Vasculopathy manifested by New stroke as assessed by the Pediatric NIH Stroke Scale (PedNIHSS) over the 3-year study while on hydroxyurea treatment, compared to baseline assessments.
Baseline assessment will be completed for all study participants and over the 3-year study, scheduled outcome assessments will be performed at study months 18 and 36.
These assessments will aim to document New (incident) stroke as assessed by the PedNIHSS
Measured: 3 years
The frequency, age specific prevalence and severity of Sickle Cell Vasculopathy manifested by a change in transcranial doppler (TCD) velocity by 15cm/sec or more over the 3-year study while on hydroxyurea treatment, compared to baseline assessment.
Baseline assessment will be completed for all study participants and over the 3-year study, scheduled outcome assessments will be performed at study months 18 and 36.These assessments will aim to document a change in TCD velocity by 15cm/sec or more.
Measured: 3 years
The frequency, age specific prevalence and severity of SCV manifested by New or worsening cognitive impairment, both quantitative and categorical over the 3-year study while on hydroxyurea treatment, compared to baseline assessments.
Baseline assessment will be completed for all study participants and over the 3-year study, scheduled outcome assessments will be performed at study months 18 and 36.These assessments will aim to document new or worsening cognitive impairment - both quantitative and categorical (by standardized criteria ≤-2 z-score) compared to established criteria
Measured: 3 years
Secondary outcomes (2)
Stabilized or worsened structural changes on brain Magnetic Resonance Imaging(MRI) over the 3 years compared to baseline
Brain magnetic resonance will be performed on a subset of subjects at baseline and study month 36 only. Stabilized or worsened structural changes on brain Magnetic Resonance Imaging(MRI) over the 3 years compared to baseline will be assessed.
Measured: 3 years
Stabilized or worsened structural changes on brain Magnetic Resonance Angiography(MRA) over the 3 years compared to baseline
Brain magnetic resonance angiography will be performed on a subset of subjects, at baseline and study month 36 only. Stabilized or worsened structural changes on brain Magnetic Resonance Angiography over the 3 years compared to baseline will be assessed.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2021
- Study start9 Mar 2021
- Primary completion10 Mar 2025
- Study completion10 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell Anemia in ChildrenStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 270 participants (actual)
Sponsor and investigators
- Lead sponsor
- Global Health Uganda LTD
- Sponsor type
- University, hospital or other
- Collaborators
- Columbia University, University of Pittsburgh, Emory University, Mulago Hospital, Uganda, Makerere University, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Sponsor
- Investigators
- Richard Idro, PhD, Principal investigator, Makerere UniversityNancy Green, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04750707
- Sponsor's protocol ID
- REC REF 2019-147
- Other IDs
- R01HD096559
- Acronym
- BRAINSAFEII
- Status last verified
- Apr 2025
- First submitted
- 25 Jan 2021
- Last updated
- 17 Apr 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Provider Investigator- Dr Richard Idro Recipient Investigator- Dr Nancy Green Permitted Uses of Data Set Scientific publication Inform policies Systematic reviews Safety investigations Other legitimate academic and policy issues. Data Set Content 1. Demographic data 2. Interventional drug data 3. Cognitive data including all neurocognitive tests administered at 0, 18 and 36 months follow-ups. 4. Laboratory data including hemograms, haemoglobin electrophoresis, serum chemistry results, inflammatory markers, SCA variants at all scheduled time points. 5. Neuroimaging data including all MRI data 6. TCD data 7. Scheduled visit clinical data including history, physical examination, PedNIHSS at baseline,18 and 36 months. 8. Unscheduled visits data including all adverse events
Publications
Galadanci NA, Umar Abdullahi S, Vance LD, Musa Tabari A, Ali S, Belonwu R, Salihu A, Amal Galadanci A, Wudil Jibir B, Bello-Manga H, Neville K, Kirkham FJ, Shyr Y, Phillips S, Covert BV, Kassim AA, Jordan LC, Aliyu MH, DeBaun MR. Feasibility trial for primary stroke prevention in children with sickle cell anemia in Nigeria (SPIN trial). Am J Hematol. 2017 Aug;92(8):780-788. doi: 10.1002/ajh.24770. Epub 2017 Jun 15.
PubMed 28439953Naggayi SK, Kalibbala D, Mboizi V, Ssenkusu J, Jin Z, Rosano C, Munube D, Wambaka B, Namazzi R, Kasirye P, Kabatabaazi M, Nambatya G, Murungi S, Nabaggala C, Nakafeero M, Troidl IR, Opoka R, Idro R, Bangirana P, Green NS. Neurocognitive gains among Ugandan children with sickle cell anemia on hydroxyurea: 18-month interim trial results. Blood Adv. 2025 Jun 24;9(12):3116-3127. doi: 10.1182/bloodadvances.2024015073.
PubMed 40085947Mboizi V, Nabaggala C, Munube D, Ssenkusu JM, Kasirye P, Kamya S, Kawooya MG, Boehme A, Minja F, Mupere E, Opoka R, Rosano C, Idro R, Green NS. Hydroxyurea therapy for neurological and cognitive protection in pediatric sickle cell anemia in Uganda (BRAIN SAFE II): Protocol for a single-arm open label trial. Contemp Clin Trials Commun. 2024 Nov 28;42:101404. doi: 10.1016/j.conctc.2024.101404. eCollection 2024 Dec.
PubMed 39717517
Study at a glance
From the registry record, last updated by the sponsor on 17 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda: A Single Arm Open Label Trial, "BRAIN SAFE II"
- ClinicalTrials.gov ID
- NCT04750707
- Acronym / sponsor ID
- BRAINSAFEII / REC REF 2019-147
- Conditions
- Sickle Cell Anemia in Children
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 270 participants (actual)
- Lead sponsor
- Global Health Uganda LTDUniversity, hospital or other
- Collaborators
- Columbia University, University of Pittsburgh, Emory University, Mulago Hospital, Uganda, Makerere University, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- 9 Mar 2021 (actual)
- Primary completion
- 10 Mar 2025 (actual)
- Study completion
- 10 Mar 2025 (actual)
- First posted
- 11 Feb 2021 (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.
Worldwide, an estimated 200,000 babies are born with Sickle Cell Anemia (SCA) annually. Affected children suffer chronic ill health with some having frequent hospitalization. The patients are also at a high risk of brain injury arising from small and large cerebral blood vessel damage in SCA, also called sickle cell vasculopathy (SCV). SCV is associated with the high risk of stroke. Such injury may manifest with neurological and cognitive impairment. An abnormal blood flow to the brain, as measured by a Doppler Ultrasound scan is a known risk factor for stroke.
The hypothesis is that hydroxyurea therapy will prevent, stabilize or improve SCV and its effects.
The study is an open label, single arm clinical trial to test the impact of hydroxyurea treatment in 270 children with SCA starting at ages 3-9 years.
Following baseline assessments, all participants will begin hydroxyurea therapy starting at about 20mg/kg/day. Changes in the frequency and severity of each test (neurological and cognitive tests and cerebral blood flow velocity) will be compared with their baseline tests (prior to hydroxyurea) by repeating these tests at 18 and 36 months. In a randomly selected subset of 90 participants, an evaluation of the impact of hydroxyurea on structural brain vascular injury using magnetic resonance brain imaging (MRI) and magnetic vessel imaging ,also called angiography (MRA) at baseline and at 36 months. Lastly, an assessment of changes to biomarkers of anemia, inflammation and malnutrition from before and during hydroxyurea therapy and determine their relationship to the outcomes. The proposed intervention with hydroxyurea is the first Africa-based trial to broadly prevent or ameliorate manifestations of SCV.
Detailed description
Annually, an estimated 200,000 babies are born with Sickle Cell Anemia (SCA) worldwide. Affected children often suffer repeated acute illnesses and chronic ill health from vascular occlusive phenomenon. In particular, they are at a high risk of stroke due to the development of sickle cell cerebral vascular (SCV) injury. SCV can also cause neurocognitive impairment. Stroke risk may be predicted by abnormal cerebral arterial flow detected by transcutaneous Doppler ultrasound (TCD).
The specific aims are:
Aim 1: Determine the impact of hydroxyurea therapy on the frequency and severity of SCV in a cohort of children treated for three years, as measured by 3 distinct outcomes: abnormal TCD, neurocognitive impairment and primary stroke, compared with baseline.
Aim 2: Evaluate the impact of hydroxyurea on structural SCV using MRI and MRA in a randomly selected subset of this cohort.
Aim 3: Assess changes to anemia, malnutrition status, C-reactive protein and other inflammatory markers during hydroxyurea therapy, over time, compared with baseline levels.
Design: This will an open label, single arm trial of hydroxyurea to test the three year impact of hydroxyurea treatment in children with SCA on each of three clinically important outcomes: abnormal cerebral arterial flow, neurocognitive impairment and primary stroke.
In aim 1, there will be enrollment of a new longitudinal cohort of 270 children ages 3-9 years selected randomly from among eligible patients attending Mulago hospital SCA clinic (MHSCC) roster, without evidence of prior stroke by examination. The hypothesis is that hydroxyurea therapy will prevent, stabilize or improve Sickle Cell Vasculopathy(SCV) and its effects. Eligible subjects will be evaluated by TCD, stroke-focused neurological examination and neurocognitive testing at baseline. Following initial assessments, study subjects will begin standardized hydroxyurea therapy. Overtime, changes in the frequency and severity of each distinct measure of SCV with baseline by repeating these three tests at 18 and 36 months and against the age specific prevalence of SCV in children who did not receive any intervention in the earlier BRAIN SAFE I observational study (our historical cohort).
In Aim 2, a subset of the study subjects, a randomly selected subset of 90 subjects, will undergo brain imaging at baseline and at 36 months to assess structural findings over time. The hypothesis is that prospective brain imaging by MRI and MRA provides critical structural definitions [1,2] of SCV changes or stabilization over three years compared to baseline, while on hydroxyurea. Results of brain imaging and biomarkers will be correlated with these outcomes over time.
In aim 3, there will be prospective examination for risk factors of SCV overtime. Biomarkers for risk of SCV may include severe anemia, the inflammatory marker C- reactive protein (CRP) and malnutrition status. The hypothesis is that a higher burden of anemia, inflammation and malnutrition predispose African children with SCA to greater burden of SCV than in the West. Changes in the values of these biomarkers will be assessed over time while on hydroxyurea
Site: Participants will be recruited from Mulago hospital Sickle Cell Anemia Clinic in Kampala, Uganda.
Follow up: Participants will be on study medication and follow-up for 36 months.
Data Analysis: The investigators will estimate the effects of hydroxyurea for each distinct endpoint, (stroke, abnormal TCD or impaired cognition) by comparing against the age specific prevalence in children who did not receive any intervention in the earlier BRAIN SAFE I study. To assess the risk of stabilization or reduction in SCV, time to event analyses using Cox proportional hazards modelling will be used after Kaplan Meier curves are generated. Multilevel regression models will be used to asses within and between subject changes over time for continuous measures of TCD velocity and cognitive z-scores.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| single arm | Other | Drug: HydroxyureaSingle arm, open label of hydroxyurea starting at 20mg/kg and increased to 30mg/kg depending on clinical need and according to standard guidelines |
Who may qualify
- Ages
- 3 to 9 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Documented laboratory diagnosis of HbSS or HbS-B 0 thalassemia (both types are treated equally in SCA studies)
- Ages 3 through 9 years (inclusive) at enrolment
- To ensure clinic follow-up, child has attended Mulago Hospital Sickle Cell Anaemia (SCA)clinic 2 times in the prior 4 years, or at least once in the past 2 years if younger than < 4 years
- No history of hydroxyurea use for longer than 6 months
- Parent/legal guardian has provided a written consent (and if child is ≥8 years of age, has provided assent)
Key exclusion criteria (12 listed)
- History of neurological abnormality known before age 4 months (to avoid those with non SCA brain injury)
- Child is currently enrolled in another clinical intervention trial
- Prior stroke as detected by standard Pediatric NIH Stroke Scale (PedNIHSS) examination
- Temporary exclusion criteria
- Pre-existing hematological toxicity
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 |
|---|---|---|
| HydroxyureaAlso: SiklosThe study intervention will be daily oral Hydroxyurea given in single doses starting at approximately 20mg/kg/day and escalated to a maximum 30mg/kg/day depending on clinical need and according to standard guidelines. | Drug | single arm |
Primary outcome
The frequency, age specific prevalence and severity of Sickle Cell Vasculopathy manifested by New stroke as assessed by the Pediatric NIH Stroke Scale (PedNIHSS) over the 3-year study while on hydroxyurea treatment, compared to baseline assessments.
Time frame: 3 years
Baseline assessment will be completed for all study participants and over the 3-year study, scheduled outcome assessments will be performed at study months 18 and 36. These assessments will aim to document New (incident) stroke as assessed by the PedNIHSS
The frequency, age specific prevalence and severity of Sickle Cell Vasculopathy manifested by a change in transcranial doppler (TCD) velocity by 15cm/sec or more over the 3-year study while on hydroxyurea treatment, compared to baseline assessment.
Time frame: 3 years
Baseline assessment will be completed for all study participants and over the 3-year study, scheduled outcome assessments will be performed at study months 18 and 36.These assessments will aim to document a change in TCD velocity by 15cm/sec or more.
The frequency, age specific prevalence and severity of SCV manifested by New or worsening cognitive impairment, both quantitative and categorical over the 3-year study while on hydroxyurea treatment, compared to baseline assessments.
Time frame: 3 years
Baseline assessment will be completed for all study participants and over the 3-year study, scheduled outcome assessments will be performed at study months 18 and 36.These assessments will aim to document new or worsening cognitive impairment - both quantitative and categorical (by standardized criteria ≤-2 z-score) compared to established criteria
2 secondary outcomes
Stabilized or worsened structural changes on brain Magnetic Resonance Imaging(MRI) over the 3 years compared to baseline
Time frame: 3 years
Stabilized or worsened structural changes on brain Magnetic Resonance Angiography(MRA) over the 3 years compared to baseline
Time frame: 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Apr 2025.
- Background: PubMed 28439953Galadanci NA, Umar Abdullahi S, Vance LD, Musa Tabari A, Ali S, Belonwu R, Salihu A, Amal Galadanci A, Wudil Jibir B, Bello-Manga H, Neville K, Kirkham FJ, Shyr Y, Phillips S, Covert BV, Kassim AA, Jordan LC, Aliyu MH, DeBaun MR. Feasibility trial for primary stroke prevention in children with sickle cell anemia in Nigeria (SPIN trial). Am J Hematol. 2017 Aug;92(8):780-788. doi: 10.1002/ajh.24770. Epub 2017 Jun 15.
- Derived from this study: PubMed 40085947Naggayi SK, Kalibbala D, Mboizi V, Ssenkusu J, Jin Z, Rosano C, Munube D, Wambaka B, Namazzi R, Kasirye P, Kabatabaazi M, Nambatya G, Murungi S, Nabaggala C, Nakafeero M, Troidl IR, Opoka R, Idro R, Bangirana P, Green NS. Neurocognitive gains among Ugandan children with sickle cell anemia on hydroxyurea: 18-month interim trial results. Blood Adv. 2025 Jun 24;9(12):3116-3127. doi: 10.1182/bloodadvances.2024015073.
- Derived from this study: PubMed 39717517Mboizi V, Nabaggala C, Munube D, Ssenkusu JM, Kasirye P, Kamya S, Kawooya MG, Boehme A, Minja F, Mupere E, Opoka R, Rosano C, Idro R, Green NS. Hydroxyurea therapy for neurological and cognitive protection in pediatric sickle cell anemia in Uganda (BRAIN SAFE II): Protocol for a single-arm open label trial. Contemp Clin Trials Commun. 2024 Nov 28;42:101404. doi: 10.1016/j.conctc.2024.101404. eCollection 2024 Dec.
- Individual participant dataSharing planned: Yes. Provider Investigator- Dr Richard Idro Recipient Investigator- Dr Nancy Green Permitted Uses of Data Set Scientific publication Inform policies Systematic reviews Safety investigations Other legitimate academic and policy issues. Data Set Content 1. Demographic data 2. Interventional drug data 3. Cognitive data including all neurocognitive tests administered at 0, 18 and 36 months follow-ups. 4. Laboratory data including hemograms, haemoglobin electrophoresis, serum chemistry results, inflammatory markers, SCA variants at all scheduled time points. 5. Neuroimaging data including all MRI data 6. TCD data 7. Scheduled visit clinical data including history, physical examination, PedNIHSS at baseline,18 and 36 months. 8. Unscheduled visits data including all adverse events
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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. This study is not recruiting at the moment.
Richard Idro, PhD principal investigator
Makerere University
Nancy Green, MD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Hydroxyurea in people with Sickle Cell Anemia in Children, run by Global Health Uganda LTD at 1 site in Uganda.
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 5 inclusion and 12 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: finished, as of the sponsor's last update on 17 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Uganda. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The frequency, age specific prevalence and severity of Sickle Cell Vasculopathy manifested by New stroke as assessed by the Pediatric NIH Stroke Scale (PedNIHSS) over the 3-year study while on hydroxyurea treatment, compared to baseline assessments. (time frame: 3 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04750707, which also lists 3 linked publications.
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 17 Apr 2025.