Sickle Cell DiseaseSPIYANCT04808778
Stroke Prevention in Young Adults With Sickle Cell Anemia
An observational study of Sickle Cell Disease and Sickle Cell Anemia, run by Vanderbilt University Medical Center at 2 sites in 2 countries.
Trial summary
Sickle cell disease (SCD) is the most common genetic disease, affecting about 25 million people worldwide. Approximately 150,000 Nigerian children are born each year with sickle cell disease (SCD), making it the country with the largest burden of SCD in the world. Read more about this study
- Who is it for?
- Ages 16 to 26 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Anemia, Stroke, Ischemic
- How many people
- 250 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 17 May 2021to 1 Jun 2029
- Phase
- Observational
- 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 16 to 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: A total of 250 participants with sickle cell anemia between 16 through 25 years of age living in northern Nigeria receiving care at the Aminu Kano Teaching Hospital in Kano Nigeria.
Who may be able to join
- Patients with hemoglobin S-S or Sβ0 thalassemia confirmed by hemoglobin electrophoresis or High-Performance Liquid Chromatography (HPLC);
- Participant is 16 through 25 years of age;
- Informed consent from participants above 18 years, and informed consent from a parent or legal guardian and assent of participants aged < 18 years (assessment can take place up until the 26th birthday);
- Participant resides within an hour driving distance from the medical center to facilitate weekly phone calls between the scheduled monthly clinic visits;
- Participant is willing to be enrolled and followed for the duration of the study.
Who cannot join
- Young adults with co-morbidities that may have an impact on neurological status, such as epilepsy;
- Young adults enrolled in clinical trials upon entry;
- Participants with an implanted defibrillator or certain other implanted electronic or metallic devices contraindicated for MRI;
- Young adults with known HIV diagnosis;
- Any other condition or chronic illness, which in the opinion of the site's Principal Investigator (PI) makes participation ill-advised or unsafe.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
United States1 site
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-9000
About this study
Sickle cell disease (SCD) is the most common genetic disease, affecting about 25 million people worldwide. Approximately 150,000 Nigerian children are born each year with sickle cell disease (SCD), making it the country with the largest burden of SCD in the world. Recent advancements in care for children with SCA have translated into improved survival of children in both high and low-resource settings. However, more complications of SCD are seen in those who survive to adulthood. Silent cerebral infarcts (SCI) and strokes are among the most devastating complications of SCD, affecting 40% and 10% of children, respectively.
The overall goal of this study is to extend the Investigator's successful capacity-building effort in the assessment of neurological morbidity in children with SCD living in northern Nigeria (Kano) to young adults with SCD living in the same region. About 50% of all adults with SCD live in Nigeria. Despite the high prevalence of SCD in Africa, the neurological morbidity is not well characterized, limiting opportunities for primary and secondary stroke prevention strategies. At least 50% of young adults with sickle cell anemia (SCA), the most severe form of the disease, will have SCIs and an estimated 10% will have strokes, based on studies in high-resource settings. In high-resource settings, screening for abnormal transcranial Doppler (TCD) velocities in children with SCA, coupled with regular blood transfusion has resulted in a 92% reduction of relative risk for strokes. Despite this effective strategy, regular blood transfusion therapy does not seem sustainable in sub-Saharan Africa due to shortages and the risk of transfusion transmissible infections. Additionally, there is a lack of evidence-based stroke prevention strategies in young adults with SCA, either in the high-income or in low-resource settings. Based on the foregoing, the Investigators propose to determine the prevalence of neurological injury (overt stroke, transient ischemic attacks, and silent cerebral infarcts) in young adults at the transition age from 16-25 years. The Investigators will also, for the first time, assess conventional risk factors of stroke in the general population to determine whether a different prevention strategy is required to reduce the incidence of neurological injury in this high-risk population.
Read the detailed description
The Investigator's global hypothesis, to be tested eventually in an NIH-funded phase III controlled trial, is that hydroxyurea at a fixed moderate dose of 20 mg/kg is safe and effective for primary and secondary stroke prevention in young adults with SCA. Prior to testing this global hypothesis, the Investigators must develop a multi-disciplinary team that provides medical care for young adults with SCD and establish the clinical epidemiology of neurological morbidity in this distinct age group. Building upon the existing research platforms of ongoing NINDS-funded primary stroke prevention trials in Nigeria, the Investigators are uniquely positioned to extend their stroke assessment and treatment to the next sequential age group, young adults with SCA.
The immediate goals of this project are 1) to estimate the prevalence of neurological morbidity in young adults with SCA (R21 application to NIH); 2) to establish a prospective cohort of young adults to determine the incidence of neurological morbidity, and 3) to determine the safety and feasibility of fixed moderate dose of hydroxyurea therapy for prevention of further neurological disease in young adults with SCA in Nigeria.
The leadership of the current pediatric primary and secondary stroke prevention trials (NCT01801423, NCT02560935, NCT02675790) in Nigeria will apply a similar effective strategy used in the Investigator's previous pediatric NINDS-R21 and current pediatric NINDS-R01 to estimate the prevalence and incidence of neurological morbidity in young adults with SCA. Young adults with SCA have different stroke risk factors than children less than 16 years of age with SCA, including risk factors for stroke seen in the general population resulting in the need for age and disease-specific evidence-based management for primary and secondary stroke prevention strategies.
the Investigators propose to enroll 250 participants with SCA between 16-25 years of age. The Investigators believe this sample size is sufficient to estimate the prevalence of stroke and SCI. This cohort will be followed for 12-18 months to determine the short-term incidence of strokes and SCI. The Investigators do not intend to calculate a precise incidence of these neurological injuries due to the short duration of the follow-up.
For young adults with SCIs, strokes, and elevated TCD measurements (based on the pediatric threshold of abnormal ≥180 cm/s in the middle cerebral artery or terminal portion of the internal carotid) the Investigators will initially offer regular blood transfusion therapy as standard care. If they refuse, the Investigators will offer a dose of 20 mg/kg/day of hydroxyurea for at least one year of therapy. The hydroxyurea will be supplied free of charge with monthly follow-up.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Stroke, Ischemic, Silent Stroke, Silent Cerebral Infarct, Stroke
- Being tested
- Hydroxyurea therapy per standard care
- Keywords
- sickle cell anemia, stroke prevention, low-resource setting, silent cerebral infarct, northern Nigeria, young adults
- Medical subject headings
- Anemia, Sickle Cell, Ischemic Stroke, Stroke
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hydroxyurea therapy per standard careDrug
Also called Hydrea
Moderate-dose of 20mg/kg/day
| Study group | Role | What they receive |
|---|---|---|
| Participants with sickle cell anemia identified with neurological morbidity | 1. Successful completion of screening procedures inclusive of 1.) Cerebral blood flow velocity greater than or equal to 180 cm/sec measured twice or 2.) At least one measurement greater than or equal to 220 cm/sec in the middle cerebral artery or 3.) Two TCD measurements above 180 cm/sec within a three-month interval;
2. MRI showing cerebral infarcts with or without (based on Silent Cerebral Infarct Trial (SIT) criteria) neurological deficits on standard neurological examination;
3. Informed consent from a participant (> 18 years) or parent/legal guardian for participants (< 18 years) and assent of the participant completed;
4. Acceptance of hydroxyurea therapy for one year as standard care. After one year of therapy, the participant will have the option to continue therapy with follow-up visits to monitor adherence to therapy with his or her care provider. Receives: Drug: Hydroxyurea therapy per standard care | |
| Participants with sickle cell anemia identified to be without neurological morbidity | 1. Successful completion of screening procedures inclusive of cerebral blood flow velocity less than or equal to 170 cm/sec in the middle cerebral artery; 2. Normal MRI and MRA; 3. No focal neurological deficit on standard neurological examination; 4. Informed consent from a participant (> 18 years), or parent/legal guardian for participants (< 18 years) and assent from the participant; 5. Agreement to be followed for at least one year in the study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The prevalence of neurological morbidity in young adults with sickle cell anemia neurological examinations and MRA/MRI
To estimate the prevalence of neurological morbidity including SCIs, strokes, and cerebral vasculopathy.
Measured: 1 year
The prevalence of neurological morbidity in young adults with sickle cell anemia utilizing Transcranial Doppler (TCD) measurement
Percentage of patients with abnormal TCD velocity (> 200 cm/s)
Measured: 2 year
Conventional risk factors of stroke
We will also screen the participants for conventional risk factors of stroke (hypertension, smoking, diabetes, obesity, renal disease, cardiomyopathy, and atrial fibrillation).
Measured: 2 year
Secondary outcomes (2)
Long-term incidence of neurological morbidity in young adults with sickle cell anemia
For this purpose, we will determine the long-term incidence rates of initial and recurrent infarcts in young adults with sickle cell anemia with repeat magnetic resonance imaging and magnetic resonance angiography (MRI/MRA), and transcranial Doppler (TCD) measurements, and neurological examinations ~ 1 to 1.5 years for at least 10 years after baseline evaluations.
Measured: 10 years
Preliminary data for safety and feasibility of hydroxyurea therapy in young adults with sickle cell anemia
To collect the preliminary data necessary to establish a safety and feasibility study for fixed moderate dose hydroxyurea treatment (~ 20 mg/kg/day) of neurological morbidity in young adults with sickle cell anemia.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2021
- Study start17 May 2021
- Primary completion1 Jun 2029 (estimated)
- Study completion1 Jun 2029 (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 Vanderbilt University Medical CenterThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 250 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Aminu Kano Teaching Hospital
- Responsible party
- Principal investigator, Michael DeBaun, Vanderbilt University Medical Center
- Investigators
- Michael R DeBaun, MD, MPH, Principal investigator, Vanderbilt University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04808778
- Sponsor's protocol ID
- 190203
- Acronym
- SPIYA
- Status last verified
- Sep 2026
- First submitted
- 17 Mar 2021
- Last updated
- 9 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- 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 9 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Stroke Prevention in Young Adults With Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT04808778
- Acronym / sponsor ID
- SPIYA / 190203
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Stroke, Ischemic, Silent Stroke, Silent Cerebral Infarct, Stroke
- Keywords
- sickle cell anemia, stroke prevention, low-resource setting, silent cerebral infarct, northern Nigeria, young adults
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 250 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Collaborators
- Aminu Kano Teaching Hospital
- Study start
- 17 May 2021 (actual)
- Primary completion
- 1 Jun 2029 (estimated)
- Study completion
- 1 Jun 2029 (estimated)
- First posted
- 22 Mar 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.
Sickle cell disease (SCD) is the most common genetic disease, affecting about 25 million people worldwide. Approximately 150,000 Nigerian children are born each year with sickle cell disease (SCD), making it the country with the largest burden of SCD in the world. Recent advancements in care for children with SCA have translated into improved survival of children in both high and low-resource settings. However, more complications of SCD are seen in those who survive to adulthood. Silent cerebral infarcts (SCI) and strokes are among the most devastating complications of SCD, affecting 40% and 10% of children, respectively.
The overall goal of this study is to extend the Investigator's successful capacity-building effort in the assessment of neurological morbidity in children with SCD living in northern Nigeria (Kano) to young adults with SCD living in the same region. About 50% of all adults with SCD live in Nigeria. Despite the high prevalence of SCD in Africa, the neurological morbidity is not well characterized, limiting opportunities for primary and secondary stroke prevention strategies. At least 50% of young adults with sickle cell anemia (SCA), the most severe form of the disease, will have SCIs and an estimated 10% will have strokes, based on studies in high-resource settings. In high-resource settings, screening for abnormal transcranial Doppler (TCD) velocities in children with SCA, coupled with regular blood transfusion has resulted in a 92% reduction of relative risk for strokes. Despite this effective strategy, regular blood transfusion therapy does not seem sustainable in sub-Saharan Africa due to shortages and the risk of transfusion transmissible infections. Additionally, there is a lack of evidence-based stroke prevention strategies in young adults with SCA, either in the high-income or in low-resource settings. Based on the foregoing, the Investigators propose to determine the prevalence of neurological injury (overt stroke, transient ischemic attacks, and silent cerebral infarcts) in young adults at the transition age from 16-25 years. The Investigators will also, for the first time, assess conventional risk factors of stroke in the general population to determine whether a different prevention strategy is required to reduce the incidence of neurological injury in this high-risk population.
Detailed description
The Investigator's global hypothesis, to be tested eventually in an NIH-funded phase III controlled trial, is that hydroxyurea at a fixed moderate dose of 20 mg/kg is safe and effective for primary and secondary stroke prevention in young adults with SCA. Prior to testing this global hypothesis, the Investigators must develop a multi-disciplinary team that provides medical care for young adults with SCD and establish the clinical epidemiology of neurological morbidity in this distinct age group. Building upon the existing research platforms of ongoing NINDS-funded primary stroke prevention trials in Nigeria, the Investigators are uniquely positioned to extend their stroke assessment and treatment to the next sequential age group, young adults with SCA.
The immediate goals of this project are 1) to estimate the prevalence of neurological morbidity in young adults with SCA (R21 application to NIH); 2) to establish a prospective cohort of young adults to determine the incidence of neurological morbidity, and 3) to determine the safety and feasibility of fixed moderate dose of hydroxyurea therapy for prevention of further neurological disease in young adults with SCA in Nigeria.
The leadership of the current pediatric primary and secondary stroke prevention trials (NCT01801423, NCT02560935, NCT02675790) in Nigeria will apply a similar effective strategy used in the Investigator's previous pediatric NINDS-R21 and current pediatric NINDS-R01 to estimate the prevalence and incidence of neurological morbidity in young adults with SCA. Young adults with SCA have different stroke risk factors than children less than 16 years of age with SCA, including risk factors for stroke seen in the general population resulting in the need for age and disease-specific evidence-based management for primary and secondary stroke prevention strategies.
the Investigators propose to enroll 250 participants with SCA between 16-25 years of age. The Investigators believe this sample size is sufficient to estimate the prevalence of stroke and SCI. This cohort will be followed for 12-18 months to determine the short-term incidence of strokes and SCI. The Investigators do not intend to calculate a precise incidence of these neurological injuries due to the short duration of the follow-up.
For young adults with SCIs, strokes, and elevated TCD measurements (based on the pediatric threshold of abnormal ≥180 cm/s in the middle cerebral artery or terminal portion of the internal carotid) the Investigators will initially offer regular blood transfusion therapy as standard care. If they refuse, the Investigators will offer a dose of 20 mg/kg/day of hydroxyurea for at least one year of therapy. The hydroxyurea will be supplied free of charge with monthly follow-up.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Participants with sickle cell anemia identified with neurological morbidity | – | Drug: Hydroxyurea therapy per standard care1. Successful completion of screening procedures inclusive of 1.) Cerebral blood flow velocity greater than or equal to 180 cm/sec measured twice or 2.) At least one measurement greater than or equal to 220 cm/sec in the middle cerebral artery or 3.) Two TCD measurements above 180 cm/sec within a three-month interval; 2. MRI showing cerebral infarcts with or without (based on Silent Cerebral Infarct Trial (SIT) criteria) neurological deficits on standard neurological examination; 3. Informed consent from a participant (> 18 years) or parent/legal guardian for participants (< 18 years) and assent of the participant completed; 4. Acceptance of hydroxyurea therapy for one year as standard care. After one year of therapy, the participant will have the option to continue therapy with follow-up visits to monitor adherence to therapy with his or her care provider. |
| Participants with sickle cell anemia identified to be without neurological morbidity | – | –1. Successful completion of screening procedures inclusive of cerebral blood flow velocity less than or equal to 170 cm/sec in the middle cerebral artery; 2. Normal MRI and MRA; 3. No focal neurological deficit on standard neurological examination; 4. Informed consent from a participant (> 18 years), or parent/legal guardian for participants (< 18 years) and assent from the participant; 5. Agreement to be followed for at least one year in the study. |
Who may qualify
- Ages
- 16 to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- A total of 250 participants with sickle cell anemia between 16 through 25 years of age living in northern Nigeria receiving care at the Aminu Kano Teaching Hospital in Kano Nigeria.
Key inclusion criteria (5 listed)
- Patients with hemoglobin S-S or Sβ0 thalassemia confirmed by hemoglobin electrophoresis or High-Performance Liquid Chromatography (HPLC);
- Participant is 16 through 25 years of age;
- Informed consent from participants above 18 years, and informed consent from a parent or legal guardian and assent of participants aged < 18 years (assessment can take place up until the 26th birthday);
- Participant resides within an hour driving distance from the medical center to facilitate weekly phone calls between the scheduled monthly clinic visits;
- Participant is willing to be enrolled and followed for the duration of the study.
Key exclusion criteria (5 listed)
- Young adults with co-morbidities that may have an impact on neurological status, such as epilepsy;
- Young adults enrolled in clinical trials upon entry;
- Participants with an implanted defibrillator or certain other implanted electronic or metallic devices contraindicated for MRI;
- Young adults with known HIV diagnosis;
- Any other condition or chronic illness, which in the opinion of the site's Principal Investigator (PI) makes participation ill-advised or unsafe.
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 |
|---|---|---|
| Hydroxyurea therapy per standard careAlso: HydreaModerate-dose of 20mg/kg/day | Drug | Participants with sickle cell anemia identified with neurological morbidity |
Primary outcome
The prevalence of neurological morbidity in young adults with sickle cell anemia neurological examinations and MRA/MRI
Time frame: 1 year
To estimate the prevalence of neurological morbidity including SCIs, strokes, and cerebral vasculopathy.
The prevalence of neurological morbidity in young adults with sickle cell anemia utilizing Transcranial Doppler (TCD) measurement
Time frame: 2 year
Percentage of patients with abnormal TCD velocity (> 200 cm/s)
Conventional risk factors of stroke
Time frame: 2 year
We will also screen the participants for conventional risk factors of stroke (hypertension, smoking, diabetes, obesity, renal disease, cardiomyopathy, and atrial fibrillation).
2 secondary outcomes
Long-term incidence of neurological morbidity in young adults with sickle cell anemia
Time frame: 10 years
Preliminary data for safety and feasibility of hydroxyurea therapy in young adults with sickle cell anemia
Time frame: 2 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 9 Sep 2026.
- Individual participant dataSharing planned: No
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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.
Michael R DeBaun, MD, MPH principal investigator
Vanderbilt University Medical Center
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Sickle Cell Anemia, run by Vanderbilt University Medical Center at 2 sites in 2 countries.
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 5 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: under way, not looking for participants, as of the sponsor's last update on 9 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, Nigeria. 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.
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?
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 prevalence of neurological morbidity in young adults with sickle cell anemia neurological examinations and MRA/MRI (time frame: 1 year). 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?
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 9 Sep 2026.