Sickle Cell Anemia in Children Clinical Trial
Official title:
Stroke Prevention With Hydroxyurea Enabled Through Research and Education (SPHERE): A Prospective Trial to Reduce Primary Stroke in Children With Sickle Cell Anaemia
Verified date | July 2023 |
Source | Children's Hospital Medical Center, Cincinnati |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This study will 1) Evaluate the prevalence of elevated (conditional or abnormal) transcranial Doppler (TCD) velocities in a cross-sectional analysis of children with Sickle Cell Anemia (SCA) living in Tanzania; 2) Obtain longitudinal data on TCD velocities in this population; and 3) Measure the effects of hydroxyurea therapy on TCD velocities and associated primary stroke risk.
Status | Enrolling by invitation |
Enrollment | 202 |
Est. completion date | December 31, 2025 |
Est. primary completion date | March 31, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 2 Years to 16 Years |
Eligibility | Inclusion Criteria: - Willingness to sign informed consent - Willingness to follow all study procedures - Available for study visits for the duration of the study and no plans to move away from study center. - Confirmed diagnosis of Sickle Cell Anemia (SCA) by haemoglobin electrophoresis. - Able to take oral medication and follow hydroxyurea treatment schedule. Exclusion Criteria: There are no permanent exclusion criteria for participants to enroll in the screening TCD portion of SPHERE. Temporary, time-limited exclusion criteria for the screening TCD portion include the following: - Febrile illness within the past two weeks. (Temporary Exclusion) - Hospitalized within the past two weeks. (Temporary Exclusion) - Transfusion within the past two weeks. (Temporary Exclusion) Patients who enroll in the screening portion, have a conditional or abnormal TCD, and are eligible to start hydroxyurea will be excluded from receiving study treatment if they meet any of the following criteria: - Abnormal pre-enrolment laboratory values (Temporary Exclusion) - Known medical condition making participation ill-advised. - Known allergic reactions to components of hydroxyurea. - Previous history of stroke. - Currently pregnant or lactating. |
Country | Name | City | State |
---|---|---|---|
Tanzania | Bugando Medical Centre | Mwanza | |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
Lead Sponsor | Collaborator |
---|---|
Children's Hospital Medical Center, Cincinnati | Bugando Medical Centre, The American Society of Hematology |
United States, Tanzania,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Prevalence of Elevated TCD | Determine the prevalence of elevated (conditional or abnormal) transcranial Doppler (TCD) velocities in a cross-sectional analysis of children with Sickle Cell Anemia (SCA) living in Tanzania | Baseline | |
Primary | Change in Primary Stroke Risk | Transcranial Doppler ultrasound (TCD) will be used to measure the change in the TAMV of arterial blood flow in the 4 major intracranial arteries bilaterally from study enrollment to 12 months after study enrollment. | Up to 12 Months at Month 12 | |
Secondary | Laboratory and Clinical Correlates | Identify laboratory and clinical correlates of elevated TCD velocities such as age, haemoglobin concentration, foetal haemoglobin, oxygen saturation, splenomegaly, history of acute chest syndrome, and previous malaria infection | Up to 24 Months | |
Secondary | Change in Hemoglobin Concentration | For those receiving hydroxyurea, the change in hemoglobin between baseline hemoglobin and follow up hemoglobin when a participant has reached maximum tolerated dose of hydroxyurea. | 6 Months | |
Secondary | Effect of Splenomegaly and Malaria Infections | Incidence of splenomegaly and malaria infection with rapid or laboratory malaria testing will be performed for any child presenting with fever. Incidence will be reported in the number of cases per 100 patient years. Abdominal ultrasound with splenic volume will be performed annually for all study participants. Quantify the degree of hypersplenism or autoinfarction and any association with malaria complications of SCA will be analyzed. | Up to 24 Months | |
Secondary | Prevalence of Co-inherited G6PD and Alpha Thalassemia | DNA will be collected at baseline to determine the prevalence of co-inherited hematologic diseases such as G6PD and alpha thalassemia. | One time at Baseline | |
Secondary | Hydroxyurea Area Under the Curve (AUC) | For those receiving hydroxyurea, the AUC will be assessed after the patient has reached MTD. | One time at 24 Months (Study Exit) | |
Secondary | Single Nucleotide Polymorphisms Associated with Change in Percent Hemoglobin F on Hydroxyurea | For those receiving hydroxyurea, we will identify single nucleotide polymorphisms that are associated with a greater change in hemoglobin F percent in response to hydroyxurea therapy. | One Time at 24 Months (Study Exit) |
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