Stroke Prevention With Hydroxyurea Enabled Through Research and Education (SPHERE)

Conditions

• Sickle Cell Anemia in Children

Eligibility Criteria

Sex

ALL

Age

2 Years - 16 Years

Healthy Volunteers

Not accepted

Interventions

• Hydroxyurea — DRUG
  Hydroxyurea treatment will be provided to reduce stroke risk. Hydroxyurea treatment will be started at a fixed dose of 20.0 ± 5.0 mg/kg/day, followed by escalation to maximum tolerated dose (MTD).
• Elevated Arm TCD Examination — DIAGNOSTIC_TEST
  TCD examination on children with SCA between ages 2 and 16 years of age will be completed to evaluate their risk of stroke. For children with elevated velocities at initial screening or at 1 Year who receive hydroxyurea therapy, TCD examinations will occur every 6 ± 2 months.
• Normal Arm TCD Examination — DIAGNOSTIC_TEST
  TCD examination on children with SCA between ages 2 and 16 years of age will be completed to evaluate their risk of stroke. TCD examination for all participants will occur at initial screening, at Year 1 (12 ± 3 months), and Year 2 (24 ± 3 months). Children with normal TCD velocities at initial screening will undergo repeat TCD 12 months after enrolment. If the TCD at 12 months has changed to an elevated velocity (conditional or abnormal), the child can begin study treatment (Hydroxyurea).

Study Details

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.

Key Dates

Start date

Apr 24, 2019

Status verified

Jul 2025

Primary completion

Dec 31, 2026

Completion

Dec 31, 2026

Study Design

Enrollment

202 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Elevated Initial Screening TCD
  Those who have an elevated initial screening TCD (either conditional or abnormal TAMV) and will be a treatment cohort that receives open-label hydroxyurea therapy as per the dosing and administration schedule.
• Experimental: Normal Initial Screening TCD
  Those who are found to have a normal TCD at enrolment are a part of the observation/control cohort and will undergo repeat TCD every 12 months after enrolment. If the TCD at 12 months has changed to an elevated velocity (conditional or abnormal), the study participant will be reassigned to the elevated initial screening TCD arm and can begin study treatment (hydroxyurea), but will not be included in the primary endpoint analysis.

Primary Outcome Measure

Prevalence of Elevated TCD [ Time Frame: Baseline ]

Locations (1)

Find similar trials in Cincinnati, OH

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