Primary Prevention of Stroke in Children with SCD in Sub-Saharan Africa II: A Multicenter, Open-label, Single-arm Type I Hybrid Clinical Trial

Phase 4

• Conditions: Haematological Disorders; Paediatrics

• Registration Number: PACTR202312522038774
• Lead Sponsor: Vanderbilt University Medical Centre

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2023-08-14
• Last Update Posted: 4 March 2024

Recruitment & Eligibility

• Status: Pending
• Sex: All
• Target Recruitment: 220

Inclusion Criteria

1) diagnosis of HbSS or HbSB0 confirmed by electrophoresis
2) informed consent from the parent/legal guardian and assent from patient
3) two TCD flow velocity readings of >200 cm/sec and < 220 cm/sec or one TCD velocity reading > 220 cm/sec; typically the repeat TCD is performed on the same day so treatment can start immediately
4) age between 5 and 12 years (assessment can take place up until the 13th birthday), which includes the peak age of onset of strokes in SCA, ~ 6 yo
5) ability to swallow the hydroxyurea capsule.

Exclusion Criteria

1) prior stroke or TIA by history, or concern for moderate or severe neurological deficit based on a positive validated 10 questions screening
2) other significant organ system dysfunction or other contraindication to hydroxyurea
3) Children who are already on therapy with either blood transfusion or hydroxyurea therapy
4) significant cytopenias (absolute neutrophil count (ANC) <1500, platelets <150,000/ul, reticulocytes <80,000/ul, unless Hb is > 9 g/dl], renal insufficiency (creatinine > 0.8 mg/dl)
5) other significant organ system dysfunction, or other contraindication to hydroxyurea therapy
6) history of seizures or diagnosis of epilepsy, and 7) metal in the body that would make MRI unsafe.

Study & Design

• Study Type: Interventional
• Study Design: Not specified