Sickle Cell Disease - Stroke Prevention in Nigeria Trial "SPIN"

Active, not recruiting

Phase 1/2 Results N/A

Trial Description

Given large absolute numbers of individuals with sickle cell disease in Nigeria, hydroxyurea therapy for all individuals with sickle cell disease may not be initially feasible; however, a targeted strategy of hydroxyurea use for primary prevention of strokes is an alternative to the standard therapy (observation) for high-risk individuals. The investigators propose a feasibility study, Sickle Cell Disease - Stroke Prevention in Nigeria (SPIN) Trial, to determine whether hydroxyurea can be used for primary prevention of strokes in Nigerian children with sickle cell anemia.

Conditions

Interventions

  • Hydroxyurea (Hydrea┬« and Droxia┬« Other names Return to top Hydroxycarbamide )Drug
    Intervention Desc: Hydroxyurea will be prescribed as an investigational therapy by the treating physician. Recommended guidelines for titration of hydroxyurea to maximal tolerated dose are below. The study intervention will include hydroxyurea to begin at ~20 mg/kg/day (range 17.5 - 26 mg/kg/day). No dose escalation will occur as this dose was shown to have some efficacy in infants with SCA and was associated with rare myelosuppression.(1)
    ARM 1: Kind: Experimental
    Label: Hydroxyurea
    Description: We propose to enroll 40 children with SCA and an elevated TCD measurement between 5 and 12 years of age in this one arm feasibility study of hydroxyurea therapy, with follow-up of at least 12 months per subject. Hydroxyurea will be prescribed as an investigational therapy by the treating physician. Recommended guidelines for titration of hydroxyurea to maximal tolerated dose are below. The study intervention will include hydroxyurea to begin at ~20 mg/kg/day (range 17.5 - 26 mg/kg/day) with 200 mg capsules of hydroxyurea. Dosing will be based on weights as follows: 18 kg to 22.9 kg- 2 capsules; 23 kg to 33.9 kg- 3 capsules; 34 kg to 44.9 kg- 4 capsules. No dose escalation will occur as this dose was shown to have some efficacy with rare myelosuppression toxicity.(2)

Trial Design

  • Masking: Open Label
  • Purpose: Treatment
  • Endpoint: Safety/Efficacy Study
  • Intervention: Single Group Assignment

Outcomes

Type Measure Time Frame Safety Issue
Primary Acceptability of hydroxyurea therapy for primary prevention of strokes in children with sickle cell anemia. 2 years No
Secondary Establish a safety protocol for using hydroxyurea for primary prevention of strokes in a clinical trial setting for one year in a low income country. 12 Months No
Primary Hydroxyurea Therapy Acceptance and Adherence 2 years No
Secondary Hydroxyurea Safety protocol for Children with Sickle Cell Anemia 12 Months No

Sponsors