SACRED A Prospective Research Study to Reduce Stroke in Children With Sickle Cell Anemia "SACRED"

Recruiting

Phase N/A Results N/A

Trial Description

Prospective screening and treatment study for children with Sickle Cell Anemia and increased stroke risk living in the Dominican Republic.

Detailed Description

SACRED involves a three-part study design, as outlined below, to include (1) initial TCD evaluation phase; (2) longitudinal TCD evaluation; and (3) treatment if warranted.
1. The initial evaluation portion of SACRED will involve obtaining TCD examinations on children with SCA between ages 3-15 years, who are followed at Hospital Infantil Robert Reid Cabral in Santo Domingo to evaluate their risk of stroke. Up to 500 patients will be enrolled. All patients, including those who are already on hydroxyurea and transfusion therapy (whether for stroke or other clinical indications), will be included to obtain a one-year cross-sectional description of TCD velocities in this patient population. Patients who are already on therapy and identified to have elevated TCD velocities will be eligible for the treatment portion of SACRED as a conditional or elevated velocity would suggest that their present therapy was not optimized.
2. In the longitudinal portion of SACRED, all enrolled children will undergo yearly TCD examination. The goal of serial examination is to help define the natural history of cerebrovascular disease, specifically to determine the incidence of new conditional or abnormal velocities. The goal is to obtain a total of 3 TCD examinations per enrolled patient, regardless of treatment status.
3. In the treatment phase of SACRED, those children with TCD velocities between 170-199 cm/sec will be eligible for protocol-directed hydroxyurea therapy. Most participants will initiate hydroxyurea treatment but those who are already on hydroxyurea and have conditional velocities will receive dose optimization. Participants will be followed until a common study termination date, defined as 3 years from the first treatment. Participants with abnormal TCD velocities ≥200 cm/sec will commence with transfusion therapy per current practice guidelines at the clinical site. Patients already on transfusion therapy identified to have conditional velocities will also be eligible for hydroxyurea and those with abnormal velocities may require re-calculation of transfusion dosing.

Conditions

Interventions

  • Hydroxyurea (Hydrea® and Droxia® Other names Return to top Hydroxycarbamide )Drug
    Intervention Desc: drug to be administered
    ARM 1: Kind: Experimental
    Label: Treatment Phase
    Description: Those children with TCD velocities between 170-199 cm/sec will be eligible for protocol-directed hydroxyurea therapy. Most participants will initiate hydroxyurea treatment but those who are already on hydroxyurea and have conditional velocities will receive dose optimization. Participants will be followed until a common study termination date, defined as 3 years from the first treatment. Participants with abnormal TCD velocities ≥200 cm/sec will commence with transfusion therapy per current practice guidelines at the clinical site. Patients already on transfusion therapy identified to have conditional velocities will also be eligible for hydroxyurea and those with abnormal velocities may require re-calculation of transfusion dosing.
  • TCD examination Procedure
    Intervention Desc: TCD examinations on children with SCA between ages 3-15 years will be completed to evaluate their risk of stroke. All enrolled children will undergo yearly TCD examination. Participants with conditional TCD velocities on hydroxyurea therapy per study protocol will undergo TCD examinations every 6 months.
    ARM 1: Kind: Experimental
    Label: Longitudinal Portion
    Description: All enrolled children will undergo yearly TCD examination. The goal of serial examination is to help define the natural history of cerebrovascular disease, specifically to determine the incidence of new conditional or abnormal velocities. The goal is to obtain a total of 3 TCD examinations per enrolled patient, regardless of treatment status.
    ARM 2: Kind: Experimental
    Label: Treatment Phase
    Description: Those children with TCD velocities between 170-199 cm/sec will be eligible for protocol-directed hydroxyurea therapy. Most participants will initiate hydroxyurea treatment but those who are already on hydroxyurea and have conditional velocities will receive dose optimization. Participants will be followed until a common study termination date, defined as 3 years from the first treatment. Participants with abnormal TCD velocities ≥200 cm/sec will commence with transfusion therapy per current practice guidelines at the clinical site. Patients already on transfusion therapy identified to have conditional velocities will also be eligible for hydroxyurea and those with abnormal velocities may require re-calculation of transfusion dosing.

Trial Design

  • Allocation: Non-Randomized
  • Masking: Open Label
  • Purpose: Supportive Care
  • Endpoint: Safety/Efficacy Study
  • Intervention: Single Group Assignment

Outcomes

Type Measure Time Frame Safety Issue
Primary Transcranial Doppler Ultrasound examinations 0-24 months Yes
Secondary Hydroxyurea toxicities 0-30 months Yes

Sponsors