Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease

Summary

This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease. The investigators plan to identify children 12 to 48 months old without central nervous system complications and randomly assign 20 to treatment with hydroxyurea and 20 to treatment with placebo for 36 months. Neither the study doctors nor the participants will know which treatment they are receiving.

Conditions

• Sickle Cell Disease
• Stroke

Interventions

DRUG

Hydroxyurea

Hydroxyurea solution 100 mg/ml with a starting dose of 20 mg/kg/day by mouth once daily and escalation by 5 mg/kg/day every 8 weeks until hematological toxicity, an Absolute Neutrophil Count of 2000 to 4000/ul, or a maximum dose of 35 mg/kg/day.

DRUG

Placebo

Sucrose solution 0.2 ml/kg/day by mouth once a day with blinded dose escalation of 0.05 ml/kg/day to match the frequency of dose escalation in the hydroxyurea arm.

Sponsors & Collaborators

• National Center for Research Resources (NCRR)

  collaborator NIH

• Washington University School of Medicine

  collaborator OTHER

• Vanderbilt University School of Medicine

  collaborator OTHER

• University of Alabama at Birmingham

  collaborator OTHER

• Children's Hospital of Philadelphia

  collaborator OTHER

• Medical University of South Carolina

  collaborator OTHER

• RTI International

  collaborator OTHER

• Columbia University

  collaborator OTHER

• Children's Mercy Hospital Kansas City

  collaborator OTHER

• Sinai Hospital of Baltimore

  collaborator OTHER

• Johns Hopkins University

  lead OTHER

Principal Investigators

• James F. Casella, MD · Johns Hopkins University

Study Design

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

TRIPLE

Model

PARALLEL

Eligibility

Min Age

12 Months

Max Age

54 Months

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2012-08-16

Primary Completion

2022-05-24

Completion

2022-05-24

FDA Drug

Yes

Countries

• United States

Study Locations