Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea

Summary

The primary goal of the Phase III TWiTCH trial is to compare 24 months of alternative therapy (hydroxyurea) to standard therapy (transfusions) for pediatric subjects with sickle cell anemia and abnormally high (≥200 cm/sec) Transcranial Doppler (TCD) velocities, who currently receive chronic transfusions to reduce the risk of primary stroke. For the alternative treatment regimen (hydroxyurea) to be declared non-inferior to the standard treatment regimen (transfusions), after adjusting for baseline differences, the hydroxyurea-treated group must have a mean TCD velocity similar to that observed with transfusion prophylaxis.

Conditions

• Sickle Cell Anemia

Interventions

DRUG

Hydroxyurea

Capsules (300 mg, 400 mg, or 500 mg) taken once daily liquid formulation (100 mg/mL)

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• Boston Children's Hospital

  collaborator OTHER

• University of Texas Southwestern Medical Center

  collaborator OTHER

• Children's Healthcare of Atlanta

  collaborator OTHER

• Children's Hospital of Philadelphia

  collaborator OTHER

• The Hospital for Sick Children

  collaborator OTHER

• Children's National Research Institute

  collaborator OTHER

• Columbia University

  collaborator OTHER

• St. Jude Children's Research Hospital

  collaborator OTHER

• University Hospitals Cleveland Medical Center

  collaborator OTHER

• University of South Alabama

  collaborator OTHER

• Medical University of South Carolina

  collaborator OTHER

• University of Alabama at Birmingham

  collaborator OTHER

• University of Miami

  collaborator OTHER

• University of Mississippi Medical Center

  collaborator OTHER

• Wayne State University

  collaborator OTHER

• Children's Hospital of The King's Daughters

  collaborator OTHER

• Nemours Children's Clinic

  collaborator OTHER

• Duke University

  collaborator OTHER

• East Carolina University

  collaborator OTHER

• Children's Hospitals and Clinics of Minnesota

  collaborator OTHER

• Ann & Robert H Lurie Children's Hospital of Chicago

  collaborator OTHER

• Baylor College of Medicine

  collaborator OTHER

• State University of New York - Downstate Medical Center

  collaborator OTHER

• Steven and Alexandra Cohen Children's Medical Center

  collaborator INDIV

• Children's Hospital Medical Center, Cincinnati

  lead OTHER

Principal Investigators

• Russell E. Ware, MD, PhD · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

4 Years

Max Age

15 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2011-08-31

Primary Completion

2015-03-31

Completion

2015-11-30