drugset / Trial / NCT01389024
Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease
Phase 2
Completed
28 enrolled
Johns Hopkins University
Children's Hospital of Philadelphia · collabChildren's Mercy Hospital Kansas City · collabColumbia University · collabMedical University of South Carolina · collabNational Center for Research Resources (NCRR) · collabRTI International · collabSinai Hospital of Baltimore · collabUniversity of Alabama at Birmingham · collabVanderbilt University School of Medicine · collabWashington University School of Medicine · collab
RandomizedParallel-groupTriple-blindPrevention
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.
Timeline
- Start
- 2012-08-16
- Primary completion
- 2022-05-24
- Completion
- 2022-05-24
Publications
- Background Kwiatkowski JL, Zimmerman RA, Pollock AN, Seto W, Smith-Whitley K, Shults J, Blackwood-Chirchir A, Ohene-Frempong K. Silent infarcts in young children with sickle cell disease. Br J Haematol. 2009 Aug;146(3):300-5. doi: 10.1111/j.1365-2141.2009.07753.x. Epub 2009 Jun 4.
- Background Zimmerman SA, Schultz WH, Burgett S, Mortier NA, Ware RE. Hydroxyurea therapy lowers transcranial Doppler flow velocities in children with sickle cell anemia. Blood. 2007 Aug 1;110(3):1043-7. doi: 10.1182/blood-2006-11-057893. Epub 2007 Apr 11.
- Background Strouse JJ, Cox CS, Melhem ER, Lu H, Kraut MA, Razumovsky A, Yohay K, van Zijl PC, Casella JF. Inverse correlation between cerebral blood flow measured by continuous arterial spin-labeling (CASL) MRI and neurocognitive function in children with sickle cell anemia (SCA). Blood. 2006 Jul 1;108(1):379-81. doi: 10.1182/blood-2005-10-4029. Epub 2006 Mar 14.
- Background Casella JF, King AA, Barton B, White DA, Noetzel MJ, Ichord RN, Terrill C, Hirtz D, McKinstry RC, Strouse JJ, Howard TH, Coates TD, Minniti CP, Campbell AD, Vendt BA, Lehmann H, Debaun MR. Design of the silent cerebral infarct transfusion (SIT) trial. Pediatr Hematol Oncol. 2010 Mar;27(2):69-89. doi: 10.3109/08880010903360367.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Hydroxyurea | Other / unclassified | 35 mg/kg | Oral |