NCT00350844

Hydroxyurea for Children and Young Adults With Sickle Cell Disease and Pulmonary Hypertension

A Pilot Study of Hydroxyurea for the Treatment of Pulmonary Hypertension in Children and Young Adults With Sickle Cell Disease

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago · Academic / Other
• Sex: All
• Age: 10 Years – 25 Years
• Healthy volunteers: Not accepted

Summary

The goal of this study is to test the hypothesis that hydroxyurea is effective for the specific treatment of secondary pulmonary hypertension found on screening in children and young adults with sickle cell disease.

Detailed description

Increasing evidence suggests that pulmonary hypertension, defined by an elevated tricuspid regurgitant jet velocity (TRJV) on echocardiogram, is a major cause of morbidity and mortality in adults with sickle cell disease (SCD). However, both the prevalence and optimal treatment of pulmonary hypertension in children and young adults with SCD are unknown. We hypothesize that short term therapy with hydroxyurea will decrease TRJV in children and young adults with pulmonary hypertension found on screening. Patients eligible for treatment will have had evidence of pulmonary hypertension on at least 2 screening echocardiograms. Baseline laboratory tests will be obtained and other causes of secondary pulmonary hypertension will be excluded prior to initiation of treatment. Patients will be treated with hydroxyurea according to a standard dose escalation schedule for a total of 12 months. A clinic visit will be required every 2 months and standard screening for toxicity will be performed monthly. There will be an interim analysis of the primary outcome at 6 months following therapy.

Conditions

• Sickle Cell Disease
• Pulmonary Hypertension

Interventions

Type	Name	Description
DRUG	Hydroxyurea	20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.

Timeline

• Start date: 2006-07-01
• Primary completion: 2008-06-01
• Completion: 2008-06-01
• First posted: 2006-07-11
• Last updated: 2019-08-06
• Results posted: 2013-05-08

Locations

1 site across 1 country: United States