Hydroxyurea for Children and Young Adults With Sickle Cell Disease and Pulmonary Hypertension
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00350844|
Recruitment Status : Terminated (Low subject accrual)
First Posted : July 11, 2006
Results First Posted : May 8, 2013
Last Update Posted : May 8, 2013
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Disease Pulmonary Hypertension||Drug: Hydroxyurea||Phase 1 Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study of Hydroxyurea for the Treatment of Pulmonary Hypertension in Children and Young Adults With Sickle Cell Disease|
|Study Start Date :||July 2006|
|Actual Primary Completion Date :||June 2008|
|Actual Study Completion Date :||June 2008|
U.S. FDA Resources
20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.
- Tricuspid Regurgitant Jet Velocity [ Time Frame: 6 and 12 months after HU therapy begins ]Primary outcome measure was tricuspid regurgitant jet velocity (TRJV) by echocardiogram after 6 and 12 months of hydroxyurea therapy.
- Compliance [ Time Frame: Throughout study ]Secondary outcome measures included compliance; laboratory measures of therapy-related toxicity; laboratory biomarkers for hemolysis, oxidative stress and endothelial injury; and quality of life measures by Child Health Questionnaire (CHQ).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00350844
|United States, Illinois|
|Children's Memorial Hospital|
|Chicago, Illinois, United States, 60614-3394|
|Principal Investigator:||Robert I Liem, MD||Ann & Robert H Lurie Children's Hospital of Chicago|