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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00350844
• Other study ID #: 12735
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: July 2006
• Est. completion date: June 2008

Study information

• Verified date: July 2019
• Source: Ann & Robert H Lurie Children's Hospital of Chicago
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 6
• Est. completion date: June 2008
• Est. primary completion date: June 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years to 25 Years

Eligibility

Inclusion Criteria:

• Age between 10 and 25 years old

• Sickle cell disease with hemoglobin SS, SC or S-B^0 thalassemia confirmed on hemoglobin electrophoresis

• Tricuspid regurgitant jet velocity (TRJV) equal to or greater than 2.5 m/sec on 2 baseline Doppler echocardiograms at least 3 months apart

Exclusion Criteria:

• Patients already being treated with hydroxyurea

• Patients on a chronic transfusion protocol

• Patients with evidence of hepatic (alanine aminotransferase [ALT] equal to or greater than 2 SD above normal) or renal dysfunction (creatinine [Cr] equal to or greater than 2 SD above normal)

• Patients who are pregnant

• Patients with documented causes of severe pulmonary hypertension other than from SCD

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Hypertension
• Hypertension, Pulmonary
• Pulmonary Hypertension
• Sickle Cell Disease

Intervention

Drug:
• Hydroxyurea
20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.

Locations

Country	Name	City	State
United States	Ann & Robert H Lurie Children's Hospital of Chicago	Chicago	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
Ann & Robert H Lurie Children's Hospital of Chicago

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Tricuspid Regurgitant Jet Velocity	Primary outcome measure was tricuspid regurgitant jet velocity (TRJV) by echocardiogram after 6 and 12 months of hydroxyurea therapy.	6 and 12 months after HU therapy begins
Secondary	Compliance	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).	Throughout study