Overview

This trial is active, not recruiting.

Condition sickle cell disease
Sponsor St. Jude Children's Research Hospital
Collaborator Children's Hospital Medical Center, Cincinnati
Start date March 2006
End date January 2026
Trial size 260 participants
Trial identifier NCT00305175, HUSTLE

Summary

The primary objectives of this prospective, observational study are (1) to describe the long-term cellular, molecular, and clinical effects of hydroxyurea therapy in sickle cell disease, and (2) to perform hydroxyurea pharmacokinetics studies.

This study will follow sickle cell patients being treated with hydroxyurea for a long period of time to evaluate the long-term cellular and molecular effects of the drug on the patients' body. This study will consist of two patient groups. One group will be made up of patients who have received hydroxyurea therapy before entering the study. The second group will be made up of patients who have not received hydroxyurea before study entry.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm
Patients who have received hydroxyurea therapy before entering the study.
Patients who have not received hydroxyurea before study entry.

Primary Outcomes

Measure
DNA damage from hydroxyurea therapy-variable-diversity-joining (VDJ) recombination events defined as the number of events per microgram of genomic DNA;
time frame: Every 3 years
DNA damage from hydroxyurea therapy-percentage of HJB in immature (CD71+) erythrocytes
time frame: Every 3 years

Secondary Outcomes

Measure
Brain function as measured by MRI/MRA and TCD
time frame: Every 3 years
Splenic function as measured by Spleen Scan
time frame: Every 3 years
Kidney function as measured by BUN/creatinine and Urinalysis, glomerular filtration rate (GFR)
time frame: Every 3 years
Lung function as measured by forced vital capacity (FVC) (%), forced vital volume in 1 second (FVC1) (%), and tricuspid regurgitation (TR) jet on Echocardiogram (ECHO)
time frame: Every 3 years
Growth as measured by height and weight
time frame: Every visit

Eligibility Criteria

Male or female participants up to 30 years old.

Inclusion Criteria: - Patients from birth up to age 30 years - Diagnosis of sickle cell disease - Patients who are receiving hydroxyurea therapy or plan to begin hydroxyurea therapy

Additional Information

Official title Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease
Principal investigator Jeremie Estepp, MD
Description Many years of study have documented the severe effects of sickle cell disease. Some of these effects include hemolysis (the break down of red blood cells), blockages in the blood vessels, and damage to the organs systems of the body. Hydroxyurea, which is given by mouth, is used to effectively prevent blockages in the blood vessels of patients with sickle cell disease. The hydroxyurea dosage varies and the responses of the body to this drug are not well understood. This study will follow sickle cell patients being treated with hydroxyurea for a long period of time to evaluate the long-term cellular and molecular effects of the drug on the patients' body. This study will consist of two patient groups. One group will be made up of patients who have received hydroxyurea therapy before entering the study (Old Cohort). The second group will be made up of patients who have not received hydroxyurea before study entry (New Cohort). This is not a therapeutic drug trial. Subjects for this study will receive hydroxyurea therapy for accepted clinical indications, and will be treated per best clinical management using treatment algorithms established at St. Jude Children's Research Hospital and other pediatric sickle cell programs across the United States. Hydroxyurea therapy data (such as dosing and duration of therapy) will not be dictated by this study, but will be collected to correlate with long-term outcomes. Hydroxyurea dose escalation to a stable MTD will occur according to published guidelines.
Trial information was received from ClinicalTrials.gov and was last updated in June 2016.
Information provided to ClinicalTrials.gov by St. Jude Children's Research Hospital.