Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease

Long Term Effects of Hydroxyurea Therapy in Children With Sickle Cell Disease

Sponsors

Lead Sponsor: St. Jude Children's Research Hospital

Collaborator: Children's Hospital Medical Center, Cincinnati

Source St. Jude Children's Research Hospital
Brief 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.

Detailed 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.

Overall Status Completed
Start Date March 3, 2006
Completion Date April 23, 2015
Primary Completion Date April 23, 2015
Study Type Observational
Primary Outcome
Measure Time Frame
DNA damage from hydroxyurea therapy-variable-diversity-joining (VDJ) recombination events defined as the number of events per microgram of genomic DNA; Every 3 years
DNA damage from hydroxyurea therapy-percentage of HJB in immature (CD71+) erythrocytes Every 3 years
Secondary Outcome
Measure Time Frame
Brain function as measured by MRI/MRA and TCD Every 3 years
Splenic function as measured by Spleen Scan Every 3 years
Kidney function as measured by BUN/creatinine and Urinalysis, glomerular filtration rate (GFR) 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) Every 3 years
Growth as measured by height and weight Every visit
Enrollment 260
Condition
Eligibility

Sampling Method: Non-Probability Sample

Criteria:

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

Gender: All

Minimum Age: N/A

Maximum Age: 30 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Jeremie Estepp, MD Principal Investigator St. Jude Children's Research Hospital
Location
Facility: St. Jude Children's Research Hospital
Location Countries

United States

Verification Date

July 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Arm Group

Label: Patients With Prior Hydroxyurea

Description: Patients who have received hydroxyurea therapy before entering the study.

Label: Patients Without Prior Hydroxyurea

Description: Patients who have not received hydroxyurea before study entry.

Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

Source: ClinicalTrials.gov