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

July 27, 2020 updated by: St. Jude Children's Research Hospital

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.

Study Overview

Status

Completed

Conditions

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.

Study Type

Observational

Enrollment (Actual)

260

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • St. Jude Children's Research Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 30 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Study participants will be patients with sickle cell disease who receive medical care from the Department of Hematology staff of St. Jude Children's Research Hospital. All patients on hydroxyurea therapy or patients who are initiating hydroxyurea therapy will be invited to participate.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Patients With Prior Hydroxyurea
Patients who have received hydroxyurea therapy before entering the study.
Patients Without Prior Hydroxyurea
Patients who have not received hydroxyurea before study entry.

What is the study measuring?

Primary Outcome Measures

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;
Time Frame: Every 3 years
Every 3 years
DNA damage from hydroxyurea therapy-percentage of HJB in immature (CD71+) erythrocytes
Time Frame: Every 3 years
Every 3 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Brain function as measured by MRI/MRA and TCD
Time Frame: Every 3 years
optional test
Every 3 years
Splenic function as measured by Spleen Scan
Time Frame: Every 3 years
optional test
Every 3 years
Kidney function as measured by BUN/creatinine and Urinalysis, glomerular filtration rate (GFR)
Time Frame: Every 3 years
optional test
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
collected if performed for clinical purposes
Every 3 years
Growth as measured by height and weight
Time Frame: Every visit
Every visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

St. Jude Children's Research Hospital

Collaborators

Children's Hospital Medical Center, Cincinnati

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 3, 2006

Primary Completion (Actual)

April 23, 2015

Study Completion (Actual)

April 23, 2015

Study Registration Dates

First Submitted

March 17, 2006

First Submitted That Met QC Criteria

March 17, 2006

First Posted (Estimate)

March 21, 2006

Study Record Updates

Last Update Posted (Actual)

July 28, 2020

Last Update Submitted That Met QC Criteria

July 27, 2020

Last Verified

July 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HUSTLE

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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