Novel Dose Escalation to Predict Treatment With Hydroxyurea

Completed · Early Phase 1

Conditions studied: Sickle Cell Disease

In brief

Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This can result in painful episodes, serious infections, chronic anemia (a decrease in the number of red blood cells), and damage to body organs. Hydroxyurea therapy offers significant benefits for infants, children, and adolescents with sickle cell anemia. These include a reduction in the frequency of pain crises and acute chest syndrome (inflammation of the lungs) and an increase in hemoglobin (the oxygen-carrying protein) in the blood. Patients on hydroxyurea who receive a maximum tolerated dose (MTD) that is specific for them have greater clinical benefit than those who receive a standard lower dose. There is, however, no way currently to predict the MTD for individual patients. As such, MTD for each patient is currently determined by gradual increases in the dose over several months. This process is time-consuming, requires monthly clinic visits, and delays the benefits of hydroxyurea therapy. Our research group has come up with an equation that could be used to predict each patient's MTD using baseline clinical and laboratory measures before starting hydroxyurea treatment. The purpose of this research study is to compare the use of our equation for predicting MTD to the current standard practice of gradually increasing the hydroxyurea dose until MTD is reached. We want to see if the use of our predictive equation will allow us to achieve MTD faster and with no more side effects than with the standard practice.

Key facts

Study ID

NCT02042222

Run by

Baylor College of Medicine

People needed

70

Starts

2013-10-01

Expected to finish

2020-07-31

Last updated by the study team

2020-11-18

Who can join

Age: 1 and older, up to 16. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• A severe form of sickle cell disease (HbSS or HbS beta-zero thalassemia)
• Age range of 1.0-15.99 years, inclusive, at the time of enrollment
• A decision by the subject's family and primary clinician to initiate hydroxyurea therapy, made independently of recruitment for the study (not applicable for the biological arm patients)
• Informed consent and assent (as applicable) obtained from parent/guardian and child.
• Ability to comply with all study related treatments, evaluations, and follow-up/study exit

You may not qualify if…

• Documented clinical stroke or transient ischemic attack (TIA).
• Known severe vasculopathy or moya-moya disease on brain imaging studies
• Asparagine aminotransferase (AST) or alanine aminotransferase (ALT) greater than three times upper limit of normal or serum creatinine greater than 0.8 mg/dl
• Current participation in other interventional trials. Subjects must have been off any alternative therapy for at least three months prior to enrollment in this study
• Erythrocyte transfusion within the prior 2 months
• Any condition or chronic illness that in the opinion of the primary clinician makes participation in the trial ill advised
• Inability or unwillingness to complete required studies
• Pregnancy or unwillingness to use a medically acceptable form of contraception if sexually active (male OR female)
• Patients excluded from participation due to laboratory abnormalities, participation in other interventional trials, or recent transfusions may be re-screened at a later date

Where it is running

• Texas Children's Hospital — Houston, Texas, United States

Full record on ClinicalTrials.gov

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