At a glance
ClinicalIndex Comparison Record- ✓Children with HbSS or sickle hemoglobin (HbS)/β0-thalassemia
- ✓Age 9 to 36 months at study initiation
- ✓Enrollment without restriction by clinical severity
- ✕Receiving chronic red blood cell transfusion therapy
- ✕Recent erythrocyte transfusion within 2 months
- ✕Recent participation (<30 days) in another clinical trial with investigational drug/device
- ✕Condition or chronic illness that makes participation unsafe in investigator's opinion
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies
In Brief
A Phase 2 clinical trial evaluating Hydroxyurea for Sickle Cell Anemia. Completed, enrolled 58 participants across 4 sites.
Detailed Summary
This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or HbSβ\^0thalassemia), regardless of disease severity, to a therapeutic trial. A prior clinical trial at St. Jude Children's Research Hospital (SJCRH) (BABYHUG, NCT01783990) demonstrated that a fixed dose (20 mg/kg/day) of hydroxyurea was safe and effective in decreasing SCA-related complications in very young children (9-18 months), and largely due to these findings, hydroxyurea is recommended to be offered to all children (≥9 months old) with SCA, independent of disease severity. Nevertheless, children in the treatment arm of BABYHUG continued to experience vaso-occlusive symptoms and to incur organ damage. In clinical trials of older children with SCA, intensification of hydroxyurea to a maximum tolerated dosage (MTD), defined by mild to moderate myelosuppression, may be associated with improved laboratory parameters compared to fixed lower-dosing, but the clinical benefits gained from dose intensification have not been described. Therefore, in this trial, children in the standard treatment arm will receive a fixed dose of hydroxyurea (20 mg/kg/day), and participants in the experimental arm will receive hydroxyurea intensified to MTD, defined by a goal absolute neutrophil count (ANC) of 1500-3000 cells/µL. This trial aims to establish a multicenter infrastructure that will identify, enroll and randomize very young children (9-36 months) to receive fixed dose versus intensified-dose hydroxyurea in a single blinded manner, and to obtain prospective pilot data comparing the clinical and laboratory outcomes between the treatment arms to facilitate design of a definitive phase III trial.
Study Details
Timeline
Interventions
Given orally once daily.