Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies

Completed · Phase 2

Conditions studied: Sickle Cell Anemia

In brief

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.

Key facts

Study ID

NCT03020615

Run by

St. Jude Children's Research Hospital

People needed

58

Starts

2017-05-12

Expected to finish

2020-06-08

Last updated by the study team

2021-06-25

Who can join

Age: 1 and older, up to 3. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Children with HbSS or sickle hemoglobin (HbS)/β\^0thalassemia
• ≥9 to ≤ 36 months of age at study initiation
• Enrollment will occur irrespective of clinical severity

You may not qualify if…

• Permanent:
• Receiving chronic red blood cell transfusion therapy.
• Condition or chronic illness, which in the opinion of the PI makes participation unsafe.
• Transient (participants may be re-evaluated after ≥14 days):
• Recent (<30 days) participation in another clinical intervention trial utilizing an investigational new drug/investigational device exemption (IND/IDE) agent.
• Erythrocyte transfusion in the past 2 months.
• Laboratory Assessments:
• Hemoglobin <6.0 g/dL
• Absolute reticulocyte count <80 * 10\^3/µL if hemoglobin <9.0 mg/dL
• Absolute neutrophil count <1.5 * 10\^3/µL
• Platelet count <100 * 10\^3/µL
• Serum creatinine > twice the upper limit of normal for age
• Alanine aminotransferase (ALT) > twice the upper limit of normal

Where it is running

• Emory University/Children's Health Care of Atlanta — Atlanta, Georgia, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• University of Texas Southwestern Medical Center at Dallas — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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