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ENHANCE: PK-Guided Hydroxyurea in Pediatric Sickle Cell Anemia
NCT07177300Phase 4
Key facts
- Status
- Recruiting
- Start
- Dec 2024
- Primary completion
- Dec 2026
- Countries
- 1
- Sponsor
- Cincinnati Children's Hospital Medical Center
- Modality
- Small Molecules
- Drugs
- hydroxyurea
- Conditions
- sickle cell anemia (SCA)sickle beta zero thalassemia (HbS/β⁰-thalassemia)
01
Overview
Phase 4, open-label, single-arm trial of hydroxyurea in sickle cell anemia (SCA) and sickle beta zero thalassemia (HbS/β⁰-thalassemia), sponsored by Cincinnati Children's Hospital Medical Center. Primary endpoint: Composite Organ Injury at 10 years. Started Dec 2024; status: recruiting.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
Observational Treatment Group
Single arm receiving PK-optimized hydroxyurea.
03
Who it enrols
Children aged 6 months to 5 years with Sickle Cell Anemia (HbSS) or Sickle-beta0-thalassemia, excluding those on regular transfusions or with HbSC/HbSbeta+ genotypes.
04
Endpoints
Primary
Composite Organ Injury
Secondary
Fetal Hemoglobin Percentage
Hemoglobin Concentration
Reticulocyte Count
Absolute Neutrophil Count
Mean Cell Volume
F-cell Fraction
Adverse Events
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