Phase 3
N=193
Hydroxyurea to Prevent Organ Damage in Children With Sickle Cell Anemia
Hematologic Diseases · Anemia, Sickle Cell
Bottom Line
View on ClinicalTrials.gov: NCT00006400 ↗Enrolled (actual)
193
Serious AEs
28.0%
Results posted
Aug 2020
Primary outcome: Primary: Treatment Differences of the Change in Qualitative Splenic Function From Baseline — 19; 28; 51; 46 Participants — p=0.21
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Hydroxyurea (Drug); Placebo (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- Primary completion
- Sep 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Treatment Differences of the Change in Qualitative Splenic Function From Baseline |
19; 28; 51; 46 | 0.21 |
| SECONDARY Change From Baseline in the Renal Function That Was Measured by Diethylenetriaminepentaacetic Acid (DTPA) Glomerular Filtration Rate (GFR) |
22.56; 20.74 | 0.93 |
| SECONDARY Change From Baseline in the Renal Function That Was Measured by Glomerular Filtration Rate (GFR) (Calculated Using Schwartz Formula) |
28.65; 33.36 | 0.43 |
| SECONDARY Change From Baseline in the Renal Function That Was Measured by GFR (Calculated Using New Schwartz Formula) |
10.57; 14.33 | 0.48 |
Summary
The purpose of this study is to determine if hydroxyurea therapy is effective in the prevention of chronic end organ damage in pediatric patients with sickle cell anemia.
Eligibility Criteria
Inclusion Criteria
- Majority fetal and sickle (FS or SF) hemoglobin pattern confirmed centrally by electrophoresis (screening may begin at 7 months of age)
Exclusion Criteria
- Chronic transfusion therapy
- Cancer
- Less than 5th percentile (10th percentile for the pilot study) height, weight, or head circumference for age
- Severe developmental delay (e.g., cerebral palsy or other mental retardation, Grade III/IV intraventricular hemorrhage)
- Stroke with neurological deficit
- Surgical splenectomy
- Participating in other clinical intervention trials
- Probable or known diagnosis of Hemoglobin S-Hereditary Persistence of Fetal Hemoglobin
- Known hemoglobin S-beta plus thalassemia (hemoglobin A present)
- Any condition or chronic illness, which in the opinion of the principal investigator, makes participation unadvised or unsafe
- Inability or unwillingness to complete baseline (pre-enrollment) studies, including blood or urine specimen collection, liver-spleen scan, abdominal sonogram, neurological examination, neuropsychological testing, or transcranial Doppler ultrasound (interpretable study not required, but confirmed velocity greater than 200 cm/sec results in ineligibility)
- Previous or current treatment with hydroxyurea (HU) or another anti-sickling drug
- The following exclusion criteria are transient; patients can be re-evaluated for eligibility:
- Hemoglobin less than 6.0 gm/dL
- Reticulocyte count less than 80, 000/cu mm if hemoglobin is less than 9 gm/dL
- Neutrophil count less than 2,000/cu mm
- Platelet count less than 130,000/cu mm
- Blood transfusion in the 2 months prior to study entry unless HbA is less than 10%
- ALT greater than twice the upper limit of normal
- Ferritin less than 10 ng/ml
- Serum creatinine greater than twice the upper limit of normal for age
- Bayley standardized mental score below 70
Data sourced from ClinicalTrials.gov (NCT00006400). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.