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Cytotoxic agent

Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study

Phase 2
Waitlist Available
Research Sponsored by Children's National Research Institute
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up every 4 weeks for the 18 months study duration
Awards & highlights
Approved for 5 Other Conditions
All Individual Drugs Already Approved

Summary

Sickle cell anemia (SCA) patients experience organ damage that begins at an early age and results in significant morbidity and early mortality. Although all SCA patients share the same genetic mutation, the clinical complications are highly variable with some patients experiencing frequent and severe complications, while others have few serious complications. If SCA severity could be predicted early in life, those patients at greatest risk for complications could receive treatment prior to the onset of organ damage. No general SCA severity predictor or one that can be informative early in life exists. The investigators preliminary research has identified the absolute reticulocyte count (ARC) as a potential early predictive risk marker for SCA complications in pediatric patients. A higher ARC between ages 2 and 6 months of age is associated with an increased risk of hospitalization in the first 3 years of life; the mean ARC for the 36 patients who were hospitalized for SCA complications was significantly higher than that of the remaining 23 in those who were not hospitalized. Moreover, total hospitalizations were nearly three times higher by age 2 years in those infants who had an ARC of \> 200 than for those infants whose ARC was \<200. The proposed study will determine if ARC can be used as a risk-stratifier in asymptomatic infants with SCA and ascertain its value in targeting hydroxyurea therapy to those infants at highest risk of SCA sequelae.

Eligible Conditions
  • Sickle Cell Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~every 4 weeks for the 18 months study duration
This trial's timeline: 3 weeks for screening, Varies for treatment, and every 4 weeks for the 18 months study duration for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Sickle Cell Clinical Change from Baseline to Study Completion ( 18 months)

Awards & Highlights

Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

2Treatment groups
Active Control
Placebo Group
Group I: HydroxyureaActive Control1 Intervention
Treatment Arm
Group II: Sugar waterPlacebo Group1 Intervention
Placebo arm

Find a Location

Who is running the clinical trial?

Children's National Research InstituteLead Sponsor
223 Previous Clinical Trials
258,395 Total Patients Enrolled
~0 spots leftby Dec 2025