Risk-based Therapy for Sickle Cell Anemia: A Feasibility Study
Information source: Children's Research Institute
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Sickle Cell Anemia
Intervention: Hydroxyurea (Drug)
Phase: Phase 2
Status: Terminated
Sponsored by: Children's Research Institute
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.
Clinical Details
Official title: Reticulocyte as Risk Marker: Targeted Therapy for Infants With Sickle Cell Anemia
Study design: Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Prevention
Primary outcome: Sickle Cell Clinical Events
Eligibility
Minimum age: 6 Months.
Maximum age: 12 Months.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- ages 6-12 months
- Sickle cell anemia (HbSS)
- steady state absolute reticulocyte count between 2-6 months is available in the
medical record
Exclusion Criteria:
- receiving hydroxyurea or chronic monthly blood transfusions
- patient enrolled in preliminary study
Locations and Contacts
Children's National Medical Center, Washington, District of Columbia 20010, United States
Additional Information
Starting date: February 2014
Last updated: June 19, 2015
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