Risk Clinical Stratification of Sickle Cell Disease in Nigeria, Assessment of Efficacy/Safety of Hydroxyurea Treatment
Information source: Loyola University
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Sickle Cell Disease; Sickle Cell Anemia
Intervention: hydroxyurea (Drug)
Phase: Phase 4
Status: Enrolling by invitation
Sponsored by: Loyola University Official(s) and/or principal investigator(s): Bamidele O Tayo, PhD, Principal Investigator, Affiliation: Loyola University Chicago Victor R Gordeuk, MD, Principal Investigator, Affiliation: University of Illinois at Chicago Titilola S Akingbola, MBBS, FWACP, Principal Investigator, Affiliation: University of Ibadan College of Medicine, Nigeria Richard S Cooper, MD, Principal Investigator, Affiliation: Loyola University Chicago Lewis Hsu, MD, Principal Investigator, Affiliation: University of Illinois at Chicago
Summary
The vast majority of births with sickle cell disease (SCD) occur in Africa and 90% are
thought to die before the age of five. Hydroxyurea (HU) is the only drug approved by the FDA
for the treatment of sickle cell anemia. Although HU is used to treat small numbers of
patients in Africa, cost, fear of toxicity, and lack of awareness and availability limit its
use. The leukopenia that may be seen with HU raises the possibility of increased
susceptibility to infection. Risk stratification - i. e., identification of patients most
likely to benefit- could focus therapy and provide confidence that the risk: benefit ratio is
favorable. Several clinical measures of future risk are well defined and findings on
modifier genes in the US, primarily related to fetal hemoglobin (HbF), have further improved
risk prediction. Whether the genetic variants predict severity in Africa is not known. The
investigators have established a SCD cohort in Ibadan, Nigeria. In the first phase of this
research the investigators will implement clinical risk examinations and assess the
relationship between clinical characteristics (including levels of HbF) and known genetic
markers. As a proxy for a birth cohort, the investigators will compare the frequency of the
genetic markers in adult patients (i. e., "survivors") to children. In the second phase the
investigators will randomize 40 high risk adult patients to fixed low dose HU or no HU
treatment in a crossover design and monitor hematologic and physiologic parameters to
document hematologic effects and safety. This work will lay the basis for a large-scale
trial to document safety and efficacy.
Clinical Details
Official title: Risk Stratification for Clinical Severity of Sickle Cell Disease in Nigeria and Assessment of Efficacy and Safety During Treatment With Hydroxyurea
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Cytopenia
Secondary outcome: Development of infection evaluated by a physician at the point of care
Eligibility
Minimum age: 18 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Age >= 18 years
- HemoglobinSS (HbSS) or beta-zero (B0) thalassemia genotype
- Hemoglobin concentration >4. 5 g/dL at steady state and time of enrollment
- Absolute neutrophil count >1,500/mircoliter
- Platelet count >95,000/microliter
- Serum creatinine <1. 2 mg/dL
- Alanine transaminase less than two times the upper limit of normal
Exclusion Criteria:
- HIVpositive
- Hepatitis B and/or C positive
Locations and Contacts
University of Ibadan College of Medicine, Ibadan, Oyo State, Nigeria
Additional Information
Starting date: December 2014
Last updated: February 20, 2015
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