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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

Page last updated: August 23, 2015

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