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Risk Clinical Stratification of Sickle Cell Disease in Nigeria, Assessment of Efficacy/Safety of Hydroxyurea Treatment

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02149537
Recruitment Status : Active, not recruiting
First Posted : May 29, 2014
Last Update Posted : January 5, 2021
University of Illinois at Chicago
University of Ibadan
Information provided by (Responsible Party):
Bamidele Tayo, Loyola University

Brief 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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Sickle Cell Anemia Drug: hydroxyurea Phase 4

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 53 participants
Allocation: Non-Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: Adult patients to start to receive fixed low dose hydroxyurea (10 mg/kg) per day for six months and then crossover to no hydroxyurea treatment for six months, or start with no hydroxyurea treatment for six months and then crossover to receive fixed low dose hydroxyurea (10 mg/kg) per day for six months.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Risk Stratification for Clinical Severity of Sickle Cell Disease in Nigeria and Assessment of Efficacy and Safety During Treatment With Hydroxyurea
Study Start Date : December 2014
Actual Primary Completion Date : September 2017
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

Arm Intervention/treatment
Experimental: hydroxyurea
500mg of hydroxyurea/day during 6 months
Drug: hydroxyurea
Other Name: Hydroxycarbamide

No Intervention: No treatment
No hydroxyurea treatment during 6 months

Primary Outcome Measures :
  1. Cytopenia [ Time Frame: every 2 weeks during a period of 6 months ]
    Neutrophil count <500/microliter, platelet count <50,000 or a reticulocyte count<95,000 with Hemoglobin of 9.0 g/dL

Secondary Outcome Measures :
  1. Development of infection evaluated by a physician at the point of care [ Time Frame: every 2 weeks for period of 6 months ]
    Infections such as malaria or tuberculosis, which may be newly acquired or recrudescent.

Other Outcome Measures:
  1. laboratory values of Hemoglobin F%, hemoglobin concentration, reticulocyte count, mean corpuscular volume and white blood cell count. [ Time Frame: baseline, 3 months and 6 months. ]
  2. Clinical complications such as acute pain episode, acute chest syndrome and need for blood transfusion. [ Time Frame: every 2 weeks for a period of 6 months. ]
    Evaluated by a nurse or physician at point of care.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02149537

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University of Ibadan College of Medicine
Ibadan, Oyo State, Nigeria
Sponsors and Collaborators
Loyola University
University of Illinois at Chicago
University of Ibadan
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Principal Investigator: Bamidele O Tayo, PhD Loyola University Chicago
Principal Investigator: Victor R Gordeuk, MD University of Illinois at Chicago
Principal Investigator: Titilola S Akingbola, MBBS, FWACP University of Ibadan College of Medicine, Nigeria
Principal Investigator: Richard S Cooper, MD Loyola University Chicago
Principal Investigator: Lewis Hsu, MD University of Illinois at Chicago
Publications of Results:
Other Publications:
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Responsible Party: Bamidele Tayo, Assistant profesor, Loyola University Identifier: NCT02149537    
Other Study ID Numbers: 205449
First Posted: May 29, 2014    Key Record Dates
Last Update Posted: January 5, 2021
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Bamidele Tayo, Loyola University:
sickle cell disease
sickle cell anemia
low income country
middle income country
developing country
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors