Status:
COMPLETED
Hydroxyurea and Erythropoietin to Treat Sickle Cell Anemia
Lead Sponsor:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Sickle Cell Disease
Chronic Kidney Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
This study will examine the use of hydroxyurea and erythropoietin for treating sickle cell disease in patients who also have kidney disease or pulmonary hypertension (high blood pressure in the lungs)...
Detailed Description
Sickle cell disease (SCD) is a genetic disease that afflicts over eighty thousand Americans, 4 to 5,000 newborns per year in the US, and 100s of thousands of children and adults world-wide. This disea...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Patients with homozygous SCD or other sickling disorders (e.g., B(0) Thalassemia/Sickle) who are 18 years of age or greater will be eligible for treatment. Patients currently being followed on an NIH study or at Howard University on stable doses of hydroxyurea are also eligible. A total of 60 patients will be recruited to the study, with the recognition from our earlier studies of a failure-to-complete rate approaching 50%.
- Patients must have documented hemoglobin S-only or S-beta(0)-thalassemia.
- Patients must have relatively well preserved hepatic function (less than 3 X upper limits of normal ALT).
- Patients must be able to provide informed consent.
- Patients must have:
- an eGFR of 15 to 60 ml/min per 1.73 m(2) BSA,
- or
- an eGFR of 61 - 90 ml/min per 1.73 m(2) BSA and greater than 16.9 mg of albumin/g creatinine (greater than 0.017 ratio g/g),
- and/or
- a trans-thoracic echocardiographic measurements of pulmonary artery pressure (PAP), as estimated by tricuspid regurgitant velocity, of greater than 2.5 m sec(-1) monthly at baseline times two.
- EXCLUSION CRITERIA:
- Patients who are doubly heterozygous for hemoglobin-S and fully or partially expressed hemoglobin-A or any other non-S beta-type globin chain, or hemoglobin A-only (non-sickle cell).
- Patients who are on a chronic transfusion program, defined as regular transfusions every 2-8 weeks.
- Patients who are pregnant or breast-feeding.
- Patients who have a history of a documented cerebrovascular accident or venous thrombosis within one year of study entry.
- Patients with active proliferative retinopathy within 1 year of study entry
- Patients with eGFR less than or equal to 14 ml/min per 1.73 M(2) BSA.
- Patients with a total Hgb at entry that is 10.5 g/dl or greater
- Patients with a known allergy to Albumin or cell-derived products
- Patients with uncontrolled hypertension, defined as a systolic blood pressure greater than 170 mmHg and diastolic blood pressure greater than 110 mm Hg that is sustained and unresponsive over 1 week to conventional anti-hypertensive therapy .
Exclusion
Key Trial Info
Start Date :
December 21 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 31 2009
Estimated Enrollment :
7 Patients enrolled
Trial Details
Trial ID
NCT00270478
Start Date
December 21 2005
End Date
August 31 2009
Last Update
December 17 2019
Active Locations (1)
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1
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892