Status:
TERMINATED
Stroke With Transfusions Changing to Hydroxyurea
Lead Sponsor:
St. Jude Children's Research Hospital
Collaborating Sponsors:
National Heart, Lung, and Blood Institute (NHLBI)
Conditions:
Hemochromatosis
Cerebrovascular Accident
Eligibility:
All Genders
5-18 years
Phase:
PHASE3
Brief Summary
The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iro...
Detailed Description
BACKGROUND: Stroke occurs in 10% of children with SCA and has a very high risk of recurrence without therapy. Affected children receive chronic erythrocyte transfusions to prevent a secondary stroke,...
Eligibility Criteria
Inclusion
- Pediatric subjects with severe forms of sickle cell anemia (HbSS, HbSβ0 thalassemia, HbSOArab)
- Age range of 5.0-18.9 years, inclusive, at the time of study entry
- Initial (primary) completed overt clinical stroke after the age of one year (12 months) with documented infarction on brain computed tomography (CT) or magnetic resonance imaging (MRI)
- At least 18 months of chronic monthly erythrocyte transfusions since primary stroke
- Transfusional iron overload, defined as a previously documented liver iron concentration (LIC) greater than or equal to 5.0 mg Fe per gram of dry weight liver or serum ferritin greater than or equal to 500 ng/mL on two independent measurements
- Adequate monthly erythrocyte transfusions with average HbS less than or equal to 45% (the upper limit of the established academic community standard) in the 6 months prior to study entry
- Parent or guardian willing and able to provide informed consent with verbal or written assent from the child (less than 18 years of age) or subject willing and able to provide informed consent (older than 18 years of age)
- Ability to comply with study-related treatments, evaluations, and follow-up
Exclusion
- Inability to receive or tolerate chronic red blood cell (RBC) transfusion therapy, due to any of the following:
- Multiple RBC alloantibodies making cross-matching difficult or impossible
- RBC autoantibodies making cross-matching difficult or impossible
- Religious objection to transfusions that preclude their chronic use
- Non-compliance with transfusions in the 6 months prior to study entry (temporary exclusion)
- Inability to take or tolerate daily oral hydroxyurea, due to any of the following:
- Known allergy to hydroxyurea therapy
- HIV infection
- Cancer
- Pregnant or breastfeeding
- Previous stem cell transplant or other myelosuppressive therapy
- Clinical and laboratory evidence of hypersplenism, due to any of the following:
- Palpable splenomegaly greater than 5 cm below the left costal margin and
- Transfusion requirement greater than 250 mL/kg in the 12 months prior to study entry
- Abnormal laboratory values at initial evaluation (temporary exclusion):
- Pre-transfusion hemoglobin concentration less than 7.0 gm/dL
- White blood cell (WBC) count less than 3.0 x 109/L
- Absolute neutrophil count (ANC) less than 1.5 x 109/L
- Platelet count less than 100 x 109/L
- Serum creatinine more than twice the upper limit for age OR greater than or equal to 1.0 mg/dL
- Current participation in other therapeutic clinical trials
- Current use of other therapeutic agents for SCA (e.g., arginine, decitabine, magnesium)
- Any condition or chronic illness, such as a positive tuberculin (PPD) test, which in the opinion of the study physician makes study participation ill-advised
- Inability or unwillingness to complete required screening studies, including blood tests, brain MRI/magnetic resonance angiography (MRA), and liver biopsy
- A sibling enrolled in SWiTCH
Key Trial Info
Start Date :
October 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2010
Estimated Enrollment :
134 Patients enrolled
Trial Details
Trial ID
NCT00122980
Start Date
October 1 2006
End Date
December 1 2010
Last Update
January 18 2013
Active Locations (28)
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1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
2
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
3
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
4
University of Miami, Jackson Memorial Hospital
Miami, Florida, United States, 33136