Stroke With Transfusions Changing to Hydroxyurea

Stopped early · Phase 3

Conditions studied: Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases

In brief

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 iron overload in children with sickle cell anemia (SCA).

Key facts

Study ID

NCT00122980

Run by

St. Jude Children's Research Hospital

People needed

134

Starts

2006-10-01

Expected to finish

2010-12-01

Last updated by the study team

2013-01-18

Who can join

Age: 5 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• Nemours Children's Clinic — Jacksonville, Florida, United States
• University of Miami, Jackson Memorial Hospital — Miami, Florida, United States
• Nemours Children's Clinic — Orlando, Florida, United States
• Children's Healthcare of Atlanta at Egleston — Atlanta, Georgia, United States
• Children's Healthcare of Atlanta at Grady — Atlanta, Georgia, United States
• Children's Healthcare of Atlanta at Scottish Rite — Atlanta, Georgia, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Wayne State University, Children's Hospital of Michigan — Detroit, Michigan, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• The Children's Mercy Hospital — Kansas City, Missouri, United States
• St. Joseph's Children's Hospital — Paterson, New Jersey, United States
• State University of New York/Downstate Medical Center — Brooklyn, New York, United States
• Schneider Children's Hospital — New Hyde Park, New York, United States
• Columbia University Medical Center, Morgan Stanley Children's Hospital of New York-Presbyterian — New York, New York, United States
• Montefiore Medical Center — The Bronx, New York, United States
• East Carolina University — Greenville, North Carolina, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• St. Jude Children's Research Hospital — Memphis, Tennessee, United States
• University of Texas Southwestern Medical Center at Dallas — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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