Protocol

SWiTCH: A Phase III Randomized Clinical Trial to Compare Standard Therapy with Alternative Therapy for the Prevention of Secondary Stroke and the Management of Iron Overload in Pediatric Subjects with Sickle Cell Anemia and Previous Stroke

Diseases

Hematologic Disorders : Sickle cell anemia

Description

Children with sickle cell anemia (SCA) who have had a stroke are at high risk of having a recurrent stroke unless therapy is initiated. To prevent a recurrent stroke, affected children receive chronic monthly blood transfusions, which are effective but almost always lead to transfusion-acquired iron overload. Iron overload can cause poor growth and development, and chronic organ damage as iron is deposited in the liver, heart and other organs. If not treated, iron overload can lead to death. Children who receive chronic blood transfusions receive chelation therapy to prevent iron build up, however, this therapy is difficulty for many to tolerate, resulting in non-compliance with the medication. 

This clinical trial will compare two different treatments for the prevention of a recurrent stroke, and treatment of iron overload in children with sickle cell anemia. The standard treatment, monthly blood transfusions for stroke prevention and iron chelation for the prevention of iron overload, will be compared with the research treatment of a daily oral medication called hydroxyurea to prevent recurrent stroke, and phlebotomy, or the removal of blood, to treat iron overload.  

Hydroxyurea, the research treatment in this trial, is approved by the Food and Drug Administration for use in adults with sickle cell anemia. Hydroxyurea has not been approved for use in children with sickle cell anemia and stroke. Hydroxyurea can be taken by mouth and has been used to prevent painful episodes in children and adults with sickle cell anemia. Phlebotomy is a way to lower iron in the body by removing blood through an IV (intravenous) catheter. Participants in this study will be randomly assigned (like flipping a coin) to either the standard treatment or research treatment.


Objectives

  • To compare alternative therapy (hydroxyurea treatment with repeated phlebotomy) with standard therapy (monthly erythrocyte transfusions with daily iron chelation) for the prevention of secondary stroke and reduction of transfusional iron overload in pediatric subjects with SCA and previous stroke.
  • To compare alternative therapy to standard therapy for effects on quality of life, the frequency of non-stroke neurological events and other sickle cell-related events, and growth and development.

Eligibility

  • Children with severe forms of sickle cell anemia between the age of 5 and 18.9 years (inclusive)
  • Initial (primary) completed overt clinical stroke after the age of one year (12 months) with documented infarction on brain computed tomography (CT) or MRI
  • At least 18 months of chronic monthly erythrocyte transfusions since the primary stroke
  • Transfusional iron overload, defined as a previously documented liver iron concentration (LIC) ≥ 5.0 mg Fe per gram of dry weight liver or serum ferritin ≥ 500 ng/mL on 2 independent measurements
  • Adequate monthly erythrocyte transfusions with average HbS ≤ 45% (the upper limit of the established academic community standard) for the past 6 months before enrollment
  • Parent or guardian willing and able to provide informed consent with verbal or written assent from the child (< 18 years of age) and subject willing and able to provide informed consent (> 18 years of age)
  • Ability to comply with study-related treatments, evaluations, and follow-up

For the current eligibility status of this clinical study, referring physicians must contact St. Jude Children's Research Hospital at 1-866-2ST-JUDE  (1-866-278-5833).


Contact

Russell E. Ware, MD, PhD
St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN  38105  USA
Voice:   1-866-2ST-JUDE  (1-866-278-5833)
FAX:     901-595-5068

Referring or consulting physicians only: protocolinfo@stjude.org
For all other inquiries about St. Jude Children's Research Hospital studies: info@stjude.org

Amendment 4.0 dated 1.07.08, IRB approved 3.20.08


The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

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