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Past Thalassemia Studies

The following studies have closed and are no longer enrolling patients. Information about the studies and their results, however, are provided in order to give thalassemia patients the most up to date information available on research outcomes.

TCRN Hepatitis C Study
This study was started in 2002 and enrolled 21 patients around the country (13 patients completed the study). Patients with Hepatitis C were treated with Ribavirin and Peg-Interferon for 6 to 12 months. We are no longer enrolling patients in this study. The first paper reporting the results of this study was published in Summer 2007 by Paul Harmatz, MD. If you would like further information about the results, please contact Nancy Sweeters at (510) 428-3885 x 4151.

Exjade (also known as ICL670) Study Update
Exjade (also known as deferasirox or ICL670) is an oral chelator produced by Novartis that was FDA approved in November 2005. In order to acquire Exjade, which is usually covered by medical insurance, one must complete an application called EPASS. For further information on the EPASS system, please talk with your provider.

Those taking Exjade should have monthly monitoring of his or her blood and urine. People with kidney disease, or, who are taking other medicines that can affect the kidneys must use caution when taking Exjade.

The FDA requested that additional data be collected from people taking Exjade for up to five years. The thalassemia team at Children's Hospital & Research Center at Oakland continues to offer participation in a clinical trial for people who have been taking Exjade since 2003 and will continue until the summer of 2008. We are no longer enrolling patients in this study.

Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease. Published in British Journal of Haematology, October 2006
Blood transfusions are a main part of therapy for patients with beta-thalassemia and sickle cell disease, but it often results in severe iron overload. This study examined if body stress (measured by inflammation and tissue injury) are different between transfused patients with thalassemia and sickle cell disease. During the analysis of the data, it was found that patients with sickle cell disease have more inflammation and increased levels of protective antioxidants compared to patients with thalassemia.

Reduced Inflammation and Oxidant-Stress in ß-Thalassemia Patients Treated with Iron Chelators Deferasirox (ICL670) or Deferoxamine: An Ancillary Study of the Novartis CICL670A0107 Trial
This study was done to see if the researchers could find a difference in the level of body stress (measured by inflammation and oxidative stress) in patients taking Exjade versus Desferal. This study was done as an extra part of the large, multicenter Exjade and Desferal study by Novartis. There were 49 patients enrolled in this study from North America and England. Blood samples were tested at different times during the study. At the conclusion of the study it was found that liver iron and ferritin decreased in both groups but the patient that took Exjade had better decrease in inflammation. Please see the article if you would like more details about this study.

Phase Ib Clinical Trial of Starch-Conjugated Deferoxamine (40SD02): A Novel Long-acting Iron Chelator. Accepted for publication in British Journal of Haematology

Most patients with thalassemia use Desferal (DFO mesylate) as their iron chelator. Desferal is given over many hours and therefore over time patients can have decreased compliance with taking Desferal. A trail was done using a new form of Desferal called Starch Desferol. Starch Desferal (S-DFO) is given IV over one hour and works chelating iron for one week because it is attached to a starch and is slowly released over time. In this study patients were given different doses to help find out what was the most effective dose of starch Desferal. Patients were asked to do various tests during the week after the infusion of S-DFO. Some patients did report hives during the infusion but none required stopping of the infusion. It was found that S-DFO given weekly has the potential to be an effective chelator.

Multi-Center Study of Iron Overload--The “MCSIO” Study
The purpose of this study is to determine if organ dysfunction (problems with the heart, liver, and reproductive organs) differs in transfused patients with sickle cell disease compared to those with thalassemia. Clinical observations suggest that organ dysfunction resulting from iron overload in patients with thalassemia is not present in patients with sickle cell disease. It is hypothesized that the mechanisms and consequences of iron deposition differ between these two diseases. To examine this hypothesis a prospective, multi-center, natural history study was designed to assess the incidence and rate of development of organ dysfunction between the two patient groups.

Recruitment for this study has closed. A total of 152 patients with thalassemia, 204 patients with sickle cell disease, and 67 non-transfused sickle cell patients were recruited and are currently being followed. Investigators hope to apply for and receive additional funding to continue the study for an additional 5 years. For more information, please contact Lynne Neumayr, MD, CHRCO, at or 510-428-3698.

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