novartis

In this study, non-invasive R2- and T2*-MRI techniques will be applied to the liver and the heart, respectively, to complement the primary variable (serum ferritin) assessed in patients with various transfusion-dependent anaemias. The main objective of this study is to assess the prevalence and severity of cardiac and liver siderosis in patients with transfusional siderosis. This study will also aim to establish possible correlations between cardiac and liver iron levels with clinical effects in patients with different transfusion-dependent anaemias. Patients will be eligible for enrollment irrespective of receiving chelation therapy or not (and irrespective of the chelating agent used).

 

More information: clinicaltrials.gov

CaridianBCT

The primary endpoint will evaluate the mean ratio of the Actual Fraction of Cells Remaining (FCRa; as measured by Post-Procedure % HbS) to the Predicted Fraction of Cells Remaining (FCRp; as predicted by the Spectra Optia system FCR algorithm), in the Evaluable subject population. The trial started in November 2012 and the estimated completion date is May 2013.

 

More information: clinicaltrials.gov

children hospital philadelphia

The combination of deferoxamine and deferiprone has been shown to be particularly beneficial for reducing cardiac iron, but it requires a painful injection/infusion, which hinders adherence. This pilot study aims to investigate the safety of an oral-only combination chelator regimen (deferasirox and deferiprone) in individuals with thalassemia major with poorly controlled iron overload and to assess how well this chelator combination lowers iron stores over one year.

 

This study is sponsored by the Children's Hospital of Philadelphia in collaboration with the Cooley’s Anemia Foundation and the Toronto General Hospital.

 

More information: clinicaltrials.gov

novartis

This study, sponsored by Novartis Pharmaceuticals, aims to assess the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 24 and 52 weeks of treatment. Also, to evaluate the impact of deferasirox on the Quality of Life for adults and pediatric patients; correlate changes in serum ferritin and LIC, evaluate efficacy changes according to genotype, evaluate higher doses of deferasirox, assess endocrine function and examine pk parameters.

 

More information: clinicaltrials.gov

bluebird bio

Gene therapy firm Bluebird Bio awarded $9.3 million from the California Institute for Regenerative Medicine (CIRM) to support a Phase I/II study evaluating bluebird’s LentiGlobin® treatment for β-thalassemia. The trial is scheduled to start in the U.S. next year. The CIRM grant is one of the first to have been made through the Institute’s new Strategic Partnership Awards initiative.

 

Read the News Release for more information