NCT01186419

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of FBS0701 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy, With a 72 Week Dosing Extension

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 51 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver. FBS0701 is a medication taken by mouth that causes the body to get rid of iron. Iron chelators are used in patients with β-thalassemia and other forms of anemia who experience iron overload - iron increases in the body as a result of regularly required blood transfusions. Patients who qualify will be randomized to receive one of two doses of FBS0701 for up to 24 weeks (6 months) with a total study duration of up to 33 weeks. These patients will be eligible to participate in a dosing extension for up to 72 weeks. The maximum duration of dosing will be up to 96 weeks. The safety of patients will be monitored frequently during the study by physical exams, ECGs, and blood tests. To assess the amount of iron in the liver and heart, each patient must undergo 6 MRI scans during the study. Patients will not need to stay in the hospital for this study but will need to visit the outpatient clinic up to 28 times over the 96 week period. Patients currently taking an iron chelator will be required to stop for a total of up to 26 weeks. The results of this study will help to determine if FBS0701 may be effective as an iron chelator.

Conditions

• Transfusional Iron Overload
• Beta-thalassemia

Interventions

Type	Name	Description
DRUG	SPD602 (FBS0701, SSP-004184)	Oral FBS0701 taken one time daily for up to 96 weeks.

Timeline

• Start date: 2010-08-13
• Primary completion: 2013-01-08
• Completion: 2013-01-08
• First posted: 2010-08-23
• Last updated: 2021-06-10
• Results posted: 2015-06-03

Locations

9 sites across 5 countries: United States, Italy, Thailand, Turkey (Türkiye), United Kingdom