Clinical Trial: Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using FBS0701 (SSP-004184)
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title: A Phase 2, Open-label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using SSP-004184 (SPD602)
Brief Summary:
The purpose of this extension study is to evaluate SSP-004184AQ in patients with transfusional iron overload and to provide data on long term safety and efficacy.
SSP-004184AQ is an iron chelator under development for chronic daily oral administration to patients with transfusional iron overload
Detailed Summary:
Sponsor: Shire
Current Primary Outcome:
- Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 1) [ Time Frame: Baseline, Week 24 (Cycle 1) ]Efficacy of SSP-004184 was assessed by determining LIC. Abdominal magnetic resonance imaging (MRI) data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
- Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 48 (Cycle 1) [ Time Frame: Baseline, Week 48 (Cycle 1) ]Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
- Change From Baseline in Ferriscan® R2 Liver Iron Concentrations (LIC) at Week 24 (Cycle 2) [ Time Frame: Baseline, Week 24 (Cycle 2) ]Efficacy of SSP-004184 was assessed by determining LIC. Abdominal MRI data were collected by using FerriScan R2 standard procedures and used to determine LIC. A negative change from baseline indicates that LIC decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
- Change From Baseline in Cardiac T2* Values at Week 24 (Cycle 1) [ Time Frame: Baseline, Week 24 (Cycle 1) ]
Original Primary Outcome:
- Change in liver iron concentration as determined by MRI [ Time Frame: up to 5 years ]
- Change in cardiac iron concentration as determined by MRI [ Time Frame: up to 5 years ]
Current Secondary Outcome: Change From Baseline in Serum Ferritin Values at Specified Visits [ Time Frame: Baseline, Weeks 24 and 48 of Cycle 1, Week 24 of Cycle 2 ]
A negative change from baseline indicates that serum ferritin decreased. A cycle consisted of 8 standard 6-weekly visits and the cycles were repeated each year for the duration of the study.
Original Secondary Outcome: Change in serum ferritin levels [ Time Frame: up to 5 years ]
Information By: Shire
Dates:
Date Received: August 16, 2012
Date Started: August 2012
Date Completion:
Last Updated: July 10, 2015
Last Verified: August 2014