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