Clinical Trial: Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Open-label Study of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients Undergoing Treatment Programs in Comparison With Control Gro

Brief Summary: To evaluate Exjade efficacy and safety in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) , in comparison with a group of patients undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) without chelation therapy.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Change of serum ferritin, iron transferrin saturation, serum total iron-binding capacity (TIBC) [ Time Frame: Screening, 4,8,12,16,20,24,28,32,36,40,44,48,52 weeks ]

Change from baseline will be summarized descriptively for all on-treatment study visits.


Original Primary Outcome: Number of patients treated by Exjade with improving serum ferritin, iron transferrin saturation, serum total iron-binding capacity (TIBC) [ Time Frame: 12 months ]

The primary endpoint is number of patients with Exjade positive effect on Iron metabolism parameters (serum ferritin, iron transferrin saturation, serum total iron-binding capacity) in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment and Exjade


Current Secondary Outcome:

  • Adverse events [ Time Frame: 52 weeks ]
    Number of participants with adverse events as a measure of safety and tolerability
  • Change of proteinuria in urinalysis over a period of 1 year treatment. [ Time Frame: 1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks ]
  • Change from baseline in creatinine clearance over a period of 1 year treatment. [ Time Frame: 1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks ]
  • Change from baseline of serum creatinine, bilirubin, ALT, AST, glucose over a period of 1 year treatment. [ Time Frame: 1,2,3,4,8,12,16,20,24,28,32,36,40,44,48,52 weeks ]


Original Secondary Outcome:

  • Number of patients treated by Exjade with deviations in complete blood cell count [ Time Frame: Baseline, every 1 month up to 12 months ]
    Number of patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment and Exjade with deviations in complete blood cell count
  • Number of patients teated by Exjade with deviations in Glucose tolerance test [ Time Frame: Baseline, 12 months ]
    Number of patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment and Exjade with deviations in Glucose tolerance test
  • Number of patients treated by Exjade with deviations in Blood chemistry panel including bilirubin, ALT, AST [ Time Frame: Baseline, every 1 month up to 12 months ]
    Number of patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment and Exjade with deviations in Blood chemistry panel
  • Number of patients treated by Exjade with deviations in analysis of abdominal ultrasound examination, ECG, echocardiography [ Time Frame: Baseline, 12 months ]
    Number of patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment and treated by Exjade with deviations in analysis of abdominal ultrasound examination, ECG, echocardiography
  • Number of patients treated by Exjade with deviations in Ophthalmological and ENT physician examinations [ Time Frame: Baseline, 12 months ]
    Number of patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment and Exjade with deviations in Ophthalmological and ENT physician examinations + audiogram
  • Number of patients treated by Exjade with level of creatinine upper limits of normal (ULN) [ Time Frame: Baseline, every 1 month up to 12 months ]
    Number of patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) and treated by Exjade with level of creatinine ULN


Information By: Novartis

Dates:
Date Received: March 5, 2013
Date Started: June 23, 2014
Date Completion:
Last Updated: April 20, 2017
Last Verified: April 2017