Clinical Trial: Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: A Multiple-dose, Subject- and Investigator-blinded, Placebo-controlled, Parallel Design Study to Assess the Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patie

Brief Summary: To determine the effect of canakinumab versus placebo on daily pain experienced by sickle cell anemia patients (Reduction of average daily pain VAS over the period of Week 8 to 12 as compared to baseline levels).

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Reduction of average daily pain [ Time Frame: Week 8 to 12 ]

Primary endpoint is the reduction of average daily pain measured with a visual analog scale over the period of Week 8 to 12 as compared to baseline levels


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Reduction of average daily pain over 24 weeks [ Time Frame: Baseline, 24 weeks ]
    Reduction of average daily pain measured with a visual analog scale over 4-week intervals up to Week 24 as compared to baseline levels
  • The effect of canakinumab versus placebo on sickle cell anemia related days missed from school or work [ Time Frame: 56 weeks ]
    The number of days absent from school or work due to pain as recorded daily in an e-diary
  • Measurement of pharmacokinetics of canakinumab in sickle cell anemia patients, which will be presented as descriptive summary statistics of pharmacokinetic concentrations. [ Time Frame: 48 weeks ]
    serial serum PK determination, 9 samples over 48 weeks, in patients with sickle cell anemia
  • Week 12 versus baseline of white blood cell count [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory markers of inflammation
  • Week 12 versus baseline of absolute counts of blood neutrophils [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory markers of inflammation
  • Week 12 versus baseline of absolute counts of blood monocytes [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory markers of inflammation
  • Week 12 versus baseline of serum hs-CRP [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory markers of inflammation
  • Week 12 versus baseline of Hemoglobin concentration [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis
  • Week 12 versus baseline of reticulocyte count [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis
  • Week 12 versus baseline of Haptoglobin [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis
  • Week 12 versus baseline of LDH [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis
  • Week 12 versus baseline of total bilirubin [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis
  • Week 12 versus baseline of oxygen percent saturation (SAO2) [ Time Frame: Baseline, 12 weeks ]
    The effect of canakinumab versus placebo measured with laboratory and functional markers of hemolysis


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: October 20, 2016
Date Started: December 2016
Date Completion: July 2018
Last Updated: November 8, 2016
Last Verified: November 2016