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 ]
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