Sickle cell anemia MedDRA version: 20.0 Level: PT Classification code 10040641 Term: Sickle cell anaemia System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: canakinumab
Product Code: ACZ885
Pharmaceutical Form: Solution for injection
INN or Proposed INN: CANAKINUMAB
CAS Number: 914613-48-2
Current Sponsor code: ACZ885
Concentration unit: mg
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male and female subjects ages 8-20 years of age (both inclusive) diagnosed with sickle cell anemia (HbSS) or sickle beta0 thalassemia (documented by family studies, or analysis of either hemoglobin or DNA). - Patient’s written informed consent from those =18 years of age must be obtained before any assessment is performed. Parent or legal guardian’s written informed consent and child’s assent, if appropriate, are required before any assessment is performed for patients =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - History of anaphylactic reaction or known hypersensitivity to canakinumab or any component thereof. - History of, or ongoing treatment with chronic red blood cell transfusion therapy, or have evidence of iron overload requiring chelation therapy. - Transcranial Doppler ultrasound in the past year in patients with an accessible transtemporal window demonstrating velocity in middle or anterior cerebral or internal carotid artery y =200 cm/sec. - Administration of any other blood products within 3 weeks prior screening visit. Other exclusion criteria as per full protocol may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the effect of canakinumab versus placebo on daily pain experienced by sickle cell anemia patients ;Secondary Objective: -To determine the duration of effects of canakinumab versus placebo on daily pain experienced by SCA patients (Reduction of average daily pain VAS over 4-week intervals up to Week 24 as compared to baseline levels) - To determine the effect of canakinumab versus placebo on laboratory markers of inflammation (Week 12 versus baseline of: Serum hsCRP, WBC count, Absolute counts of blood neutrophils, Absolute counts of blood monocytes). - To determine the effect of canakinumab versus placebo on laboratory and functional markers of hemolysis. - To determine the effect of canakinumab versus placebo on SCA-related days missed from school or work -To determine the effect of canakinumab vs placebo on reducing the need for acute blood transfusion - To assess the safety and tolerability of canakinumab in patients with SCA as measured by adverse events (AEs), including immunogenicity as indicated by the presence of anti-drug antibodies - To determine the PK of canakinumab in SCA patients ;Primary end point(s): Reduction of average daily pain VAS over the period of Week 8 to 12 as compared to baseline levels;Timepoint(s) of evaluation of this end point: Week 8 to 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Reduction of average daily pain VAS over 4-week intervals up to Week 24 as compared to baseline levels Week 12 versus baseline of: - Serum hs-CRP - WBC count - Absolute counts of blood neutrophils - Absolute counts of blood monocytes Week 12 versus baseline of: - Hemoglobin concentration - Reticulocyte count - Haptoglobin - LDH - - bilirubin (total, direct, indirect) - Oxygen percent saturation (SaO2) - Number of days absent from school or work due to pain as recorded by daily e-diary - the rate of SCA-related acute transfusion - Adverse events in patients taking ACZ885 compared to placebo up to a total of 56 weeks treatment - Serial serum PK determinations in patients with SCA;Timepoint(s) of evaluation of this end point: 12 weeks | — |
Countries
Canada, Germany, Israel, South Africa, Turkey, United Kingdom, United States
Contacts
Public ContactMedica Information Services
Novartis Pharmaceuticals UK Limited
Outcome results
None listed