Clinical Trial: Efficacy and Safety Study of ACZ885 in Patients With Active Recurrent or Chronic TNF-receptor Associated Periodic Syndrome (TRAPS).

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

Official Title: An Open-label, Multicenter, Efficacy and Safety Study of 4-month Canakinumab Treatment With 6-month Follow-up in Patients With Active Recurrent or Chronic TNF-receptor Associated Periodic Syndrome (TR

Brief Summary: This trial will assess the safety and efficacy of ACZ885 in patients with active recurrent or chronic TNF-receptor associated periodic syndrome (TRAPS).

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Percentage of Participants With Complete or Almost Complete Response at Day 15 [ Time Frame: Day 15 ]

Complete response was defined as clinical remission and serological remission. Clinical remission was defined as Physician's Global Assessment of TRAPS activity absent or minimal and serological remission was defined as C reactive protein (CRP) and/or Serum amyloid A protein (SAA) to be less than (<) 10 milligram per liter (mg/L). Almost complete response was defined as clinical remission and a partial serological remission (equal to or more than [≥] 70% reduction of baseline CRP and/or SAA).


Original Primary Outcome: Complete/almost complete response in patients with active TRAPS determined by results of The Physician's Global Assessment, C-reactive protein (CRP) and serum amyloid A (SAA) results [ Time Frame: Day 15 and throughout treatment period ]

Current Secondary Outcome:

  • Percentage of Participants With Complete or Almost Complete Response at Day 8 [ Time Frame: Day 8 ]
    Complete response was defined as clinical remission and serological remission. Clinical remission was defined as Physician's Global Assessment of TRAPS activity absent or minimal and serological remission was defined as CRP and/or SAA < 10 mg/L. Almost complete response was defined as clinical remission and a partial serological remission (≥70% reduction of baseline CRP and/or SAA).
  • Percentage of Participants With Complete Clinical Remission at Day 8 and 15 [ Time Frame: Day 8 and Day 15 ]
    Complete clinical remission was defined as Physician's Global Assessment of TRAPS activity to be absent or minimal (1). TRAPS associated clinical signs and symptoms were assessed by the investigator at every visit on a 5-point scale: 0 = Absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe.
  • Percentage of Participant With Target Levels of C-reactive Protein (CRP) and Serum Amyloid A Protein (SAA) at Day 8 and 15 [ Time Frame: Day 8 and Day 15 ]
    The CRP and SAA were used as inflammatory markers. The target level concentration was ≤ 10 mg/L.
  • Time to Physician's Assessed Clinical Remission [ Time Frame: Baseline up to Day 15 ]
    Time period for complete remission after initial canakinumab treatment as assessed by participants was defined as a Physician's Global Assessment of TRAPS symptoms of scale 1 or less. The physician's Global Assessment was based on a 5-point scale: 0 = None/absent ; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe.
  • Percentage of Participants With Complete or Almost Complete Response at Day 15 After Receiving Additional Dose at Day 8 [ Time Frame: Day 15 ]
    Participants who had not achieved a complete response at Day 8 were given an additional dose of canakinumab. Complete response was defined as clinical remission (Physician's Global Assessment of TRAPS activity absent or minimal) and serological remission (CRP and/or SAA < 10 mg/L). Almost complete response was defined as clinical remission and a partial serological remission (≥ 70% reduction of baseline CRP and/or SAA).
  • Time to Participant's Assessed Clinical Remission [ Time Frame: Baseline up to Day 15 ]
    Time period for complete remission after initial canakinumab treatment as assessed by participants was defined as a participant's Global Assessment of TRAPS symptoms of scale 1 or less. The participant's Global Assessment was based on a 5-point scale: 0 = None/absent (no) ; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe.
  • Percentage Change From Baseline in C-reactive Protein (CRP) and Serum Amyloid A (SAA) Concentration to End of Study [ Time Frame: Day 1 up to Day 953 (End of study) ]
    The CRP and SAA were used as inflammatory markers. The target level concentration was ≤ 10 mg/L. Negative percent change in concentration of inflammatory markers indicated improvement.
  • Percentage of Participants With Defined Grades for Skin Rash, Eye Manifestations, Extremity Pain and Abdominal Pain [ Time Frame: Day 113 (end of treatment period) up to Day 925 (End of study) ]
    TRAPS signs and symptoms were assessed in 4 key categories: skin disease (skin rash), eye manifestations, extremity pain (musculoskeletal), and abdominal pain. Participants were assessed for TRAPS associated signs and symptoms a 5-point Physician's global assessment scale: None/absent (no); 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe.
  • Percentage of Participants With Defined Grades in Physician's Global Assessment Score [ Time Frame: Day 1 up to Day 953 (End of study) ]
    Participants were assessed based by physician on Physician's Global Assessment measured on a 5-point scale for TRAPS associated signs and symptoms as: 0 = None/absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe.
  • Percentage of Participants With Defined Grades in Participant's Global Assessment Score [ Time Frame: Day 1 up to Day 253 (End of follow-up period) ]
    Participants assessed the disease condition based on a 5-point participant's global assessment scale based on TRAPS associated signs and symptoms as: 0 = None/absent; 1 = Minimal; 2 = Mild; 3 = Moderate; 4 = Severe.
  • Percentage of Relapsed Participants [ Time Frame: Day 15, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449,477,505, 533, 561, 589, 617, 645, 673,701, 729,757, 785, 813, 841,869, 897, 925 and 953 ]
    Relapse was defined as a Physician's Global Assessment score of 2 (and an increase of at least 1 point compared to Day 15) and CRP and/or SAA ≥ 30 mg/L representing a 30% increase from Day 15.
  • Time to Relapse After Last Dose of Canakinumab [ Time Frame: Day 85 to Day 253 (End of treatment period to F

    Original Secondary Outcome:

    • Percentage of patients with complete clinical remission as measured by The Physician's Global Assessment score [ Time Frame: at Day 15 for patients who received an additional dose on Day 8 ]
    • percentage of patients with C-reactive protein (CRP) < 10mg/L and serum amyloid A (SAA) < 10mg/L [ Time Frame: at Day 15 for patients who received an additional dose on Day 8. ]
    • complete or almost complete response (defined by the Physician's Global Assessment, C-reactive protein (CRP) [ Time Frame: at Day 15 for patients who received an additional dose on Day 8. ]


    Information By: Novartis

    Dates:
    Date Received: November 16, 2010
    Date Started: October 2010
    Date Completion:
    Last Updated: January 5, 2016
    Last Verified: January 2016