Clinical Trial: Canakinumab in Patients With Active Hyper-IgD Syndrome
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Open-label, Multicenter, Efficacy and Safety Pilot Study of 6-month Canakinumab Treatment With up to 6-month Follow-up in Patients With Active Hyper-IgD Syndrome (HIDS)
Brief Summary: This pilot study is designed to evaluate the efficacy, the safety, and the pharmacokinetics (PK) / pharmacodynamics (PD) of canakinumab treatment in patients with HIDS.
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: Number of Flares Per Participant During Historical Period and Treatment Period [ Time Frame: Historical period, Month 6 (End of treatment period) ]
Original Primary Outcome: The primary efficacy variable is the number of flares observed during the 6-month treatment period. [ Time Frame: 1 year ]
Current Secondary Outcome:
- Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period [ Time Frame: Month 6 (End of treatment period), Month 36 (End of Long term treatment Period 2) ]A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value > 10 mg/L.
- Number of Participants Who Flared at Month 6, Month 24 and Month 36 [ Time Frame: Baseline, Month 6 (End of treatment period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value > 10 mg/L.
- Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score [ Time Frame: Any flare event [Baseline up to Month 36 (End of long term treatment period 2)] ]Physician global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5- point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe.
- Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]Participant's global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5-point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe. Same investigator assessed the same participant throughout the study to ensure consistency between assessments. Investigators reviewed every participant's diary at each visit after their own clinical assessment.
- Percentage of Participants With Defined Grades of Participants Assessed Symptom Control [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]Participants were assessed by participants/parent (participants aged 6-18 years) for control of signs and symptoms associated with HIDS based on 5-point scale: 0 = No control; 1 = Poor control; 2 = Somewhat control; 3 = Good control; and 4= Excellent control.
- Percentage of Participants With Defined Grades of Physician Assessed Symptom Control [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]Participants were assessed by physician for control of signs and symptoms associated with HIDS based on 5-point scale: 0 = No control; 1 = Poor control; 2 = Somewhat control; 3 = Good control; and 4= Excellent control.
- Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]Fever severity was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
- Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]Apthus ulcers were assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
- Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]Lymphadenopathy severity was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
- Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment [ Time Frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2) ]Abdominal pain was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
- Time to Resolution of the Initial Flare After First Canakinumab Treatment [ Time Frame: Day 1 (Baseline), Day 28 ]
Original Secondary Outcome:
- Number of patients who flare during treatment period [ Time Frame: 1 year ]
- Flare severity during the treatment period for patients who have at least 1 flare during the treatment period as assessed by the physician's global assessment of HIDS flare severity score. [ Time Frame: 1 year ]
- Flare severity during the treatment period for patients who have at least 1 flare during the treatment period as assessed by patient's global assessment of HIDS flare severity score. [ Time Frame: 1 year ]
- HIDS signs and symptoms severity during the treatment period for patients who have at least 1 flare during the treatment period as assessed by physician assessment of 4 key signs and symptoms severity score. [ Time Frame: 1 year ]
- HIDS signs and symptoms severity during the treatment period for patients who have at least 1 flare during the treatment period as assessed by patient assessment of 4 key signs and symptoms severity score. [ Time Frame: 1 year ]
Information By: Novartis
Dates:
Date Received: February 23, 2011
Date Started: March 2011
Date Completion:
Last Updated: October 13, 2015
Last Verified: October 2015