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ß-SPECIFIC 4 Patients: Study of Pediatric EffiCacy and Safety wIth FIrst-line Use of Canakinumab (ß-SPECIFIC 4)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02296424
Recruitment Status : Completed
First Posted : November 20, 2014
Results First Posted : July 9, 2019
Last Update Posted : July 9, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Tracking Information
First Submitted Date  ICMJE October 10, 2014
First Posted Date  ICMJE November 20, 2014
Results First Submitted Date  ICMJE September 24, 2018
Results First Posted Date  ICMJE July 9, 2019
Last Update Posted Date July 9, 2019
Actual Study Start Date  ICMJE November 17, 2014
Actual Primary Completion Date October 14, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 17, 2019)
Number of Participants in Clinical Remission on Canakinumab Who Are Able to Remain at an Initial Reduced Canakinumab Dose or Prolonged Canakinumab Dose Interval. [ Time Frame: baseline to 24 weeks ]
The primary efficacy variable for Part II was the proportion of patients in clinical remission on canakinumab 4 mg/kg (+/- concomitant NSAID only) who were able to remain on a reduced dose or on prolonged dose interval for at least 24 consecutive weeks. As the primary objective was to show statistically significance in at least one of canakinumab treatment arms (reduced dose and prolonged dose interval arms) in Part II then the Type I error rate 5% was controlled and split to 2.5%. Clinical remission per protocol is defined as the maintenance of inactive disease for at least 6 months (24consecutive weeks) while on therapy. The primary analysis considered both inactive disease status and the patient dose step duration. In the event the inactive disease status was missing, yet the patient remained at the same dose level through the next visit with the same disease status, it was concluded that inactive disease was maintained during this time period and was carried forward.
Original Primary Outcome Measures  ICMJE
 (submitted: November 17, 2014)
Proportion of patients in clinical remission on canakinumab who are able to remain at an initial reduced canakinumab dose or prolonged canakinumab dose interval. [ Time Frame: During study part II, defined as completion of 24-weeks of randomized treatment. ]
To evaluate if patients in clinical remission who have successfully discontinued any concomitant corticosteroid and/or methotrexate can maintain inactive disease for 24 weeks (clinical remission) at a reduced canakinumab dose of 2mg/kg (150mg max) every 4 weeks or 4mg/kg (300mg max) every 8 weeks.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 17, 2019)
  • Number and Percentage of Patients With Adverse Events as a Measure of Long-term Safety and Tolerability of Canakinumab - PART 1 [ Time Frame: During study parts I and II. The estimated study duration is not more than 216 weeks (with an average expected duration of 108 weeks). ]
    AEs, Deaths, other serious adverse events or discontinuations due to AE, Part I (Safety set)
  • Number and Percentage of Patients With Adverse Events as a Measure of Long-term Safety and Tolerability of Canakinumab - PART 2 [ Time Frame: During study parts I and II, estimated study duration was not more than 216 weeks (with an average duration of 108 weeks). ]
    AEs, Deaths, other serious adverse events or discontinuations due to AE, Part II (Safety set)
Original Secondary Outcome Measures  ICMJE
 (submitted: November 17, 2014)
Number and percentage of patients with adverse events as a measure of long-term safety and tolerability of canakinumab (Parts I and II). [ Time Frame: During study parts I and II. The estimated study duration is not more than 216 weeks (with an average expected duration of 108 weeks). ]
To assess the long-term safety and tolerability of canakinumab by overall frequency of adverse events.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE ß-SPECIFIC 4 Patients: Study of Pediatric EffiCacy and Safety wIth FIrst-line Use of Canakinumab
Official Title  ICMJE β-SPECIFIC 4 Patients: Study of Pediatric EffiCacy and Safety wIth FIrst-line Use of Canakinumab An Open-label Canakinumab (ACZ885) Dose Reduction or Dose Interval Prolongation Efficacy and Safety Study in Patients With Systemic Juvenile Idiopathic Arthritis (SJIA)
Brief Summary The purpose of this study was to evaluate the efficacy observed with canakinumab dose reduction in a subgroup of patients in the extension study CACZ885G2301E1.
Detailed Description This two-part open-label study was to assess 2 different canakinumab taper regimens in patients with clinical remission (inactive disease for at least 24 continuous weeks) on canakinumab treatment without concomitant corticosteroids (CS) or methotrexate (MTX). The study was also to collect long term safety and tolerability data on SJIA patients treated with canakinumab.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
A two-part open-label study that collected long-term efficacy, safety, and tolerability data from SJIA patients receiving canakinumab treatment who had inactive disease at the last visit in Study CACZ885G2301E1 (Cohort 1), and from SJIA patients who were canakinumab treatment-naïve and had active disease at the time of screening for this study (Cohort 2)
Masking: None (Open Label)
Masking Description:
No blinding was required in this open-label study
Primary Purpose: Treatment
Condition  ICMJE Systemic Juvenile Idiopathic Arthritis (SJIA)
Intervention  ICMJE Drug: ACZ885 150 mg (Canakinumab)
Active canakinumab in individual 2 mL glass vials, each containing 150 mg canakinumab liquid in vial.
Other Name: ACZ885 150 mg
Study Arms  ICMJE
  • Experimental: Canakinumab Dose Reduction
    All patients received canakinumab 4mg/kg (300 mg max) every 4 weeks in Part I of the study. Patients eligible for Part II of the study were randomized to one of two treatment arms. This is Treatment Arm 1 in Part II of the study: Canakinumab was administered at a reduced dose (2 mg/kg every 4 weeks). If the patient continued to maintain inactive disease for 24 additional weeks, canakinumab was administered at 1mg/kg every 4 weeks. If the patient continued to maintain inactive disease for another 24 additional weeks, canakinumab treatment was discontinued.
    Intervention: Drug: ACZ885 150 mg (Canakinumab)
  • Experimental: Canakinumab Dose Interval Prolongation
    All participants received canakinumab 4mg/kg (300 mg max) every 4 weeks in Part I of the study. Patients eligible for Part II of the study were randomized to one of two treatment arms. This is Treatment Arm 2 in Part II of the study: Canakinumab dose interval was prolonged to a regimen of 4mg/kg every 8 weeks. If the patient continued to be stable with inactive disease for 24 additional weeks, canakinumab dose interval was prolonged to a regimen of 4mg/kg every 12 weeks. If the patient was clinically stable with inactive disease for another 24 additional weeks, canakinumab treatment was discontinued.
    Intervention: Drug: ACZ885 150 mg (Canakinumab)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 21, 2018)
182
Original Estimated Enrollment  ICMJE
 (submitted: November 17, 2014)
180
Actual Study Completion Date  ICMJE September 25, 2017
Actual Primary Completion Date October 14, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

Cohort 1:

• Patients who are receiving canakinumab treatment (4 mg/kg every 4 weeks) for Systemic Juvenile Idiopathic Arthritis (SJIA) and have inactive disease at the last visit in Study CACZ885G2301E1

Cohort 2:

  • Confirmed diagnosis of SJIA as per International League Against Rheumatism (ILAR) definition that must have occurred at least 2 months prior to enrollment with an onset of disease < 16 years of age.
  • Active SJIA defined as having 2 or more of the following:
  • Documented spiking, intermittent fever (body temperature > 38°C) for at least 1 day within 1 week before first canakinumab dose;
  • At least 2 joints with active arthritis
  • C-reactive protein (CRP) > 30 mg/L (normal range < 10 mg/L)
  • Rash due to SJIA
  • Serositis
  • Lymphadenopathy
  • Hepatosplenomegaly
  • Negative TB screen (QuantiFERON or, if required by local guidelines, Purified Protein Derivative).

Exclusion Criteria:

  • With active or recurrent bacterial, fungal or viral infection at the time of enrollment, including patients with evidence of Human Immunodeficiency Virus (HIV) infection, Hepatitis B and Hepatitis C infection.
  • With underlying metabolic, renal, hepatic, infectious or gastrointestinal conditions which in the opinion of the investigator immunocompromises the patient and /or places the patient at unacceptable risk for participation.
  • With neutropenia (absolute neutrophil count < 1500/mm3) at screening.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 20 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Belgium,   Brazil,   Canada,   France,   Germany,   Hungary,   Israel,   Italy,   Netherlands,   Poland,   Russian Federation,   Spain,   Sweden,   Turkey,   United States
Removed Location Countries Argentina,   Hong Kong,   Mexico
 
Administrative Information
NCT Number  ICMJE NCT02296424
Other Study ID Numbers  ICMJE CACZ885G2306
2013-004867-29 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Responsible Party Novartis ( Novartis Pharmaceuticals )
Study Sponsor  ICMJE Novartis Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
PRS Account Novartis
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP