An Open-label, Multi-arm, Non-comparative Safety and Tolerability Study of Canakinumab (ACZ885) in Patients With Active Systemic Juvenile Idiopathic Arthritis (SJIA)

• Register: ClinicalTrials.gov
• Last refreshed on: 26 October 2015
• Main ID: NCT01676948
• Date of registration: 29/08/2012
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: An Open-label, Multi-arm, Non-comparative Safety and Tolerability Study of Canakinumab (ACZ885) in Patients With Active Systemic Juvenile Idiopathic Arthritis (SJIA) ß-SPECIFIC 4Pa
• Scientific title: An Open-label, Multi-arm, Non-comparative Safety and Tolerability Study of Canakinumab (ACZ885) in Patients With Active Systemic Juvenile Idiopathic Arthritis (SJIA)
• Date of first enrolment: January 2013
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT01676948
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

Argentina, Austria, Belgium, Brazil, Canada, France, Germany, Greece, Hungary, Israel, Italy, Netherlands, Poland, Russian Federation, Spain, Sweden, Switzerland, Turkey, United Kingdom, United States

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion criteria

Key inclusion criteria:

Cohort 1:

1. All patients currently enrolled in study CACZ885G2301E1, including patients who
discontinued canakinumab therapy for inactive disease in CACZ885G2301E1 as per physician
discretion and who are now currently in a flare and require canakinumab therapy again

Cohort 2:

1. Male and female patients aged = 2 to < 20 years at the time of the screening visit

2. Confirmed diagnosis of SJIA as per ILAR definition that must have occurred at least 2
months prior to enrollment with an onset of disease < 16 years of age:

• Arthritis in one or more joints, with or preceded by fever of at least 2 weeks
duration that is documented to be daily/quotidian for at least 3 days and accompanied
by one or more of the following:

- Evanescent non-fixed erythematous rash,

- Generalized lymph node enlargement,

- Hepatomegaly and/ or splenomegaly,

- Serositis

3. Active systemic disease at the time of baseline visit defined as having 2 or more of
the following:

- Documented spiking, intermittent fever (body temperature > 38°C) for at least 1
day during the screening period and within 1 week before first canakinumab dose,

- At least 2 joints with active arthritis (using ACR definition of active joint),

- C-reactive protein (CRP) > 30 mg/L (normal range < 10 mg/L),

- Rash,

- Serositis,

- Lymphadenopathy,

- Hepatosplenomegaly

4. Patient's willingness to discontinue anakinra, rilonacept, tocilizumab or other
experimental drug under close monitoring

5. Patients who are scheduled to receive an immunization, according to their local
vaccination guidelines, with an inactivated vaccine and willing to participate in the
assessment schedule for vaccinated patients

Key exclusion criteria:

Cohort 1 and Cohort 2:

1. Active or recurrent bacterial, fungal or viral infection at the time of enrollment

2. 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 in an immunomodulatory therapy.

3. History of malignancy of any organ system (other than localized basal cell carcinoma
of the skin), treated or untreated, within the past 5 years, regardless of whether
there is evidence of local recurrence or metastases.

4. Live vaccinations within 3 months prior to the start of the study.

Cohort 2:

The following additional key exclusion criteria apply for Cohort 2.

1. Presence of moderate to severe impaired renal function

2. Clinical evidence of liver disease or liver injury as indicated by abnormal liver
function tests at screening

3. History/evidence of macrophage activation syndrome within the previous 6 months

Other protocol-defined inclusion/exclusion criteria may apply.



Age minimum: 2 Years
Age maximum: 20 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Systemic Juvenile Idiopathic Arthritis

Intervention(s)

Drug: ACZ885

Drug: Canakinumab

Primary Outcome(s)

Long-term safety and tolerability of canakinumab and the retention rate of canakinumab-treated patients [Time Frame: Days 1 to 533]

Secondary Outcome(s)

The percentage of patients who meet the adapted pediatric ACR, its individual components, and the Juvenile Arthritis Disease Activity Score [JADAS] over time [Time Frame: Days 1 to 533]

The time to treatment failure in Part II [Time Frame: from start of Part II to Day 533]

The level of canakinumab tapering achieved after randomization to the dose reduction arm or dose interval prolongation treatment arm in Part II [Time Frame: from start of Part II to Day 533]

The level of systemic corticosteroid tapering achieved in Part I [Time Frame: Day 1 to start of Part II]

Secondary ID(s)

2012-003054-92

CACZ885G2402

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

PRINTO/PRCSG