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Systemic Juvenile Idiopathic ArthritisPhase 3β-SPECIFIC 4PaNCT01676948

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

A Phase 3 study testing ACZ885 and Canakinumab in people with Systemic Juvenile Idiopathic Arthritis, run by Novartis Pharmaceuticals.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This two-part open-label, multi-arm, non-comparative study will collect long-term safety, efficacy and tolerability data from patients who were responsive to canakinumab from study CACZ885G2301E1 (Cohort 1), and from patients who are treatment naïve to canakinumab (Cohort 2). Read more about this study

Who is it for?
Ages 2 to 20 years
Conditions the study is for
Systemic Juvenile Idiopathic Arthritis
What is being tested?
ACZ885 and Canakinumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
Jan 2013to Jan 2015
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 20 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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:
  • Male and female patients aged ≥ 2 to < 20 years at the time of the screening visit
  • 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:
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  • Evanescent non-fixed erythematous rash,
  • Generalized lymph node enlargement,
  • Hepatomegaly and/ or splenomegaly,
  • Serositis
  • 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
  • Patient's willingness to discontinue anakinra, rilonacept, tocilizumab or other experimental drug under close monitoring
  • 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

Who cannot join

  • Cohort 1 and Cohort 2:
  • Active or recurrent bacterial, fungal or viral infection at the time of enrollment
  • 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.
  • 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.
  • Live vaccinations within 3 months prior to the start of the study.
  • Cohort 2:
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  • The following additional key exclusion criteria apply for Cohort 2.
  • Presence of moderate to severe impaired renal function
  • Clinical evidence of liver disease or liver injury as indicated by abnormal liver function tests at screening
  • History/evidence of macrophage activation syndrome within the previous 6 months
  • Other protocol-defined inclusion/exclusion criteria may apply.

About this study

This two-part open-label, multi-arm, non-comparative study will collect long-term safety, efficacy and tolerability data from patients who were responsive to canakinumab from study CACZ885G2301E1 (Cohort 1), and from patients who are treatment naïve to canakinumab (Cohort 2). In addition, the effect of inactivated vaccines in an SJIA patient population will be assessed for the development of adequate (protective) antibody levels following immunization according to respective local vaccination guidelines.

Study Part I:

Read more

All patients will be treated with canakinumab 4 mg/kg every 4 weeks (or 2 mg/kg every 4 weeks for Cohort 1 patients who are receiving that dose in CACZ885G2301E1) until study end unless discontinuation occurs, or until they qualify for Part II of the study.

Study Part II:

Patients who are eligible will be randomized to receive canakinumab at a reduced dose or prolonged dose interval (see requirements for dose reduction/dose interval prolongation below).

Patients in Cohort 1 receiving 2 mg/kg q4wk in CACZ885G2301E1 will not be randomized but will be part of the treatment arm canakinumab dose reduction if they are eligible.

Being tested
ACZ885, Canakinumab
Keywords
SJIA, Systemic Juvenile Idiopathic Arthritis, children, systemic autoinflammatory disease, ACZ885, canakinumab, human monoclonal anti-interleukin-1β (IL-1 β) antibody, hildhood immunizations vaccinations
Medical subject headings
Arthritis, Juvenile

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ACZ885Drug

  • CanakinumabDrug

  • CanakinumabDrug

    Patients rolling over into this study from CACZ885G2301E1 (Cohort 1) will continue on their established canakinumab dose of either 4 mg/kg SC given every 4 weeks or 2mg/kg SC given every 4 weeks, until eligible for dose reduction.

    Canakinumab-naïve patients

  • CanakinumabDrug

    Patients rolling over into this study from CACZ885G2301E1(Cohort 2) will receive a standard canakinumab dose of 4mg/kg SC given every 4 weeks, until eligible for dose reduction

Study groups in NCT01676948
Study groupRoleWhat they receive
Canakinumab - Cohort 1, 2mgExperimental2 mg/kg q4wk (followed by taper to 1 mg/kg q4wk and drug discontinuation if appropriate)
Receives: Drug: Canakinumab
Canakinumab - Cohort 1, 4mgExperimental4 mg/kg q8wk (followed by taper to 4 mg/kg q12wk and drug discontinuation if appropriate)
Receives: Drug: Canakinumab
Canakinumab - Cohort 2, 2mgExperimental2 mg/kg q4wk (followed by taper to 1 mg/kg q4wk and drug discontinuation if appropriate)
Receives: Drug: Canakinumab
Canakinumab - Cohort 2, 4mgExperimental4 mg/kg q8wk (followed by taper to 4 mg/kg q12wk and drug discontinuation if appropriate)
Receives: Drug: Canakinumab
Cohort 2 - canakinumab dose reductionExperimentalNot described
Receives: Drug: ACZ885
Cohort 1 - canakinumab dose reductionExperimentalNot described
Receives: Drug: ACZ885

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Long-term safety and tolerability of canakinumab and the retention rate of canakinumab-treated patients

    Outcome Measure Description: The long-term safety and tolerability of canakinumab and the retention rate of canakinumab-treated patients will be evaluated by monitoring of serious adverse events and adverse events leading to discontinuation of study drug.

    Measured: Days 1 to 533

Secondary outcomes (4)

  1. The percentage of patients who meet the adapted pediatric ACR, its individual components, and the Juvenile Arthritis Disease Activity Score [JADAS] over time

    Outcome Measure Description: Patients will be classified into the adapted pediatric ACR categories to characterize their magnitude of efficacy response. JADAS will be derived from physician global assessment, parent/patient global assessment, active joint count and CRP.

    Measured: Days 1 to 533

  2. The level of systemic corticosteroid tapering achieved in Part I

    Patients will be classified into the following 3 categories: ≥ 0.2 mg/kg at end of Part I, those who reach a corticosteroid dose between >0-<0.2 mg/kg and those who reach corticosteroid free regimen.

    Measured: Day 1 to start of Part II

  3. The level of canakinumab tapering achieved after randomization to the dose reduction arm or dose interval prolongation treatment arm in Part II

    In the canakinumab dose reduction arm, patients will be classified into the following 3 categories: Number of patients who are able to reach 1mg/kg q4wk at the end of Part II, canakinumab free regimen by the end of Part II and those who come back to 2mg/kg q4wk at the end of Part II.

    In the canakinumab dose interval arm, patients will be classified into the following 3 categories: Number of patients who are able to reach 4mg/kg q12wk at the end of Part II, canakinumab-free regimen by the end of Part II and those who come back to 4mg/kg q8wk at the end of Part II.

    Measured: from start of Part II to Day 533

  4. The time to treatment failure in Part II

    Time to treatment failure (TTF) is defined as time from randomization to the date when the patient's worsened SJIA disease activity requires increasing the doze or shortening the treatment interval of canakinumab or the date the patient is withdrawn from the study due to a safety concern.

    Measured: from start of Part II to Day 533

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Aug 2012 (estimated)
  2. Study startJan 2013
  3. Primary completionJan 2015
  4. Study completionJan 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01676948
Sponsor's protocol ID
CACZ885G2402
Other IDs
2012-003054-92
Acronym
β-SPECIFIC 4Pa
Status last verified
Oct 2015
First submitted
29 Aug 2012
Last updated
15 Oct 2015
Results posted
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing ACZ885 and Canakinumab in people with Systemic Juvenile Idiopathic Arthritis, run by Novartis Pharmaceuticals.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 20 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 15 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Long-term safety and tolerability of canakinumab and the retention rate of canakinumab-treated patients (time frame: Days 1 to 533). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01676948.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Oct 2015.

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