Cryopyrin Associated Periodic Syndromes (CAPS) MedDRA version: 12.1 Level: LLT Classification code 10068850 Term: Cryopyrin associated periodic syndrome
Conditions
Interventions
Trade Name: ILARIS
Product Name: Canakinumab
Product Code: ACZ885
Pharmaceutical Form: Powder for solution for injection
INN or Proposed
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female patients that are 28 days up to 60 months of age at the time of the screening visit. 2. Body weight = 2.5 kg. 3. Parent or legal guardian’s written informed consent is required before any assessment is performed for patients. 4. At study entry, patients should have a clinical diagnosis of FCAS, MWS, or NOMID and symptoms requiring pharmacological intervention. Prior agreement between the Investigator and Novartis for study eligibility is required for patients who do not have a molecular diagnosis of NALP3 mutations available (either testing not performed, or testing performed but negative) upon study entry. For those patients who have not been molecularly tested for NALP3 mutations, molecular testing should be performed during the course of the study. 5. For patients treated with an IL-1 blocking agent (i.e. anakinra, rilonacept), these treatments should be discontinued prior to the baseline visit and patients must demonstrate active disease prior to treatment. 6. Patients who are scheduled to receive an immunization, according to their local vaccination guidelines, with an inactivated vaccine must be willing to participate in the assessment schedule for vaccinated patients. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: ?1. Preterm neonates for whom, in the Investigator’s judgment, participation in the study is not deemed appropriate. 2. History of recurrent and/or evidence of active bacterial, fungal, or viral infections (including HIV). 3. Patients with immunodeficiency or treatment with immunosuppressive drugs. 4. Live vaccinations within =3 months prior to screening. No live vaccinations will be allowed throughout the course of this study and up to 3 months following the last dose. ?5. Patients with an increased risk of tuberculosis (TB) infection according to following risk factors: • Patients with recent close contact with persons known to have active pulmonary TB disease • Foreign-born patients from countries with a high prevalence of tuberculosis • Patients with recent tuberculosis infection (including children > 6 months with a positive PPD test [defined as an induration of at least 10mm]) • Patients with end-stage renal disease • Patients with diabetes mellitus • Patients receiving immunosuppressive therapy • Patients with hematologic cancers. 6. Participation in another trial within the last 30 days or 5 half-lives of the investigational compound (whichever is longer). 7. Familial and social conditions rendering regular medical assessment not possible.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of canakinumab with respect to the treatment response in patients 4 years and younger; Secondary Objective: • To assess the efficacy of canakinumab with respect to the treatment response in patients 2 years and younger • Safety and tolerability as assessed by overall frequency of adverse events and number of patients completing the study in patients 2 years and younger and the overall population • To assess the presence of protective antibody levels following immunization with inactivated (killed) vaccines • To evaluate the safety of canakinumab treatment in pediatric patients receiving a concomitant vaccination • The proportion of patients with vaccination-associated reactions • To assess the number of patients who relapse as determined by the Physician’s global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers • To assess the reduction of inflammation markers (C-reactive protein (CRP) and/or serum amyloid A (SAA) after treatment initiation See other objectives into the protocol page 20. ;Primary end point(s): To assess the safety, tolerability and efficacy of canakinumab with respect to the treatment response in CAPS patients 4 years and younger. | — |
Countries
Belgium, France, Germany, Ireland, Spain, United Kingdom
Outcome results
None listed