The following cryopyrin-associated periodic syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease MedDRA version: 9.1 Level: LLT Classification code 10064569 Term: Muckle-Wells syndrome
Conditions
Interventions
Product Name: ACZ885
Product Code: ACZ885
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Canakinumab (WHO appro
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female patients at least 4 years of age at the time of the screening visit. 2. Patient’s informed consent for = 18 years of age before any assessment is performed. 3. Parent or legal guardian’s written informed consent and child’s assent, if appropriate, are required before any assessment is performed for patients =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Pregnant or nursing (lactating) women. 2. Women of child-bearing potential unless they meet the following definition of postmenopausal state: 12 months of natural (spontaneous) amenorrhea or 6 months of spontaneous amenorrhea with serum FSH levels >40 mIU/mL or are using one or more of the acceptable methods of contraception detailed in the protocol are used. 3. Participation in any clinical investigation within 4 weeks prior to dosing or longer if required by local regulation with the exception of trials with anakinra, other investigational IL-1 blocking therapies, and/or ACZ885. 4. History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result. 5. A positive HBsAg or Hepatitis C antibody test result. 6. Live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose. 7. History of drug or alcohol abuse within the 12 months prior to dosing. 8. Donation or loss of 400 mL or more of blood within 8 weeks prior to dosing for adults (For children the cut-off amount of donation or loss of blood should be based on the Investigator's judgment & according to the local regulations. Please also refer to Appendix 4 for the recommended sample volume per blood draw for children). 9. History of significant medical conditions, which in the Investigator’s opinion would exclude the patient from participating in this trial (this can be discussed with Novartis on a case by case basis in case of uncertainty). 10. History of recurrent and/or evidence of active bacterial, fungal, or viral infections. 11. Positive tuberculin skin test reaction (PPD 5 tuberculin units or as according to local standard practice) (= 5 mm induration) at 48 to 72 hours after administration at the screening visit or within 2 months prior to the screening visit, according to national guidelines. Patients who have a positive PPD skin test with a documentation of BCG vaccination, who are at low environmental risk for tuberculosis (TB) infection or reactivation, and have a negative chest X-ray can be included. A positive PPD test has been defined using the [MMWR 2000 guidance], summarized as criteria for tuberculin positivity by risk group: • equal or greater than 15 mm of induration for persons with no risk factors for TB • equal or greater than 10 mm of induration for persons with an increased probability of recent infection or with other clinical conditions that increased the risk for TB • equal or greater than 5 mm of induration for very high risk population (HIV), contact TB cases, immunosuppression (organ transplantation, steroids > 15 mg/day of prednisone for 1 month or more). 12.Precaution against tuberculosis should be handled according to the best medical practice consistent to the local standards in each country with prior consultation with Novartis. Patients requi
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Long-term safety and tolerability of ACZ885 in patients who participated in the A2102, D2201 or D2304 studies or in newly identified patients with the following cryopyrin-associated periodic syndromes (CAPS): Familial Cold Autoinflammatory Syndrome FCAS), Muckle-Wells Syndrome (MWS) or Neonatal Onset Multisystem Inflammatory Disease (NOMID).; Secondary Objective: 1. Maintenance of response over time defined by the number of patients who do not relapse as determined by the Physician’s global assessment of autoinflammatory disease activity, assessment of skin disease and inflammation markers. 2. to Assess number of patients who require a dose adjustment or an administration frequency adjustment, with subsequent maintenance of response over time as determined by the Physician’s global assessment of autoinflammatory disease activity, assessment of skin disease and CRP and/or SAA. 3. Immunogenicity of ACZ885. PK of ACZ885, in particular, comparison of the new HSA- (human serum albumin) drug formulation with previously used HSA+ formulation. 4. Long-term effects of ACZ885 on disease progression with regards to deafness, kidney function, neurological and ophthalmological symptoms. 5. Long term maintenance of Health-Related Quality of Life (HRQoL) of ACZ885 by using the HAQ©, SF-36®, FACIT-Fatigue©, and CHQ-PF28©. ;Primary end point(s): Assess Long term safety and tolerability | — |
Countries
Belgium, France, Germany, Italy, Spain, United Kingdom
Outcome results
None listed