TNF-receptor associated periodic syndrome (TRAPS) MedDRA version: 14.1 Level: LLT Classification code 10067783 Term: Tumor necrosis factor receptor-associated periodic syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patient’s written informed consent for = 18 years of age before any assessment is performed. Parent or legal guardian’s written informed consent and child’s assent, if appropriate, are required before any assessment is performed for patients 10mg/L (Normal CRP range = 10 mg/L) and/or SAA > 10 mg/L (Normal SAA range = 10 mg/L) at time of first canakinumab treatment. Are the trial subjects under 18? yes Number of subjects for this age range: 6 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 13 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: 1. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test. 2. Women of child-bearing potential, defined as pre-menarche females aged 8 years and above or all women physiologically capable of becoming pregnant, UNLESS they are • women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner • women whose partners have been sterilized by vasectomy or other means • using a highly effective method of birth control (i.e. one that results in a less than 1% per year failure rate when used consistently and correctly, such as implants, injectables, combined oral contraceptives, and some intrauterine devices [IUDs]; periodic abstinence [e.g. calendar, ovulation, symptothermal, post-ovulation methods] is not acceptable) or total abstinence at the discretion of the investigator in cases where the age, career, lifestyle, or sexual orientation of the patient ensures compliance. • Women of child-bearing potential should be willing to use a reliable contraception throughout the study and for 3 months after study drug discontinuation. • Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or six months of spontaneous amenorrhea with serum FSH levels > 40 mIU/mL or have had surgical bilateral oophorectomy (with or without hysterectomy) at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential. 3. History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result. 4. Positive QuantiFERON (QFT-TB G In-Tube) test or positive Purified Protein Derivative (PPD) test (= 5 mm induration) at screening or within 2 month prior to the screening visit, according to the national guidelines. Patients with a positive PPD test (= 5 mm induration) at screening may be enrolled only if they have either a negative chest x-ray or a negative QuantiFERON test. 5. Live vaccinations within 3 months prior to the start of the trial, during the trial, and up months following the last dose. 6. History of significant other medical conditions, which in the Investigator’s opinion would exclude the patient from participating in this trial. 7. History of recurrent and/or evidence of active bacterial, fungal, or viral infection(s). 8. Use of the following therapies: a. Corticosteroids (oral prednisone (or equivalent)) > 0.2 mg/kg/day (or greater than the maximum of 15 mg/day for children over 60 kg) within 1 week prior to the Baseline visit b. Anakinra within 24 hours prior to Baseline visit c. Canakinumab within 3 months prior to Baseline visit d. Rilonacept within 1 week prior to Baseline visit e. Tocilizumab within 3 weeks prior to Baseline visit f. Etanercept within 4 weeks prior to Baseline visit g. Rituximab within 26 weeks prior to the Baseline visit h. Leflunomide within 4 weeks prior to the Baseline visit. Documentation of a completion of a full cholestyramine elimination treatment after most recent leflunomide use will be required. i. Thalidomide within 4 weeks prior to the Baseline visit j. Cyclosporine within 4 weeks prior t
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess if canakinumab induces complete or almost complete response in patients with active TRAPS at Day 15 (defined as 15 days after the first dose).;Secondary Objective: 1. To assess if canakinumab can induce complete or almost complete response in patients with active TRAPS at Day 8. 2. To assess the percentage of patients with complete clinical remission (Physician’s Global Assessment score = 1) at Day 8 and Day 15. 3. To assess the percentage of patients with C-reactive protein (CRP) < 10mg/L and serum amyloid A (SAA) < 10mg/L at Day 8 and Day 15. 4. To assess if canakinumab can induce complete or almost complete response at Day 15 for patients who received an additional dose on Day 8. 5. To assess time to patient’s assessed clinical remission (Patient’s Global Assessment score =1) after initial canakinumab treatment. 6. To assess time to physician’s assessed clinical remission (Physician’s Global Assessment score = 1) after initial canakinumab treatment. 7. To assess the profile over time in CRP and SAA from baseline to end of study.;Primary end point(s): To assess if canakinumab induces complete or almost complete response active TRAPS at Day 15 (defined as 15 days after the first dose).;Timepoint(s) of evaluation of this end point: At Day 15 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Percentage of patients with complete clinical remission as measured by The Physician's Global Assessment score Percentage of patients with C-reactive protein (CRP) < 10mg/L and serum amyloid A (SAA) < 10mg/L Complete or almost complete response (defined by the Physician's Global Assessment, C-reactive protein [CRP]);Timepoint(s) of evaluation of this end point: Day 8 & Day 15/ 4-month and long-term treatment period | — |
Countries
Ireland, Italy, United Kingdom
Contacts
Public ContactChief Scientific Officer
Novartis Ireland
Outcome results
None listed