Efficacy and Safety Study of Canakinumab Administered for 6 Months (24 Weeks) in Japanese Patients With Cryopyrin-associated Periodic Syndromes Followed by an Extension Phase

• Conditions: Cryopyrin-associated Periodic SyndromesFamilial Cold Autoinflammatory SyndromeMuckle-Wells SyndromeNeonatal Onset Multisystem Inflammatory Disease; MedDRA version: 19.0Level: PTClassification code 10064570Term: Familial cold autoinflammatory syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 19.0Level: LLTClassification code 10064574Term: NOMIDSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 19.0Level: PTClassification code 10068850Term: Cryopyrin associated periodic syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; MedDRA version: 19.0Level: PTClassification code 10064569Term: Muckle-Wells syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders

• Registration Number: EUCTR2015-003490-15-Outside-EU/EEA
• Lead Sponsor: ovartis Pharmaceuticals

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-04-14
• Last Update Posted: 18 April 2016

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1. At study entry, patients should have a clinical diagnosis of FCAS, MWS or NOMID and require medication. At the time of screening, patients can be either untreated or treated with other medication.
2. Presence, or history of at least 2 of the following symptoms:
- For NOMID patients:
?Typical NOMID urticarial rash
?Signs of central nervous system (CNS) involvement such as increased intracranial pressure and/or papilledema and/or cerebral spinal fluid pleiocytosis and/or stroke and/or seizures, and/or sensorineural hearing loss
?Typical arthropatic changes on X-rays: epiphysal and/or patellar overgrowth With start of NOMID symptoms before or at 6 months of age
- For MWS patients:
?periodic fever
?headache/migraine
?arthralgia
?urticarial skin rash
?conjunctivitis
?myalgia
?sensorineural hearing impairment
- For FCAS patients:
?urticarial skin rash
?fever/chills
?conjunctivitis
?joint pain
3. Patients requiring oral steroids, NSAIDs and/or disease-modifying antirheumatic drugs (DMARDs) can be enrolled if they are on a stable dose (oral steroids: < 20 mg/day or < or = 0.4 mg/kg prednisone or prednisone equivalent, whichever applies) for at least 4 weeks prior to the screening visit.
4. Able to communicate with the investigator and comply with the requirements of the study (for children the parent can assist when necessary).

Other protocol-defined inclusion/exclusion criteria may apply
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Pregnant or nursing (lactating) women.
2. All women capable of becoming pregnant unless they are postmenopausal or are using one or more methods of contraception.
3. Participation in any other study within 30 days
4. Infection with HIV, Hepatitis B or C.
5. Live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose.
6. History of drug or alcohol abuse within the 12 months prior to dosing.
7. Donation or loss of 400 mL or more of blood within 8 weeks prior to dosing for adults.
8. History of significant medical conditions, which in the doctor's opinion would exclude the patient from participating in this trial.
9. History of renal transplantation.
10.Presence of any additional rheumatic diseases or significant systemic diseases. For example, major chronic infectious/ inflammatory/ immunologic disease (such as inflammatory bowel disease, psoriatic arthritis, spondyloarthropathy, systemic lupus erythematosus in addition to the autoinflammatory disease).
11. Presence of any of the following laboratory abnormalities: ALT or AST greater than 2 times the upper limit of normal (ULN), platelet count less than 100x109/L.
12. History of recurrent and/or evidence of clinically significant active bacterial, fungal, or viral infections.
13. History of contact with patients with suspected tuberculosis symptoms; or history or complication of tuberculosis infection.
14. Use of the following therapies:
- Etanercept in the 4 weeks prior to the baseline visit (Day 1) and thereafter
- Adalimumab in the 8 weeks prior to the baseline visit (Day 1) and thereafter
- Infliximab in the 12 weeks prior to the baseline visit (Day 1) and thereafter
- Rituximab in the 26 weeks prior to the baseline visit (Day 1) and thereafter
- Tocilizumab in the 3 weeks prior to the baseline visit (Day 1) and thereafter
- Any other investigational biologics in the 8 weeks prior to the baseline visit (Day 1) and thereafter (with the exception of anakinra therapy-see below)
- Anakinra therapy after the baseline visit (Day 1). Last anakinra injection should occur not less than 6 hours prior to the canakinumab injection at Day 1
- Leflunomide in the 4 weeks prior to the baseline visit (Day 1) and thereafter. After the completion of leflunomide treatment a cholestiramine in dose 8 g 3 times per day for 14 days is recommended.
- Thalidomide in the 4 weeks prior to the baseline visit (Day 1) and thereafter
- Cyclosporine in the 4 weeks prior to the baseline visit (Day 1) and thereafter
- i.v. immunoglobulin (i.v. Ig) in the 8 weeks prior to the baseline visit (Day 1) and thereafter
- 6-Mercaptopurine, azathioprine, cyclophosphamide, or chlorambucil in the 12 weeks prior to the baseline visit (Day 1) and thereafter
- Dapsone, mycophenolate mofetil in the 3 weeks prior to the baseline visit (Day 1) and thereafter
- > or = 20 mg/day or >0.4 mg/kg, whichever applies, of prednisone or prednisone equivalent in the 4 week prior to the baseline visit (Day 1) and thereafter
- Methyl prednisone pulse therapy in the 4 weeks prior to baseline visit and thereafter
15. History of allergic reaction to similar drugs. No additional exclusions may be applied by the doctor, in order to ensure that the study population will be representative of all eligible patients.

Other protocol-defined inclusion/exclusion criteria may apply

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified