Ilaris (Canakinumab) in Patient With Periodic Fever, Aphthous Stomatitis, Pharyngitis and Cervical Adenitis (PFAPA)

• Status: Unknown status
• Trial end date: 2020-03-01
• Target enrollment: 0
• Participant gender: All

Summary

Periodic Fever, Aphthous stomatitis, Pharyngitis and cervical Adenitis (PFAPA) is one of the most common, least explored periodic fever syndrome in childhood. This study aims to investigate whether a single dose of an interleukin beta (IL-1) antagonist, Canakinumab will be able to abort PFAPA flares in patients who experience a flare in an average of 2 weeks or less. This will be a single arm open label pilot study. 10 patients will be recruited from 1 center (Pediatric rheumatology unit -Schneider children's medical center of Israel).

Patients in ages 2-10 years old who are diagnosed with PFAPA according to clinical criteria at least 3 months prior to enrollment and who are under regular care for this disease (single dose of glucocorticoids during flare) and who suffer from more than 4 PFAPA flares for the last 2 months, will be screened for this study. In the second documented flare, patients will be enrolled to receive a single dose of subcutaneous (SC) Canakinumab 4 mg/kg. The primary outcome is defined as - 50% reduction in PFAPA flares for the next 2 consecutive months as reported by the patient (use of diary) and documented by the patient primary care physician and/ or the researcher in a monthly follow up visits. Secondary outcome measure are define as time to flare (days) and Parent/patient quality of life assessment measured by 100mm visual analog scale (VAS).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Rabin Medical Center
• Treatments: Antibodies, Monoclonal

Criteria

Key inclusion criteria

1. Patients in ages 2-10 years old who are diagnosed with PFAPA at least 3 months prior to enrollment according to clinical criteria will be screened for this study.

2. Suffer from more than 4 PFAPA flares 2 months prior to the screening period.

3. Have signed informed consent for the study.

4. Have at least 2 documented PFAPA flare during the screening period.

Key exclusion criteria

1. Patients who are diagnosed with chronic disease including another auto inflammatory disease.

2. Patients who suffer from neutropenia or elevated liver enzymes.

3. Patients who receive treatment with cimetidine/famotidine, montelukast or colchicine 2 weeks or less prior to enrollment.