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

Sponsor
Rabin Medical Center
Study ID
NCT02775994
Phase
PHASE1
Status
Unknown

Conditions

  • Periodic Fever

Eligibility Criteria

Sex
ALL
Age
2 Years - 10 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

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).

Key Dates

Start date
Aug 31, 2016
Status verified
Aug 2018
Primary completion
Jan 31, 2020
Completion
Mar 31, 2020

Study Design

Enrollment
10 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment Arm
    Single dose of Canakinumab 4mg/kg

Primary Outcome Measure

Change in average number of flares - documented by the use of diary [ Time Frame: baseline (canakinumab administration) and 2 months after baseline ]

Central Contacts

Related Studies