Ilaris (Canakinumab) in Patient With Periodic Fever, Aphthous Stomatitis, Pharyngitis and Cervical Adenitis (PFAPA)
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|ClinicalTrials.gov Identifier: NCT02775994|
Recruitment Status : Unknown
Verified August 2018 by Rabin Medical Center.
Recruitment status was: Recruiting
First Posted : May 18, 2016
Last Update Posted : September 4, 2018
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).
|Condition or disease||Intervention/treatment||Phase|
|Periodic Fever||Drug: Canakinumab||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Ilaris (Canakinumab) in Patient With Periodic Fever, Aphthous Stomatitis, Pharyngitis and Cervical Adenitis (PFAPA)|
|Actual Study Start Date :||August 2016|
|Estimated Primary Completion Date :||January 2020|
|Estimated Study Completion Date :||March 2020|
Experimental: Treatment Arm
Single dose of Canakinumab 4mg/kg
interleukin beta (IL-1) antagonist
Other Name: Ilaris
- Change in average number of flares - documented by the use of diary [ Time Frame: baseline (canakinumab administration) and 2 months after baseline ]Efficacy measure - 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. This will be compared to the average number of flares (per 2 months) during the last 3 consecutive months before therapy (i.e number of flares in last 3 months before therapy divided by 3 and multiplied by 2).
- Time to flare - documented by the use of diary [ Time Frame: baseline (canakinumab administration) and up to 24 weeks after baseline ]Time to flare - period between administration of a single dose of canakinumab until the first documented PFAPA flare 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. Will be compared to the average time between 2 consecutive flares before therapy (e.g: first documented flare- will be day 1. In the following 90 days 5 more flares (3 before screening and 2 after) = 90/5= 18d mean time to flare).
- Change in quality of life - documented by the use of questionnaire [ Time Frame: at the time of screening visit and 2 months after baseline (canakinumab administration) ]Parent/patient quality of life assessment measured by 100mm visual analog scale (VAS) difference at screening vs. 2 months after administration of a single dose of canakinumab.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02775994
|Contact: Liora Harel, MDfirstname.lastname@example.org|
|Contact: Gil Amarilyo, MDemail@example.com|
|Petah tikva, Israel|
|Principal Investigator:||Liora Harel, MD||SCMCI|