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

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ClinicalTrials.gov Identifier: NCT02775994
Recruitment Status : Recruiting
First Posted : May 18, 2016
Last Update Posted : September 3, 2018
Sponsor:
Information provided by (Responsible Party):
Rabin Medical Center

Brief 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).


Condition or disease Intervention/treatment Phase
Periodic Fever Drug: Canakinumab Phase 1

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Experimental: Treatment Arm
Single dose of Canakinumab 4mg/kg
Drug: Canakinumab
interleukin beta (IL-1) antagonist
Other Name: Ilaris




Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. 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).

  2. 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.



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Ages Eligible for Study:   2 Years to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

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


Contacts
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Contact: Liora Harel, MD +972544334856 liorahar@clalit.org.il
Contact: Gil Amarilyo, MD +972504057153 gamarilyo@clalit.org.il

Locations
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Israel
SCMCI Recruiting
Petah tikva, Israel
Sponsors and Collaborators
Rabin Medical Center
Investigators
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Principal Investigator: Liora Harel, MD SCMCI

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Responsible Party: Rabin Medical Center
ClinicalTrials.gov Identifier: NCT02775994     History of Changes
Other Study ID Numbers: 0032-16-RMC
First Posted: May 18, 2016    Key Record Dates
Last Update Posted: September 3, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
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Fever
Pharyngitis
Stomatitis
Stomatitis, Aphthous
Familial Mediterranean Fever
Amyloidosis
Body Temperature Changes
Signs and Symptoms
Pharyngeal Diseases
Stomatognathic Diseases
Respiratory Tract Infections
Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Mouth Diseases
Hereditary Autoinflammatory Diseases
Genetic Diseases, Inborn
Proteostasis Deficiencies
Metabolic Diseases
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs