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

NCT02775994 | Unknown Phase 1

• 1 other identifier: 0032-16-RMC
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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).

Conditions

Periodic Fever

Outcome Measures

Primary Outcomes (1)

• Change in average number of flares - documented by the use of diary

  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).

  baseline (canakinumab administration) and 2 months after baseline

Secondary Outcomes (2)

• Time to flare - documented by the use of diary

  baseline (canakinumab administration) and up to 24 weeks after baseline

• Change in quality of life - documented by the use of questionnaire

  at the time of screening visit and 2 months after baseline (canakinumab administration)

Study Arms (1)

Treatment Arm

EXPERIMENTAL

Single dose of Canakinumab 4mg/kg

Drug: Canakinumab

Interventions

Canakinumab DRUG

interleukin beta (IL-1) antagonist

Also known as: Ilaris

Treatment Arm

Eligibility Criteria

• Age: 2 Years - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Suffer from more than 4 PFAPA flares 2 months prior to the screening period.
• Have signed informed consent for the study.
• Have at least 2 documented PFAPA flare during the screening period.

You may not qualify if:

• Patients who are diagnosed with chronic disease including another auto inflammatory disease.
• Patients who suffer from neutropenia or elevated liver enzymes.
• Patients who receive treatment with cimetidine/famotidine, montelukast or colchicine 2 weeks or less prior to enrollment.

Sponsors & Collaborators

• Rabin Medical Center: lead

Study Sites (1)

SCMCI

Petah Tikva, Israel

RECRUITING

MeSH Terms

Conditions

Hereditary Autoinflammatory Diseases

Interventions

canakinumab

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Skin Diseases; Skin and Connective Tissue Diseases

Study Officials

• Liora Harel, MD

  SCMCI

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Liora Harel, MD

CONTACT

+972544334856; liorahar@clalit.org.il

Gil Amarilyo, MD

CONTACT

+972504057153; gamarilyo@clalit.org.il

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 27, 2016
• First Posted: May 18, 2016
• Study Start: August 1, 2016
• Primary Completion: January 1, 2020
• Study Completion: March 1, 2020
• Last Updated: September 4, 2018

Record last verified: 2018-08

Locations

IL (1)