PAPA syndrome (Pyogenic Arthritis, Pyoderma gangrenosum and Acne) MedDRA version: 20.0 Level: LLT Classification code 10072225 Term: PAPA syndrome System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10072225 Term: PAPA syndrome System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10072225 Term: PAPA syndrome System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10072225 Term: PAPA syndrome System
Conditions
Interventions
Product Name: Canakinumab
Product Code: [Canakinumab]
Pharmaceutical Form: Solution for injection
INN or Proposed INN: CANAKINUMAB
CAS Number: 914613-48-2
Current Sponsor code: ilaris, Canakinumab
Con
Sponsors
IRCCS ISTITUTO GIANNINA GASLINI
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Clinical diagnosis of PAPA with active flare at the time of enrolment. 2. Mutation of the PSTPIP1 gene. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1. Use of the following therapies: a. Corticosteroids (prednisone equivalent > 0.2 mg/kg/day [or greater than the maximum of 15 mg/ day for children over 60 kg]) within 1 week prior to Baseline b. Anakinra within 72 hours prior to Baseline c. Other biologics (including Canakinumab) or immunosuprassant 2 half-life prior to Baseline. 2. Any conditions or significant medical problems which in the opinion of the investigator immunocompromises the patient and/ or places the patient at unacceptable risk for immunomodulatory therapy 3. Live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose 4. Pregnant or nursing women 5. Female adolescents (=18 years of age) of childbearing potential who do not agree to abstinence or, if sexually active, do not agree to the use of contraception .
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine whether canakinumab administered every 4 weeks is able to maintain disease remission compared to placebo in canakinumab-responder patients;Secondary Objective: 1)To determine the efficacy of canakinumab to achieve a complete or almost complete response after 24 weeks 2)To evaluate optimal dose of canakinumab to induce disease remission 3)To assess the profile over time in each of the 4 key PAPA signs and symptoms (arthritis, foruncolosis, pyoderma gangrenosum, hydrosiadenite suppurativa) from baseline to end of study. 4)To assess the profile over time in physician’s and patients’s global assessment score from baseline to end of study. 5)To assess the profile over time in acute phase reactants (ESR, CRP, SAA) from baseline to end of study.;Primary end point(s): Proportion of patients not experiencing a disease flare in the treatment arm compared to the placebo arm at week 48.;Timepoint(s) of evaluation of this end point: 48 Weeks | — |
Countries
Italy
Contacts
Public ContactMal. Autoinf. e Immunodeficienze
IRCCS Istituto Giannina Gaslini
Outcome results
None listed