E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
PAPA syndrome (Pyogenic Arthritis, Pyoderma gangrenosum and Acne) |
Sindrome PAPA (artrite piogenica, pioderma gangrenoso e acne) |
|
E.1.1.1 | Medical condition in easily understood language |
PAPA syndrome (Pyogenic Arthritis, Pyoderma gangrenosum and Acne) |
Sindrome PAPA (artrite piogenica, pioderma gangrenoso e acne) |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10072225 |
E.1.2 | Term | PAPA syndrome |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10072225 |
E.1.2 | Term | PAPA syndrome |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10072225 |
E.1.2 | Term | PAPA syndrome |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10072225 |
E.1.2 | Term | PAPA syndrome |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine whether canakinumab administered every 4 weeks is able to maintain disease remission compared to placebo in canakinumab-responder patients |
Valutare se il canakinumab somministrato ogni 4 settimane sia superiore al placebo nel mantenere la remissione della malattia nei pazienti affetti da sindrome PAPA. |
|
E.2.2 | Secondary objectives of the trial |
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. |
1. Valutare la percentuale di pazienti in cui canakinumab è in grado di indurre remissione del quadro clinico dopo 24 settimane 2. Individuare la dose ottimale di canakinumab per ottenere la remissione della malattia. 3. Valutare il profilo di efficacia di canakinumab nei 4 sintomi tipici della PAPA (artrite, acne, pyoderma gangrenoso, idrosadenite) dall’arruolamento alla fine dello studio. 4. Valutare il profilo di efficacia di canakinumab nel tempo tramite il physician’s and patient’s global assessment score dall’arruolamento alla fine dello studio. 5. Valutare il profilo di efficacia di canakinumab nel tempo sul controllo degli indici di flogosi (VES, PCR,SAA) dall’arruolamento alla fine dello studio. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Clinical diagnosis of PAPA with active flare at the time of enrolment. 2. Mutation of the PSTPIP1 gene. |
1. Diagnosi clinica di PAPA con attività di malattia al momento dell'arruolamento 2. Mutazione del gene PSTPIP1 |
|
E.4 | Principal 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 . |
1. Utilizzo delle seguenti terapie: a. Corticosteroidi (prednisone > 0,2 mg/kg/giorno [o superiore al massimo di 15 mg/giorno per i bambini di peso superiore a 60 kg]) entro 1 settimana prima del basale b. Anakinra entro 72 ore prima del basale c. Altri farmaci biologici (incluso Canakinumab) o immunosoppressori per un intervallo di tempo di almeno di 2 emivita prima del basale. 2. Qualsiasi condizione o problema clinico significativo che, a parere dello sperimentatore, renda immunocompromesso il paziente e/o le espone a un rischio inaccettabile per la terapia immunomodulante. 3. Vaccinazioni vive entro 3 mesi prima dell'inizio della sperimentazione, durante la sperimentazione e fino a 3 mesi dopo l'ultima dose 4. Donne in gravidanza o in allattamento 5. Adolescenti femmine (=18 anni di età) in età fertile che non acconsentono all'astinenza o, se sessualmente attive, non acconsentono all'uso della contraccezione |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of patients not experiencing a disease flare in the treatment arm compared to the placebo arm at week 48. |
Confronto tra la percentuale di soggetti senza ricaduta di malattia nel braccio di trattamento e quelli nel braccio con placebo alla settimana 48. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |