E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Disease is characterized by persistent fever, redness of the eyes, of the lips and of the mouth, swelling of palms and soles often with desquamation, rash and swelling of lymph nodes in the neck. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10023320 |
E.1.2 | Term | Kawasaki's disease |
E.1.2 | System Organ Class | 10047065 - Vascular disorders |
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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 |
The primary objective of this study is to evaluate the proportion of patients with resolution of fever, defined as body temperature ≤37.5 °C ≥48 to <72 hours (Day 3/4) after the administration of canakinumab. |
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E.2.2 | Secondary objectives of the trial |
To evaluate:
· proportion of patients with CRP ≤15 mg/L by ≥48 to <72 hours (study Days 3/4), 8 and 29
· proportion of patients developing new coronary artery aneurysms at Day 29
· time to first resolution of fever (≤37.5 °C)
· proportion of patients with remittent fever (>37.5 °C) by Day 29
· location, number and size of CAA at Days 29 and 85
· CAA evolution over time by echography at Days 43, 57 and 85
Other protocol-defined secondary objectives may apply. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Female and male patients ≥28 days and ≤10 years of age.
Active Kawasaki disease defined as:
· fever ≥38.5°C for ≥5 days
· four out of five of the following criteria: (i) conjunctival injection, (ii) oral mucous membrane changes, (e.g., injected pharynx, or strawberry tongue), (iii) erythema of hands or feet, (iv) polymorphous rash, (v) cervical lymphadenopathy
Other protocol-defined inclusion criteria may apply |
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E.4 | Principal exclusion criteria |
Patients fulfilling any of the following criteria are not eligible for inclusion in this study:
· Previous KD diagnosis, refractory and/or incomplete Kawasaki disease (for IVIG-naïve patients, Cohort 1)
· Patients who had fever for longer than a week (for IVIG-naïve patients, Cohort 1), or longer than 14 days (for IVIG-refractory patients, Cohort 2)
· History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or excipients (e.g. citric acid and anhydrous; sodium chloride disodium edetate dehydrate polysorbate 80; sodium hydroxide; water for injections)
Other protocol-defined exclusion criteria may apply |
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E.5 End points |
E.5.1 | Primary end point(s) |
the proportion of patients with resolution of fever, defined as body temperature ≤37.5 °C ≥48 to <72 hours (Day 3/4) after the administration of canakinumab. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
≥48 to <72 hours (Day 3/4) after the administration of canakinumab |
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E.5.2 | Secondary end point(s) |
· proportion of patients with CRP ≤15 mg/L by ≥48 to <72 hours (study Days 3/4), 8 and 29
· proportion of patients developing new coronary artery aneurysms at Day 29
· time to first resolution of fever (≤37.5 °C)
· proportion of patients with remittent fever (>37.5 °C) by Day 29
· location, number and size of CAA at Days 29 and 85
· CAA evolution over time by echography at Days 43, 57 and 85
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
• Immunogenicity
• Antibodies against vaccine antigen
• Soluble and cellular biomarkers |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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 | No |
E.8.2.3 | Other | No |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
Germany |
Italy |
Netherlands |
Poland |
Switzerland |
Turkey |
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E.8.7 | Trial has a data monitoring committee | Yes |
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
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The study as a whole will end after the last patient completed the End of Study on Day 85 or follow up visit (Visit 299) if applicable, prematurely discontinues the trial or is lost to follow up.
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |