E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Three disease of periodic fever syndromes, TNF receptor Associated Periodic Syndrome (TRAPS), Hyper IgDSyndrome (HIDS)/ Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF |
|
E.1.1.1 | Medical condition in easily understood language |
Periodic Fever Syndromes, also referred to as hereditary recurrent fevers or mono-genic disorders. A key feature is the recurrent episodes of systemic inflammation with high and disabling fever |
|
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 | SOC |
E.1.2 | Classification code | 10010331 |
E.1.2 | Term | Congenital, familial and genetic disorders |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
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 safety and tolerability of ACZ885 in this extension study. This extension study offers the opportunity for patients who completed Epoch 4 of the preceding CACZ885N2301 study to continue to be treated with ACZ885 until approval in Japan of the drug in Periodic Fever Syndromes or until development of ACZ885 in Periodic Fever Syndromes is suspended. |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Key Inclusion criteria: Completed Epoch 4 of the CACZ885N2301 study in Japan before the approval of canakinumab in Japan. Written informed consent. Parent or legal guardian's written informed consent and child's assent, if appropriate, are required before any assessment is performed for patients < 20 years of age
|
|
E.4 | Principal exclusion criteria |
Key Exclusion criteria: Any conditions or significant medical problems in which the investigator judges the patient should not enter this extension study |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Timeframe: Participants will be followed for the duration until approval, an expected average of 3 months.
Outcome Measure Description: To evaluate the safety and tolerability of canakinumab
|
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Timeframe: Participants will be followed for the duration until approval, an expected average of 3 months.
Outcome Measure Description: To evaluate the safety and tolerability of canakinumab
|
|
E.5.2 | Secondary end point(s) |
Participants will be followed for the duration until approval, an expected average of 3 months.
Outcome Measure Description: To evaluate the safety and tolerability of canakinumab
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Participants will be followed for the duration until approval, an expected average of 3 months.
Outcome Measure Description: To evaluate the safety and tolerability of canakinumab
|
|
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 | No |
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 | 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 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
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.8.9.2 | In all countries concerned by the trial months | 4 |