E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cryopyrin Associated Periodic Syndrome |
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E.1.1.1 | Medical condition in easily understood language |
Cryopyrin Associated Periodic Syndrome |
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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 | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10068850 |
E.1.2 | Term | Cryopyrin associated periodic syndrome |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate safety and tolerability of canakinumab in Canadian patients with CAPS defined as:
• Familial Cold Autoinflammatory Syndrome (FCAS)/ Familial Cold Urticaria (FCU);
• Muckle-Wells Syndrome (MWS);
• Neonatal-Onset Multisystem Inflammatory Disease (NOMID)/ Chronic Infantile Neurological, Cutaneous, Articular Syndrome (CINCA).
To assess the maintenance of response over time under routine medical care in patients with the following CAPS related diseases: FCAS/FCU, MWS, NOMID/CINCA. |
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E.2.2 | Secondary objectives of the trial |
• To assess the long term maintenance of Health-Related Quality of Life (HRQOL) and productivity;
• To assess treatment adherence with canakinumab for the duration of the study. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male and female patients at least 4 years of age at the time of the screening visit
2. Patient's informed consent for > or = 18 years of age before any assessment is performed
3. Parent or legal guardian's written informed consent and child's assent, if appropriate, are required before any assessment is performed for patients < 18 years of age.
4. Patients with a diagnosis of CAPS: FCAS/FCU, MWS, or NOMID/CINCA
5. Body weight > or = 15 kg
6. Able to communicate with the investigator and comply with the requirements of the study (for children the parent can assist when necessary)
Other protocol-defined inclusion/exclusion criteria may apply |
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E.4 | Principal exclusion criteria |
1. Patient is pregnant or breastfeeding. Women of childbearing potential must have a negative urine pregnancy test at baseline
2. Patient of childbearing potential (man or woman) and unwilling to use double barrier method of contraception. It is required that double barrier method of contraception be used (i.e. condom with spermicide or diaphragm with spermicide) by patients of childbearing potential (man or woman) regardless of whether a hormonal agent is also used as a method of contraception.
3. Participation in any clinical investigation within 4 weeks prior to dosing
4. Live vaccinations within 3 months prior to the start of the trial, during the trial and up to 3 months following the last dose.
5. History of significant medical conditions, which in the Investigator's opinion would exclude the patient from participating in this trial (this can be discussed with Novartis on a case by case basis in case of uncertainty).
6. History of drug or alcohol abuse within 12 months prior to dosing
7. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes
Other protocol-defined inclusion/exclusion criteria may apply
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety & tolerability of canakinumab by measuring the maintenance of response over time using the Investigators Clinical Assessment of Disease Activity scale and the evaluation of serum inflammation markers CRP (C-Reactive Protein), SAA (Serum Amyloid A. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
- Change in long term maintenance of Health-Related Quality of Life and productivity measured from baseline to end of study.
- Treatment adherence with canakinumab for the duration of the study.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Point 1: Baseline, 18 months
Point 2: 18 months |
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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 | No |
E.6.4 | Safety | Yes |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
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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) | 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.8.2.4 | Number of treatment arms in the trial | 1 |
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 |
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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
|
17-May-2012 (last patient completed) |
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E.8.9 Initial estimate of the duration of the trial |
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 | 9 |
E.8.9.2 | In all countries concerned by the trial days | 8 |