E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Familial Cold Autoinflammatory Syndrome (FCAS). |
Sindrome Familiare auto-infiammatoria da freddo (FCAS). |
|
E.1.1.1 | Medical condition in easily understood language |
Familial Cold Autoinflammatory Syndrome (FCAS). |
Sindrome Familiare auto-infiammatoria da freddo (FCAS). |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
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 |
|
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 assess the efficacy of DFV890 to reduce cold-induced inflammation in participants with FCAS. |
Valutare l’efficacia di DFV890 nel ridurre l’infiammazione indotta dal freddo in soggetti con FCAS. |
|
E.2.2 | Secondary objectives of the trial |
- To assess safety and tolerability of DFV890; - To assess the efficacy of DFV890 to improve the signs and symptoms of FCAS; - To assess the effect of DFV890 on patient reported outcomes. |
- Valutare la sicurezza e la tollerabilità di DFV890; - Valutare l’efficacia di DFV890 nel migliorare i segni e i sintomi della FCAS; - Valutare l’effetto di DFV890 sugli esiti riportati dai pazienti. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Written informed consent must be obtained before any study-specific assessment is performed. - Body mass index within the range of 18-35 kg/m2. - Patients with a genetic diagnosis of FCAS. - Patients with a clinical history and investigations consistent with FCAS. |
- |
|
E.4 | Principal exclusion criteria |
- Participants currently being treated with anti-rejection and/or immunomodulatory drugs and the treatment cannot be discontinued or switched to a different medication. - Clinically significant, suspected active or chronic bacterial (including Mycobacterium tuberculosis), viral or fungal infection within 30 days prior to dosing. - Patients with innate (e.g. TLR immunodeficiencies, defects in IFN-¿ signaling) or acquired immune deficiencies (e.g. AIDS). - Presence of human immunodeficiency virus (HIV) infection, hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (anti-HBc), or hepatitis C antibodies at screening. - Live vaccines within 4 weeks of dosing. - Pregnant or nursing (lactating) women. - Women of child-bearing potential unless they are using highly effective methods of contraception. Other protocol-defined inclusion/exclusion criteria may apply. |
- |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline for white cell count (WCC). |
- |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Pre-baseline to post-baseline. |
- |
|
E.5.2 | Secondary end point(s) |
- Number and severity of safety assessments and adverse events. - Change from baseline in physician assessed disease scores. - Change from baseline in Patient's global assessment. |
- |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Number and severity of safety assessments and adverse events: up to End of Study. - Change from baseline in physician assessed disease scores: prebaseline to post-baseline - Change from baseline in Patient's global assessment: pre-baseline to post-baseline. |
- |
|
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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 |
Tolerability. |
Tollerabilità. |
|
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
Proof of Principle (Ph1/2) |
Prova di Principio (Ph1/2) |
|
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 | No |
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 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
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 | Information not present in EudraCT |
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 |
United States |
Germany |
Italy |
|
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 | 0 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 3 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
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 | 3 |