E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Familial Cold Autoinflammatory Syndrome (FCAS) |
Urticaire familiale au froid |
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E.1.1.1 | Medical condition in easily understood language |
Familial Cold Autoinflammatory Syndrome (FCAS) |
Urticaire familiale au froid |
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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 |
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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 |
To assess the efficacy of DFV890 to reduce cold-induced inflammation in participants with FCAS. |
Evaluer l'efficacité du DFV890 dans la réduction de l'inflammation induite par le froid chez des patients atteints de FCAS. |
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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. |
- Evaluer la sécurité d’emploi et la tolérance du DFV890 ; - Evaluer l'efficacité du DFV890 dans l’amélioration des signes et symptômes d'un FCAS ; - Evaluer l'effet du DFV890 sur les résultats rapportés par le patient (PRO); |
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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. |
-Recueil du formulaire de consentement éclairé par écrit et signé avant toute évaluation spécifique à l'étude. - Indice de masse corporelle (IMC) compris entre 18 et 35 kg/m2 (IMC = poids corporel (kg) / [taille (m)]2) au moment de la sélection. -Diagnostic génétique de FCAS (cette analyse peut être réalisée dans le cadre de la sélection si elle n'est pas déja disponible dans le dossier du patient). -Antécédents et investigations cliniques compatibles avec la FCAS, en l'absence d'antécédents ou de diagnostic d'amylose et/ou de lesions organiques (par ex. surdité, oedème périorbitaire, lymphadénopathie et sérite).
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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-γ 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. |
- Traitement actuel par des médicaments anti-rejet et/ou immunomodulateurs qui ne peut pas être interrompu ou remplacé par un autre médicament dans les 28 jours ou 5 demi-vies (la durée la plus longue prévalant si la réglementation locale l'exige), ou jusqu'à ce que l’effet pharmacodynamique attendu soit revenu à la baseline pour les anticorps thérapeutiques immunomodulateurs, avant le test de dépistage par provocation au froid et pendant la durée de l'étude. - Infection bactérienne, virale ou fongique cliniquement significative, suspectée active ou chronique (y compris Mycobacterium tuberculosis) dans les 30 jours précédant l'administration. -Déficits immunitaires innés (par ex. Toll-Like Récepteurs (TLR), défauts de la voie de signalisation de l'interféron gamma (IFN-γ)) ou déficits immunitaires acquis (par ex. SIDA). -Présence d'une infection par le virus de l'immunodéficience humaine (VIH), de l'antigène de surface de l'hépatite B (AgHBs) ou de l’antigène de la nucléocapside du virus de l'hépatite B (anti-HBc), ou d'anticorps de l'hépatite C à la sélection. -Vaccination par vaccins vivants dans les 4 semaines précédant le jour 1 (c.-à-d. l première dose de DFV890). -Femmes en âge de procréer, à savoir toute femme physiologiquement apte à être enceinte, sauf si elles utilisent des méthodes de contraception très efficaces pendant toute la durée du traitement et pendant 10 jours après l’arrêt du traitement à l'étude. D'autres critères d'inclusion/exclusion définis par le protocole peuvent s'appliquer.
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline for white cell count (WCC). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Pre-baseline to post-baseline. |
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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. |
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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: pre-baseline to post-baseline - Change from baseline in Patient’s global assessment: pre-baseline to post-baseline. |
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
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 |
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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 | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 3 |
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 |
France |
Germany |
Italy |
United Kingdom |
United States |
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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
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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 | 0 |
E.8.9.1 | In the Member State concerned months | 5 |
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 | 10 |
E.8.9.2 | In all countries concerned by the trial days | 28 |