E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
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Polyradiculonévrite inflammatoire démyélinisante chronique (PIDC) |
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E.1.1.1 | Medical condition in easily understood language |
Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is a rare immune-mediated disorder of the peripheral nerves.
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La polyradiculonévrite inflammatoire démyélinisante chronique (PIDC) est un trouble rare des nerfs périphériques. |
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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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10057645 |
E.1.2 | Term | Chronic inflammatory demyelinating polyradiculoneuropathy |
E.1.2 | System Organ Class | 10029205 - Nervous system 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 evaluate the clinical efficacy of rozanolixizumab as a treatment for subjects with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)
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Évaluer l’efficacité clinique du rozanolixizumab dans le traitement des patients présentant une PIDC |
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E.2.2 | Secondary objectives of the trial |
- To evaluate the safety and tolerability of rozanolixizumab in subjects with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) - To assess the pharmacodynamic (PD) effect of rozanolixizumab as measured by the total immunoglobulin G (IgG) concentrations in serum |
- Évaluer la tolérance et la sécurité d’emploi du rozanolixizumab chez des patients présentant une PIDC - Évaluer l’effet pharmacodynamique (PD) du rozanolixizumab d’après la mesure des concentrations sériques d’IgG totales |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Subject is ≥ 18 years of age at Visit 1 (Screening) - Subject has a documented definite or probable diagnosis of Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) according to the European Federation of Neurological Societies (EFNS)/ Peripheral Nerve Society (PNS) criteria 2010 - Subject has an immunoglobulin-dependency confirmed by clinical examination during therapy or upon interruption or reduction of therapy within 18 months prior to Screening and documented in medical history - Subject is on a stable dosage (not more than ±20% deviation) for subcutaneous immunoglobulin (SCIg) or intravenous immunoglobulin (IVIg) and a fixed interval for at least 4 months of either treatment - Female subjects of childbearing potential must agree to use a highly effective method of birth control, during the study and for a period of 2 months after their final dose of IMP - Male subjects with a partner of childbearing potential must be willing to use a condom when sexually active during the study and for 3 months after the final administration of IMP |
- Patient(e) âgé(e) de ≥ 18 ans à la visite 1 (sélection) - Diagnostic certain ou probable documenté de PIDC d’après les critères EFNS/PNS de 2010 - Dépendance aux Ig confirmée par les examens cliniques pendant le traitement, ou lors de l’arrêt ou la diminution du traitement dans les 18 mois précédant la sélection, et documentée dans les antécédents médicaux - Immunoglobulinothérapie par voie sous-cutanée (SCIg) ou intraveineuse (IVIg) à dose stable (écart inférieur à ± 20 %) et à intervalle posologique fixe depuis au moins 4 mois pour l’une ou l’autre voie d’administration - Les femmes en âge de procréer devront accepter d’utiliser un moyen de contraception hautement efficace pendant l’étude et pendant une période de 2 mois après leur dose finale de médicament à l’étude - Les patients ayant une partenaire en âge de procréer doivent accepter d’utiliser un préservatif lors des rapports sexuels pendant l’étude et pendant les 3 mois suivant la dernière administration du médicament à l’étude |
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E.4 | Principal exclusion criteria |
- Previously received treatment in this study or subject has previously been exposed to rozanolixizumab - Current diagnosis or has a history of Type 1 or Type 2 diabetes mellitus - Known immunoglobulin M (IgM)-mediated neuropathy - Clinical or known evidence of associated systemic diseases that might cause neuropathy or treatment with agents that might lead to neuropathy - History of clinically relevant ongoing chronic infections - Family history of primary immunodeficiency - Received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP - Received any experimental biological agent within or outside of a clinical study in the past 3 months or within 5 half-lives prior to Baseline - Prior treatment with rituximab, ofatumumab, or ocrelizumab in the 6 months prior to the Baseline Visit or subject has had prior treatment with rituximab, ofatumumab, or ocrelizumab in the 12 months prior to Baseline and B cells are not within the normal range - Female subject who is pregnant or lactating |
- Le patient a déjà reçu le traitement dans le cadre de la présente étude ou a eu une exposition antérieure au rozanolixizumab - Diagnostic en cours ou antécédents de diabète de Type 1 ou de Type 2 - Neuropathie connue à IgM - Signes cliniques ou connus de maladies systémiques associées susceptibles de provoquer une neuropathie ou traitement par des agents susceptibles de conduire à une neuropathie - Antécédents d’infections chroniques cliniquement significatives en cours - Antécédents familiaux d’immunodéficience primitive - Administration d’un vaccin vivant dans les 8 semaines précédant la visite d’inclusion ou administration prévue d’un vaccin vivant pendant l’étude ou dans les 7 semaines suivant la dose finale de médicament à l’étude - Administration d’un agent biologique expérimental dans le cadre d’une étude clinique ou en dehors d’une étude clinique, au cours des 3 mois ou des 5 demi-vies précédant l’inclusion - Traitement antérieur par rituximab, ofatumumab ou ocrélizumab dans les 6 mois précédant la visite d’inclusion ou traitement antérieur par rituximab, ofatumumab ou ocrélizumab dans les 12 mois précédant la visite d’inclusion sans normalisation des lymphocytes B - Grossesse ou allaitement en cours |
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from Baseline to Week 13 (Day 85) in inflammatory Rasch-built Overall Disability Scale (iRODS) score
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Modification du score iRODS à la Semaine 13 (Jour 85) par rapport aux valeurs initiales |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
From Baseline up to Week 13 (Day 85) |
À la Semaine 13 (Jour 85) par rapport aux valeurs initiales |
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E.5.2 | Secondary end point(s) |
No secondary end points are defined. |
Aucun objectif secondaire est définié. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
No secondary end points are defined. |
Aucun objectif secondaire est définié. |
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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 | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability and Immunogenicity |
Tolérance et l’immunogénicité |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
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 |
Canada |
Denmark |
France |
Germany |
Netherlands |
Spain |
United Kingdom |
United States |
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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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Last Subject Last Visit (LSLV) |
Dernière visite du dernier patient |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 14 |
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 |
E.8.9.2 | In all countries concerned by the trial days | 15 |