E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) |
Polirradiculoneuropatía desmielinizante inflamatoria crónica (CIDP) |
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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. |
La Polirradiculoneuropatía desmielinizante inflamatoria crónica (CIDP) es trastorno inmunitario poco frecuente de los nervios periféricos |
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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) |
Evaluar la eficacia clínica del rozanolixizumab como tratamiento de sujetos con Polirradiculoneuropatía desmielinizante inflamatoria crónica (CIDP) |
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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 |
- Evaluar la seguridad y la tolerabilidad del rozanolixizumab en sujetos con Polirradiculoneuropatía desmielinizante inflamatoria crónica (CIDP) - Evaluar el efecto farmacodinámico (PD) del rozanolixizumab en su determinación mediante las concentraciones de inmunoglobulina G (IgG) total en suero |
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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 |
- Edad del sujeto >=18 años en la Visita 1 (Selección) - Sujeto con diagnóstico confirmado de polirradiculoneuropatía desmielinizante inflamatoria crónica (PDIC) definitiva o probable según los criterios de 2010 de la European Federation of Neurological Societies (EFNS)/Peripheral Nerve Society (PNS) - Sujeto con dependencia inmunoglobulínica confirmada mediante exploración física durante el tratamiento o tras la interrupción o reducción del tratamiento, en el plazo de los 18 meses anteriores a la Selección y documentada en la historia clínica - Sujeto en tratamiento inmunoglobulínico por vía subcutánea (SCIg) o intravenosa (IVIg) a dosis estable (desviación no superior a ±20%) e intervalo fijo durante por lo menos 4 meses - Las mujeres potencialmente fértiles deben estar de acuerdo en utilizar un método anticonceptivo muy efectivo durante el estudio y hasta 2 meses después de la última administración del medicamento en investigación - Los varones con parejas potencialmente fértiles deben estar dispuestos a usar preservativos en sus relaciones sexuales durante el estudio y hasta 3 meses después de la última administración del medicamento en investigación |
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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 |
- Sujeto que ha recibido tratamiento en este estudio con anterioridad o que ha estado expuesto previamente al rozanolixizumab - Diagnóstico actual o antecedentes de diabetes mellitus de tipo 1 o de tipo 2 - Neuropatía mediada por inmunoglobulina M (IgM) - Manifestaciones clínicas o evidencia de enfermedades sistémicas asociadas que puedan causar neuropatía o tratamiento con agentes que puedan causar neuropatía - Historia de infecciones crónicas en curso, de importancia clínica - Antecedentes familiares de inmunodeficiencia primaria - Sujeto que ha recibido una vacuna de gérmenes vivos en el plazo de las 8 semanas anteriores a la Visita Basal, o que tiene previsto recibirla en el transcurso del estudio o en el plazo de las 7 semanas siguientes a la última administración del medicamento en investigación - Sujeto que ha recibido un producto biológico experimental dentro o fuera de un estudio clínico en los últimos 3 meses o en el plazo de 5 semividas del producto antes de la Visita Basal - Sujeto que ha recibido tratamiento previo con rituximab, ofatumumab u ocrelizumab en los 6 meses anteriores a la Visita Basal, o en los 12 meses anteriores a la Visita Basal y los linfocitos B no se encuentran dentro del intervalo de la normalidad - Mujer embarazada o en periodo de lactancia |
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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 |
Variación de la puntuación de la Inflammatory Rasch-built Overall Disability Scale (iRODS) entre el momento Basal y la Semana 13 (Día 85) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
From Baseline up to Week 13 (Day 85) |
Entre el momento Basal y la Semana 13 (Día 85) |
|
E.5.2 | Secondary end point(s) |
No secondary end points are defined. |
No se han establecido criterios secundarios de valoración. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
No secondary end points are defined. |
No se han establecido criterios secundarios de valoración. |
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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 |
Tolerabilidad e Inmunogenética |
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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 | 2 |
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
|
Last Subject Last Visit (LSLV) |
Ultima Visita del Ultimo Sujeto (LSLV) |
|
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 | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
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 |