E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) |
Polineuropatía desmielinizante inflamatoria crónica (CIDP) |
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E.1.1.1 | Medical condition in easily understood language |
Inflammatory disease of peripheral nervous system |
Enfermedad inflamatoria del sistema nervioso periférico. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061811 |
E.1.2 | Term | Demyelinating polyneuropathy |
E.1.2 | System Organ Class | 10029205 - Nervous system 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 determine the efficacy of 2 different doses of IgPro20 (0.2 g/kg bw and/or 0.4 g/kg bw) in the maintenance treatment of CIDP in comparison to placebo. |
Determinar la eficacia de 2 dosis diferentes de IgPro20 (0,2 g/kg de peso corporal o 0,4 g/kg de peso corporal) en el tratamiento de mantenimiento de la CIDP, en comparación con placebo. |
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E.2.2 | Secondary objectives of the trial |
- To investigate the efficacy of IgPro20 with additional clinical outcome measures in comparison to placebo. - To investigate the safety and tolerability of IgPro20 in comparison to placebo. - To investigate health-related quality of life (HRQL) following treatment with IgPro20. |
-Investigar otras formas de medir la eficacia de IgPro20 en la CIDP, en comparación con placebo. -Investigar la seguridad y la tolerabilidad de IgPro20 en comparación con las de placebo. -Investigar la calidad de vida relacionada con la salud (CdVS) tras el tratamiento con IgPro20. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Definite or probable CIDP according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria 2010. 2. Repeated treatment with IVIG (? 4 infusions) within the last 9 months prior to enrollment. 3. An IVIG treatment during the last 8 weeks prior to enrollment. 4. Age ?18 years. 5. Written informed consent for study participation obtained before undergoing any study-specific procedures. |
1. CIDP clara o probable según los criterios EFNS/PNS de 2010. 2. Tratamiento repetido con IVIG (? 4 infusiones) en los 9 meses previos al reclutamiento. 3. Tratamiento con IVIG durante las 8 semanas previas al reclutamiento 4. Edad ? 18 años. 5. De ambos sexos. 6. Consentimiento informado por escrito para la participación en el estudio, obtenido antes de someterse a cualquiera de los procedimientos específicos del estudio. |
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E.4 | Principal exclusion criteria |
1. Any polyneuropathy of other causes 2. Any other disease (mainly neurological or chronic orthopedic) that has caused neurological symptoms or may interfere with treatment or outcome assessments 3. Severe diseases and conditions that are likely to interfere with evaluation of the study product or satisfactory conduct of the study 4. History of thrombotic episodes within the 2 years prior to enrolment 5. Known allergic or other severe reactions to blood products including intolerability to previous IVIG |
1. Cualquier polineuropatía por otras causas. 2. Cualquier otra enfermedad (principalmente neurológica u ortopédica crónica) que haya causado síntomas neurológicos o que pueda interferir en el tratamiento o en las evaluaciones de los resultados. Enfermedades y trastornos severos que probablemente interfieran en la evaluación del producto del estudio o en la realización satisfactoria del estudio. 4. Antecedentes de episodios de trombosis en los 2 años previos al reclutamiento. 5. Reacciones alérgicas o severas de otro tipo a los hemoderivados, incluida la intolerancia previa de IVIG. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Percentage (%) of subjects who relapse during the SC treatment period |
Porcentaje (%) de sujetos que recidiven durante el tratamiento SC. |
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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) |
1. Change in mean Inflammatory Neuropathy Cause and Treatment (INCAT) scores during the SC treatment period 2. Change in mean maximum grip strength scores during the SC treatment period 3. Change in mean Medical Research Council (MRC) sum scores during the SC treatment period 4. Change in mean Rasch-built Overall Disability Scale (R-ODS) scores during the SC treatment period 5. Time to CIDP relapse 6. Rate of adverse events per SC infusion 7. Number of subjects with adverse events during the SC Treatment Period 8. Percentage of subjects with adverse events during the SC Treatment Period |
Eficacia: Variaciones de las medias durante el periodo de tratamiento SC entre los grupos en: 1.Puntuación INCAT. 2.Fuerza de prensión máxima (mano dominante/no dominante). 3.Puntuación acumulada del MRC. 4.R-ODS. 5.Diferencia en el ?tiempo hasta la recidiva de la CIDP? utilizando una estimación de Kaplan-Meier para la comparación de los grupos de IgPro20 con placebo y de los dos grupos entre ellos. Seguridad: 6.Tasa de AA por infusión durante el período de tratamiento SC, agrupados según los criterios SOC y PT del MedDRA. 7.Número y porcentaje de sujetos con AA durante el período de tratamiento SC, agrupados según los criterios de SOC y PT del MedDRA. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. - 4. SC week 1, SC week 25 5. up to 24 weeks 6. - 8. 24 weeks |
1. - 4. SC semana 1, SC semana 25 5. hasta 24 semanas 6. - 7. 24 semanas |
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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 | 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 |
Health-related quality of life (HRQL) |
Calidad de Vida relacionada con la salud (CdVS) |
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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 | 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 | 3 |
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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 70 |
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 |
Argentina |
Australia |
Austria |
Belgium |
Canada |
Czech Republic |
Finland |
France |
Germany |
Hungary |
Italy |
Korea, Republic of |
Netherlands |
Spain |
Switzerland |
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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LVLS |
Última visita del último sujeto |
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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 | 2 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |