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 | 16.1 |
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 | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate long-term safety of high-dose IgPro20 in subjects who have participated in the SC Treatment Period of pivotal study IgPro20_3003 |
Evaluar la seguridad a largo plazo de la dosis alta de IgPro20 en sujetos que hayan participado en el período de tratamiento SC del estudio fundamental IgPro20_3003 |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives: ? To evaluate the long-term safety of low-dose IgPro20. ? To evaluate the long-term efficacy of high-dose and low-dose IgPro20. |
Objetivos secundarios: ? Evaluar la seguridad a largo plazo de la dosis baja de IgPro20. ? Evaluar la eficacia de la dosis alta y de la dosis baja de IgPro20. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Subjects having participated in the SC Treatment Period of pivotal study IgPro20_3003 (NCT01545076). - Written informed consent for study participation obtained before undergoing any study-specific procedures. - If treatment for CIDP is required, it has to include IgGs. - Subjects who completed study IgPro20_3003 before study IgPro20_3004 study was open for enrollment at the site must be enrolled no later than 8 weeks after study IgPro20_3004 is open for enrollment at the site. - Subjects who completed study IgPro20_3003 after study IgPro20_3004 study was open for enrollment at the site must be enrolled no later than 8 weeks after the completion visit of study IgPro20_3003 |
- Haber participado en el período de tratamiento SC del estudio fundamental IgPro20_3003(NCT01545076). - Haber proporcionado el consentimiento informado por escrito para participar en el estudio antes de realizar ninguno de los procedimientos específicos del estudio. - En caso de que se requiera tratamiento para la CIDP, este deberá incluir la administración de IgG. - En relación con los pacientes que hayan realizado la visita de finalización del estudio IgPro20_3003 antes de que se iniciara el reclutamiento en el estudio IgPro20_3004 en el centro del estudio en el que participará el paciente, el reclutamiento no deberá llevarse a cabo más de 8 semanas después de que se haya iniciado el reclutamiento del estudio IgPro20_3004 en el centro del estudio en el que participará el paciente. - En relación con los pacientes que hayan completado el estudio IgPro20_3003 después de que se iniciara el reclutamiento en el estudio IgPro20_3004 en el centro del estudio en el que participará el paciente, el reclutamiento no deberá realizarse más de 8 semanas después de que el paciente haya completado la visita de finalización del estudio IgPro20_3003 |
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E.4 | Principal exclusion criteria |
- Any polyneuropathy of other causes - Any other disease (mainly neurological or chronic orthopedic) that has caused neurological symptoms or may interfere with treatment or outcome assessments - Severe diseases and conditions that are likely to interfere with evaluation of the study product or satisfactory conduct of the study - History of thrombotic episodes within the 2 years prior to enrolment - Known allergic or other severe reactions to blood products including intolerability to previous IVIG (normal human immunoglobulin for intravenous administration) and/or SCIG (subcutaneous immunoglobulin) |
- Cualquier polineuropatía motivada por otras causas - Cualquier otra enfermedad (principalmente neurológicas, u ortopédicas de carácter crónico), que hayan provocado síntomas neurológicos o que puedan interferir con el tratamiento o la evaluación de los resultados - Enfermedades y trastornos graves que puedan interferir con la evaluación del producto en estudio o con el desarrollo satisfactorio del estudio - Antecedentes de episodios trombóticos en el transcurso de los 2 años previos al reclutamiento - Reacción alérgica u otra reacción intensa conocida a los productos sanguíneos. entre otras, intolerancia a IgIV y/o IgSC previos |
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall rate of adverse events (AEs) per infusion - high-dose IgPro20 |
Tasa general de AA por infusión durante el período en el que se administre la dosis alta |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Up to 48 weeks |
Hasta 48 semanas |
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E.5.2 | Secondary end point(s) |
Safety: 1. Rate of adverse events (AEs) per infusion by severity, causality, and seriousness - high-dose IgPro20 2. Percentage of subjects with adverse events (AEs) - high-dose IgPro20 3. Rate of adverse events (AEs) per infusion - low-dose IgPro20 4. Percentage of subjects with adverse events (AEs) - low-dose IgPro20 Efficacy: 1. Total INCAT score at all study visits 2. Time to first CIDP relapse |
Seguridad: 1. Tasa de AA por infusión por intensidad, causalidad y gravedad) dosis alta IgPro20 2. Porcentaje de pacientes con AA durante el período en el que se administre la dosis alta IgPro20 3. Tasa de AA por infusión durante el período en el que se administre la dosis baja IgPro20 4. Porcentaje de pacientes con AA durante el período en el que se administre la dosis baja IgPro20 Eficacia: 1. Puntuación total de la escala INCAT a lo largo de todas las visitas del estudio. 2. Tiempo transcurrido hasta la primera recaída de CIDP. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Safety 1. and 2.= Up to 48 weeks Safety 3. and 4.= Up to 28 weeks
Efficacy 1.= Up to 49 weeks (baseline [Week 1], Weeks 2, 9, 17, 25, 33, 41, and at completion visit) Efficacy 2.= Up to 49 weeks |
Seguridad 1. and 2. = Hasta 48 semanas Seguridad 3. and 4. = Hasta 28 semanas
Eficacia 1. = Hasta 49 semanas (basal [semana 1], semanas 2, 9, 17, 25, 33, 41 y visita de finalización) Eficacia 2. = Hasta 49 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) |
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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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
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
| 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 | 47 |
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 |
Canada |
Japan |
European Union |
Australia |
Israel |
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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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 | 9 |
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 | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |