E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Pediatric patients with Atypical Hemolytic-Uremic Syndrome (aHUS) |
PACIENTES PEDIÁTRICOS CON SÍNDROME HEMOLÍTICO URÉMICO ATÍPICO |
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E.1.1.1 | Medical condition in easily understood language |
Atypical Hemolytic-Uremic Syndrome |
SÍNDROME HEMOLÍTICO URÉMICO ATÍPICO |
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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 | 14.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10019515 |
E.1.2 | Term | Hemolytic uremic syndrome |
E.1.2 | System Organ Class | 10005329 - Blood and lymphatic 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 |
Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment. |
Evaluar la eficacia y seguridad de eculizumab en pacientes pediátricos con SHUa para controlar la MAT, que se caracteriza por trombocitopenia, hemólisis e insuficiencia renal. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patient?s parent/legal guardian must be willing and able to give written informed consent and the patient must be willing to give written informed assent [if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)]. 2. Pediatric patients with aHUS. Patients may be newly diagnosed, or with previously diagnosed disease, or post-kidney transplant with the disease. 3. Patients from 1 month up to 18 years of age and body weight ? 5 kg. 4. Patient exhibit Thrombocytopenia, hemolysis and elevated Serum Creatinine |
1. El progenitor o el tutor legal deben estar dispuestos a dar el consentimiento informado por escrito y tener capacidad para hacerlo, y el paciente debe estar dispuesto a dar el asentimiento informado por escrito [si corresponde, según lo determine el Comité Ético de Investigación Clínica (CEIC) del centro]. 2.Pacientes pediátricos con SHUa. El diagnóstico podrá ser reciente, antiguo o posterior al trasplante de riñón. 3.Pacientes desde 1 mes hasta 18 años de edad y peso corporal ? 5 kg. 4.Pacientes presentan trombocitopenia, hemólisis, elevado creatinina sérica |
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E.4 | Principal exclusion criteria |
1. Plasma Therapy for > 5 weeks prior to enrollment. 2. Chronic dialysis 3. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients. |
1.Plasmaterapia durante > 5 semanas antes del reclutamiento. 2.Diálisis crónica 3.Uso previo de eculizumab o hipersensibilidad al eculizumab, a las proteínas murinas o a alguno de los excipientes |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Efficacy Endpoint: Proportion of patients with complete TMA response.
Primary Safety Endpoint: Safety assessments. |
Criterio de valoración principal de la eficacia: Porcentaje de pacientes con respuesta completa frente a la MAT
Criterio de valoración principal de la seguridad: seguridad |
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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) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | 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 |
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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 | No |
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.8.3 |
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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 28 |
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 |
Australia |
Canada |
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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The last visit of the last patient |
última visita del último paciente |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |