E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary haemophagocytic lymphohistiocytosis which has reactivated. |
linfohistiocitosis hemofagocítica primaria que se ha reactivado |
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E.1.1.1 | Medical condition in easily understood language |
A rare, life-threatening condition affecting predominantly children. The disease is characterized by uncontrolled hyper-inflammation on the basis of underlying immune deficiencies. |
Rara y potencialmente enfermedad mortal que afecta predominantemente a niños. La enfermedad se caracteriza por la incontrolada hiper- inflamación basándose en subyacente deficiencias inmunológicas |
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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 | 14.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10010331 |
E.1.2 | Term | Congenital, familial and genetic disorders |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic 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 monitor the long-term safety profile of patients who received NI-0501. - To assess HLH patients' survival after NI-0501 treatment. - To study the elimination profile of NI-0501. - To assess the immunogenicity of NI-0501. |
- Vigilar el perfil de seguridad a largo plazo de los pacientes que han recibido NI-0501 - Evaluar la supervivencia de los pacientes con LHH después del tratamiento con NI-0501 - Estudiar el perfil de eliminación del NI-0501 - Evaluar la inmunogenia del NI-0501 |
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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. Having received at least one dose of NI-0501 during a previous NI-0501 study. 2. Having signed the Informed Consent by the patient or the patient's legal representative(s), as applicable, with the assent of patients who are legally capable of providing it. 3. Having agreed to continue using adequate methods of birth control until 6 months after the last administered dose of NI-0501, when relevant |
1. El paciente ha recibido como mínimo una dosis de NI-0501 2. El paciente o su representante legal, según proceda, ha firmado el consentimiento informado y se ha obtenido el asentimiento de los pacientes con capacidad legal para otorgarlo 3. El paciente se ha comprometido continuar utilizando métodos anticonceptivos hasta 6 meses después de la última dosis administrada de NI-0501, si procede |
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E.4 | Principal exclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
* Safety: - Incidence, intensity, relationship to NI-0501 and outcomes of Adverse Events (serious and non-serious) - Evolution of vital signs, physical examination and laboratory values over time * Efficacy: - Clinical Response mostly based on HLH diagnostic criteria and existence of neurological symptomatology - Survival before HSCT, 100 days and one year after post HSCT * Pharmacokinetics: NI-0501 elimination profile (clearance and elimination half-life) * Pharmacodynamics: NI-0501 activity on IFN? neutralization * Immunogenicity: presence of ADA * Exploratory endpoints: e.g. additional markers of disease activity |
* Seguridad: - Incidencia, intensidad, relación con el NI-0501 y desenlaces de los acontecimientos adversos (graves y no graves) - Evolución de las constantes vitales, la exploración física y los valores de laboratorio a lo largo del tiempo * Eficacia: - Respuesta clínica basada fundamentalmente en los criterios diagnósticos de LHH y en la existencia de síntomas neurológicos - Supervivencia antes del TCMH, 100 días y un año después del TCMH * Farmacocinética: perfil de eliminación del NI-0501 (aclaramiento y semivida de eliminación) * Farmacodinámica: actividad del NI-0501 sobre la neutralización del IFN? * Inmunogenia: presencia de ADA * Criterios de valoración exploratorios: por ejemplo, otros marcadores de la actividad de la enfermedad |
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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 | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
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 | 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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 |
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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 | 6 |
E.8.9.1 | In the Member State concerned days | |
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 | 6 |