E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Diffuse intrinsic pontine glioma (DIPG) |
Glioma intrínseco difuso de tronco (DIPG) |
|
E.1.1.1 | Medical condition in easily understood language |
Diffuse intrinsic pontine glioma (DIPG) |
Glioma intrínseco difuso de tronco (DIPG) |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10006143 |
E.1.2 | Term | Brain stem glioma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
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 assess the safety of a first in human administration of DCs vaccination pulsed with tumor lysate from a pool of eight K27M positive DIPG cell lines |
Evaluar la seguridad de una primera administración en humanos de la vacunación con CDs pulsadas con lisados procedentes de un pool de 8 líneas celulares de tumores DIPG K27M positivos en una cohorte de pacientes con DIPG |
|
E.2.2 | Secondary objectives of the trial |
Evaluate the nonspecific immune response generated in peripheral blood and CSF by proposed treatment Evaluate the specific antitumor immunity response generated in peripheral blood and CSF Assess overall survival and progression free survival Correlate the neuroradiological changes with the clinical course and immune response generated in peripheral blood and CSF Quality of life evaluation |
Evaluar la respuesta inmunogénica inespecífica generada en sangre periférica y LCR Evaluar la respuesta inmunogénica antitumoral generada en sangre periférica y LCR Estudiar la supervivencia global y el periodo libre de progresión Relacionar los cambios neuroradiológicos con la evolución clínica y la respuesta inmunológica generada en sangre periférica y LCR. Calidad de vida se valorará mediante la evaluación prospectiva del performance de cada paciente |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Newly diagnosed DIPG Patients without progressive disease Aged between 3 and 18 yo Lansky scale >50 (Karnosfsky for patients aged more than 16 yr) Life expectancy > 8 weeks Preserved bone marrow function Normal hepatic and renal function |
Pacientes con el diagnóstico reciente de DIPG Pacientes que hayan completado radioterapia focal en los 100 días previos a la inclusión Edad esté comprendida entre los 3 y 18 años de edad Escala funcional de Lansky> 50 ( Karnofsky> 50 para los mayores de 16 años) Expectativa de vida mayor a 8 semanas Función medular conservada No haber recibido tratamiento farmacológico antitumoral en el último mes |
|
E.4 | Principal exclusion criteria |
Impossibility to perform aphaeresis Patient part of other experimental study within the last 3 months Patient under antitumor treatment in the last 4 weeks Co-morbidity that does not allow the study treatment Patients requiring > 2mg/day of dexametasone treatment Corticoid-dependent patients Patients under uncontrolled infection Positive serologies of HIV, HCV or HBV |
Imposibilidad de realizar aféresis Participación en otro estudio experimental en los últimos tres meses Paciente que ha recibido tratamiento antitumoral las últimas 4 semanas Comorbilidad que impida tolerar el tratamiento en estudio pacientes que requieren >2mg/día de dexametasona pacientes corticoide-dependientes Pacientes con enfermedad diseminada Paciente con alguna infección incontrolada Serologías positivas de VIH, VHC o VHB |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Number of serious adverse events per patient (after treatment administration) |
Número de acontecimientos adversos graves (AAG) por paciente tras la primera administración del tratamiento en estudio |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Overall survival Progression free survival Time to first SAE (after treatment) Immunologic parameters Changes in radiological images Relationship between immunological response and clinical response Relationship between histological, and molecular characterization, and clinical and cellular response generated by vaccination Safety parameters evaluation Performance |
Tiempo de supervivencia global Tiempo libre de progresión Tiempo hasta primer AAG (tras la administración del tratamiento en estudio) Evaluación de parámetros inmunológicos Evaluación de cambios radiológicos Relación de la presencia de respuesta inmunológica con evolución clínica. Relación entre la caracterización histológica y molecular, la respuesta clínica y la respuesta celular Evaluación de parámetros de seguridad Evaluación del performance |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | No |
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 | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | Yes |
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) | 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.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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
|
LVLS |
última visita de último paciente |
|
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 |