E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
B-cell malignancies and Acute Myeloid Leukemia |
Neoplasias de células B y leucemia mieloide aguda |
|
E.1.1.1 | Medical condition in easily understood language |
Cancers of white blood cells; Cancer of the blood and bone marrow |
|
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 | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10026801 |
E.1.2 | Term | Mantle cell lymphoma refractory |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10008977 |
E.1.2 | Term | Chronic lymphocytic leukemia recurrent |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10008978 |
E.1.2 | Term | Chronic lymphocytic leukemia refractory |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 23.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10065856 |
E.1.2 | Term | Non-Hodgkin's lymphoma unspecified histology indolent |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10012822 |
E.1.2 | Term | Diffuse large B-cell lymphoma refractory |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10060558 |
E.1.2 | Term | Acute myeloid leukemia recurrent |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10081514 |
E.1.2 | Term | Acute myeloid leukemia refractory |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10063621 |
E.1.2 | Term | Acute lymphoblastic leukaemia recurrent |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10006599 |
E.1.2 | Term | Burkitt's lymphoma refractory |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10036714 |
E.1.2 | Term | Primary mediastinal large B-cell lymphoma refractory |
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 | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Evaluate the incidence and severity of late-onset targeted adverse events (AEs)/serious adverse events (SAEs) suspected to be possibly related to gene-modified cells, including neurologic disorders, autoimmune disorders, hematologic disorders, serious infections, and secondary malignancies Evaluate mechanism of replication-competent retrovirus/replication competent lentivirus (RCR/RCL) and/or insertional mutagenesis for confirmed events related to the cell therapy product Evaluate the growth, development, and sexual maturity of pediatric and adolescent subjects treated with gene modified cells |
Evaluar la incidencia y la gravedad de los acontecimientos adversos (AA)/acontecimientos adversos graves (AAG) específicos de aparición tardía que se sospecha que pueden estar relacionados con las células genomodificadas, incluidos los trastornos neurológicos, autoinmunitarios y hemáticos, las infecciones graves y las neoplasias malignas secundarias Evaluar el mecanismo del retrovirus con capacidad de reproducirse/lentivirus con capacidad de reproducirse (RCR/LCR) o la mutagénesis insercional para los acontecimientos confirmados relacionados con el producto de terapia celular Evaluar el crecimiento, el desarrollo y la madurez sexual de los pacientes pediátricos y adolescentes tratados con células genomodificadas |
|
E.2.2 | Secondary objectives of the trial |
Determine the time to next treatment after administration of gene-modified cell therapy in the completed parent study Determine survival status Determine cause of death Evaluate immune reconstitution Evaluate the incidence of RCR/RCL |
Determinar el tiempo transcurrido hasta el siguiente tratamiento tras la administración de la terapia con células genomodificadas en el estudio principal completado Determinar el estado de supervivencia Determinar la causa de la muerte Evaluar la reconstitución inmunitaria Evaluar la incidencia del RCR/LCR |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
The subject must have received an infusion of gene-modified cells in a completed Kite sponsored parent study, has not withdrawn full consent, and has discontinued or completed the post-treatment follow-up period in the parent study, as applicable The subject must understand and voluntarily sign an Informed Consent Form (ICF) or an Informed Assent Form prior to any study-related assessments or procedures being conducted In the investigator’s judgment, the subject is willing and able to complete the protocol required follow-up schedule and comply with the study requirements for participation |
El sujeto debe haber recibido una infusión de células modificadas genéticamente en un estudio principal patrocinado por Kite, no debe haber retirado su pleno consentimiento y debe haber interrumpido o completado el periodo de seguimiento posterior al tratamiento en el estudio principal, según corresponda. El sujeto debe entender y firmar voluntariamente un Formulario de Consentimiento Informado (ICF) o un Formulario de Asentimiento Informado antes de que se lleve a cabo cualquier evaluación o procedimiento relacionado con el estudio. A juicio del investigador, el sujeto está dispuesto y es capaz de completar el programa de seguimiento requerido por el protocolo y cumplir con los requisitos de participación del estudio. |
|
E.4 | Principal exclusion criteria |
There are no specific exclusion criteria for this study. |
No existen criterios de exclusión específicos para este estudio. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Assess the occurrence of the following late-onset targeted AEs/SAEs suspected to be possibly related to gene-modified cells: Neurologic disorders Autoimmune disorders Hematologic disorders Serious infections Secondary malignancies Mechanism of RCR/RCL and/or insertional mutagenesis Height, weight, and sexual maturation of pediatric and adolescent subjects |
Evaluar los casos de los siguientes AEs/SAEs específicos de aparición tardía que se sospecha que pueden estar relacionados con las células genomodificadas: Trastornos neurológicos Trastornos autoinmunitarios Trastornos hemáticos Infecciones graves Neoplasias malignas secundarias El mecanismo del RCR/LCR o la mutagénesis insercional La altura, el peso y la madurez sexual de los pacientes pediátricos y adolescentes |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Approximately every 3 months from Month 6 to Month 18 after initial infusion in parent study, Month 24, and then annually. |
Aproximadamente cada 3 meses desde el Mes 6 hasta el Mes 18 después de la infusión inicial en el estudio principal, Mes 24, y después anualmente |
|
E.5.2 | Secondary end point(s) |
Subsequent anticancer therapies Survival status Cause of death Evidence of immune reconstitution Rates of RCR/RCL |
Tratamientos antineoplásicos posteriores Estado de supervivencia Causa de la muerte Indicios de reconstitución inmunitaria Tasas de RCR/LRC |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Approximately every 3 months from Month 6 to Month 18 after initial infusion in parent study, Month 24, and then annually. |
Aproximadamente cada 3 meses desde el Mes 6 hasta el Mes 18 después de la infusión inicial en el estudio principal, Mes 24, y después anualmente. |
|
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Long-term safety follow up |
Seguimiento de la seguridad a largo plazo |
|
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 23 |
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 |
Israel |
United States |
Austria |
France |
Poland |
Sweden |
Netherlands |
Spain |
Switzerland |
Czechia |
Germany |
Italy |
Belgium |
United Kingdom |
|
E.8.7 | Trial has a data monitoring committee | No |
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
|
End-of-study is defined as the time at which the last subject completes the last LTFU study visit, or is considered lost to follow-up, withdraws consent, or dies. |
El final del estudio se define como el momento en el que el último sujeto completa la última visita del estudio LTFU, o se considera perdido para el seguimiento, retira el consentimiento o fallece. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 15 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 15 |