E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
B-cell malignancies and Acute Myeloid Leukemia |
Neoplasie a cellule B e leucemia mieloide acuta |
|
E.1.1.1 | Medical condition in easily understood language |
Cancers of white blood cells; Cancer of the blood and bone marrow |
Tumori dei globuli bianchi; Cancro del sangue e del midollo osseo |
|
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 |
- Valutare l’incidenza e la gravità degli eventi avversi (EA)/eventi avversi gravi (SAE) ad insorgenza tardiva mirati, che si sospetta possano essere correlati alle cellule geneticamente modificate, tra cui disturbi neurologici, disturbi autoimmuni, disturbi ematologici, infezioni gravi e neoplasie secondarie. - Valutare il meccanismo di retrovirus/lentivirus replicazione-competenti (RCR/RCL) e/o la mutagenesi inserzionale per gli eventi confermati e correlati al prodotto per la terapia cellulare - Valutare la crescita, lo sviluppo e la maturità sessuale dei soggetti pediatrici e adolescenti trattati con cellule geneticamente modificate |
|
E.2.2 | Secondary objectives of the trial |
- Determine the time to next treatment after administration of genemodified cell therapy in the completed parent study - Determine survival status - Determine cause of death - Evaluate immune reconstitution - Evaluate the incidence of RCR/RCL |
- Determinare il tempo al trattamento successivo dopo la somministrazione della terapia a base di cellule geneticamente modificate nell’ambito dello studio originario completato - Determinare lo stato di sopravvivenza - Determinare la causa del decesso - Valutare la ricostituzione immunitaria - Valutare l’incidenza di retrovirus/lentivirus replicazione-competenti (RCR/RCL) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) 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 2) The subject must understand and voluntarily sign an Informed Consent Form (ICF) or an Informed Assent Form prior to any studyrelated assessments or procedures being conducted 3) 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 |
1) Il soggetto deve avere ricevuto un’infusione di cellule geneticamente modificate nell’ambito di uno studio originario completato sponsorizzato da Kite, non ha ritirato il pieno consenso e deve aver interrotto o completato il periodo di follow-up post-trattamento nello studio di originario,, a seconda dei casi. 2) Il soggetto deve comprendere e firmare volontariamente un modulo di consenso informato e/o un modulo di assenso informato prima che sia condotta qualsiasi valutazione/procedura correlata allo studio. 3) A giudizio dello sperimentatore, il soggetto è disposto ed in grado di completare il programma di follow-up richiesto dal protocollo e di rispettare i requisiti per la partecipazione previsti dallo studio. |
|
E.4 | Principal exclusion criteria |
There are no specific exclusion criteria for this study. |
Non ci sono criteri di esclusione specifici per questo studio |
|
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 |
- Valutare l’insorgenza dei seguenti AE/SAE ad insorgenza tardiva mirati, che si sospetta possano essere correlati alle cellule geneticamente modificate: - Disturbi neurologici - Disturbi autoimmuni - Disturbi ematologici - Infezioni gravi - Neoplasie secondarie - Meccanismo di RCR/RCL e/o mutagenesi inserzionale - Altezza, peso e maturazione sessuale dei soggetti pediatrici e adolescenti |
|
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. |
Approssimativamente ogni 3 mesi dal Mese 6 al Mese 18 dopo l'infusione iniziale nello studio sui genitori, Mese 24 e poi annualmente. |
|
E.5.2 | Secondary end point(s) |
- Subsequent anticancer therapies - Survival status - Cause of death - Evidence of immune reconstitution - Rates of RCR/RCL |
- Terapie antitumorali successive - Stato di sopravvivenza - Causa del decesso - Evidenza di ricostituzione immunitaria - Tasso di RCR/RCL |
|
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. |
Approssimativamente ogni 3 mesi dal Mese 6 al Mese 18 dopo l'infusione iniziale nello studio sui genitori, Mese 24 e poi annualmente. |
|
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 |
Follow up di sicurezza a lungo termine |
|
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.8.2.4 | Number of treatment arms in the trial | 1 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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 | Information not present in EudraCT |
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 |
Belgium |
France |
Germany |
Italy |
Netherlands |
Poland |
Spain |
Sweden |
Switzerland |
United Kingdom |
Czechia |
|
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. |
La fine dello studio è definita come il momento in cui l'ultimo soggetto completa l'ultima visita di studio LTFU, o è considerato perso al follow-up, revoca il consenso o muore. |
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 15 |
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 |