E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Defined by parent protocol. The study will enroll all adult and paediatric subjects who received at least one genetically modified T cells infusion in a previous Celgene sponsored study |
Definito dal protocollo di trattamento principale. Lo studio arruoler¿ tutti i soggetti adulti e pediatrici che avranno ricevuto almeno un¿infusione di cellule T geneticamente modificate in un precedente studio sponsorizzato da Celgene |
|
E.1.1.1 | Medical condition in easily understood language |
Defined by parent protocol |
Definito dal protocollo principale |
|
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 | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10025631 |
E.1.2 | Term | Malignant lymphoid neoplasm NOS |
E.1.2 | System Organ Class | 100000004864 |
|
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 assess the risk of delayed adverse events (AEs) following exposure to gene-modified (GM) T cells - To monitor for long-term persistence of GM T cells, including analysis of vector integration sites, as appropriate. - To monitor for generation of replication competent retroviruses (RCR) - To assess long-term efficacy following treatment with GM T cells - Describe growth, developmental outcome, and sexual maturity status for subjects who were aged < 18 years at time of GM T cell treatment - To assess long term health-related quality of life following treatment with GM T cells |
- Valutare il rischio di eventi avversi (EA) ritardati in seguito all¿esposizione alle cellule T geneticamente modificate (GM) - Monitorare la persistenza a lungo termine delle cellule T GM, inclusa l¿analisi dei siti di integrazione del vettore, come opportuno. - Monitorare la generazione di retrovirus competenti per la replicazione (RCR) - Valutare l¿efficacia a lungo termine in seguito a trattamento con cellule T GM - Descrivere la crescita, l¿esito evolutivo e lo stato di maturit¿ sessuale dei soggetti di et¿ inferiore a 18 anni al momento del trattamento con cellule T GM - Valutare la qualit¿ della vita correlata alla salute nel lungo termine, in seguito a trattamento con cellule T GM
|
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. All adult and pediatric subjects who received at least one gene modified (GM) T cell infusion in a previous Celgene sponsored or Celgene alliance partner sponsored study, and have discontinued, or completed the post-treatment follow-up period in the parent treatment protocol, as applicable. 2. Subject (and, parental/legal representative, when applicable) must understand and voluntarily sign an ICF/IAF prior to any study-related assessments/procedures being conducted. 3. Subject is willing and able to adhere to the study visit schedule and other protocol requirements. |
1. Tutti i soggetti adulti e pediatrici che hanno ricevuto almeno un’infusione di cellule T geneticamente modificate in un precedente studio sponsorizzato da Celgene o da un partner di alleanza di Celgene, e che hanno interrotto o che hanno completato il periodo di follow-up post-trattamento nel protocollo di trattamento principale, a seconda dei casi. 2. Il soggetto (e il rappresentante genitoriale/legale, se del caso) deve comprendere e firmare volontariamente un modulo ICF/IAF prima che sia condotta alcuna valutazione/procedura correlata allo studio. 3. Il soggetto è disposto e in grado di aderire al piano di visite dello studio e ad altri requisiti del protocollo.
|
|
E.4 | Principal exclusion criteria |
Not applicable |
Non applicabile |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Safety: - Incidence of delayed Adverse Events suspected to be related to prior gene-modified (GM) T cell therapy, including: - New malignancies (hematologic or solid) - New neurologic disorder, or exacerbation of a pre-existing neurologic disorder - New rheumatologic or autoimmune disorder, or exacerbation of a prior rheumatologic or other autoimmune disorder - New hematologic disorder - Other new clinical conditions considered related to the prior GM T cell therapy by the investigator. Hospitalizations, regardless of relationship to prior treatment, including reasons and dates - Persistence of GM T cells - Analysis of vector integration sites - Incidence of replication-competent retroviruses (RCR) - Height, weight, growth, and organ development will be assessed for all pediatric subjects - Sexual maturity status for pediatric subjects Efficacy Where applicable: - Tumor Response Status - Date of Disease Progression - Date of Relapse - Survival Status HRQoL Measurement of health-related quality of life (HRQoL) changes as assessed using instruments administered in the parent treatment protocol |
Sicurezza: - Incidenza degli eventi avversi ritardati sospettati di essere correlati alla terapia con cellule T geneticamente modificate (GM), compresi: - Nuove neoplasie (ematologiche o solide) - Nuovi disturbi neurologici, o esacerbazione di un disturbo neurologico preesistente - Nuovo disturbo reumatologico o autoimmune, o esacerbazione di un disturbo reumatologico precedente o altro disturbo autoimmune - Nuovo disturbo ematologico - Altre condizioni cliniche nuove considerate dallo sperimentatore correlate alla precedente terapia con cellule T GM. Ricoveri, a prescindere dal rapporto con il trattamento precedente, inclusi i motivi del ricovero e le date
- Persistenza delle cellule T GM - Analisi dei siti di integrazione del vettore - Incidenza dei retrovirus competenti per la replicazione (RCR) - Saranno valutati l’altezza, il peso, la crescita e lo sviluppo dell’organo in tutti i soggetti pediatrici - Stato di maturità sessuale dei soggetti pediatrici
Efficacia Se pertinente: - Stato di risposta del tumore - Data della progressione della malattia - Data della ricaduta - Stato di sopravvivenza
HRQoL Misurazione dei cambiamenti della qualità della vita correlati alla salute (HRQoL) valutati mediante strumenti somministrati nel protocollo di trattamento principale
|
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
HRQoL: Up to 5 years from last GM T cells infusion Other endpoints: Up to 15 years from last GM T cells infusion |
HRQoL: Fino a 5 anni dall’ultima infusione di cellule T GM Altri endpoint: fino a 15 anni dall’ultima infusione di cellule T GM
|
|
E.5.2 | Secondary end point(s) |
Not applicable |
Non applicabile |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Not applicable |
Non applicabile |
|
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 | 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 |
Health-related quality of life (HRQoL) |
Qualit¿ della vita correlata alla salute (HRQoL) |
|
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 | Yes |
E.8.1.7.1 | Other trial design description |
Protocollo di follow-up a lungo termine |
Long-Term Follow-up Protocol |
|
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 | 57 |
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 |
Canada |
Japan |
United States |
Austria |
Belgium |
Finland |
France |
Germany |
Italy |
Netherlands |
Norway |
Spain |
Sweden |
Switzerland |
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
|
The End of Trial is defined as either the date of the last visit of the last subject or the date of receipt of the last data point from the last subject that is required for primary analysis, as pre-specified in the protocol, whichever is the later date |
Il termine dello studio ¿ definito come la data dell¿ultima visita dell¿ultimo soggetto oppure la data di ricevimento dell¿ultimo punto dati dell¿ultimo soggetto, richiesto per l¿analisi primaria, come predefinito nel protocollo, qualunque sia la data pi¿ lontana |
|
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 |