E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
patients with advanced and non-resectable sarcoma |
pazienti con diagnosi di sarcoma avanzato, inoperabile |
|
E.1.1.1 | Medical condition in easily understood language |
advanced and non-resectable sarcomas |
sarcomi avanzati, inoperabili |
|
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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10039491 |
E.1.2 | Term | Sarcoma |
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 |
To determine the maximum tolerated dose (MTD) of CIK |
Identificare la massima dose tollerata (MTD) delle CIK autologhe |
|
E.2.2 | Secondary objectives of the trial |
-Recommended Phase 2 dose (RP2D) -To characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of autologous CIK cells -To explore preliminary evidence of anti-tumour activity of CIK cells -To identify potential (predictive/prognostic) relationships between several bio-markers in the tumor tissue and the observed results; -To evaluate the quality of life of enrolled patients. |
-Valutazione della dose raccomandata per la Fase II (RP2D) -Analisi di farmacocinetica (PK) e farmacodinamica (PD) delle cellule CIK -Valutazione preliminare di attività antitumorale delle cellule CIK -Valutazione di potenziali fattori bioumorali predittivi/prognostici del paziente e/o delle caratteristiche biologiche del tumore -Valutazione della qualità di vita dei pazienti |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients with histologically documented unresectable sarcomas which progressed after the first or further line treatments for relapse disease Age >/= 10 years and </= 25 years Karnofsky or Lansky >/= 70% (patients who present a Karnofsky or Lansky >/= 50% are eligible if it depends solely on orthopedic problems) or ECOG </= 1 (patients who ECOG = 2 are eligible if it depends solely on orthopedic problems) Estimated life expectancy > 3 months; Signed informed consent There are also specific additional criteria for the two phases of the study. |
Pazienti con diagnosi istologica documentata di sarcoma, non operabile in modo completo, in recidiva e/o progressione di malattia dopo prima o successive linee di trattamento standard; Età >/= 10 anni e </= 25 anni Karnofsky o Lansky >/= 70% (possono essere arruolati i pazienti che presentino un Karnofsky o Lansky >/= 50% solo per la presenza di problemi ortopedici) oppure ECOG </= 1 (possono essere arruolati i pazienti che presentino ECOG = 2 solo per la presenza di problemi ortopedici) Aspettativa di vita > 3 mesi; Avvenuta firma del consenso informato da parte del paziente e/o dei genitori/tutore legale. Esistono inoltre criteri aggiuntivi specifici per le due parti dello Studio. |
|
E.4 | Principal exclusion criteria |
Patients with neuro-psychiatric disorders or social or geographic problems that would prohibit the understanding or rendering informed consent and compliance with the requirements of this protocol are excluded. There are also specific additional criteria for the two phases of the study. |
Disturbi neuro-psichiatrici, sociali, problematiche geografiche o familiari gravi che rendano difficoltosa la partecipazione del paziente allo Studio. Esistono inoltre criteri aggiuntivi specifici per le due parti dello Studio. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of Dose Limiting Toxicities (DLTs) |
Valutazione dell’incidenza di tossicità limitanti la dose (DLTs) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
during the first two cycles of treatment (6 weeks fos dose levels -1, 1 and 2; 4 weeks for dose levels 3 and 4) |
durante i primi due cicli di trattamento (pari a 6 seetimane per i livelli di dose -1, 1 e 2; pari a 4 settimane per i livelli di dose 3 e 4) |
|
E.5.2 | Secondary end point(s) |
pharmacokinetics and pharmacodynamics; PFS; MIC A/B and NKGD2 evaluation |
- farmacocinetica e farmacodinamica; - PFS; - valutazione MIC A/B e NKGD2 |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
24 months; 36 months; 24 months |
nei primi 24 mesi dello studio.; 36 mesi; nei primi 24 mesi |
|
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 | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
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 | 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 |
dose finding |
dose finding |
|
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 | 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 | Information not present in EudraCT |
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
|
|
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 | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |