ETOPOSIDE for Medulloblastoma (metastatic) | Embryonal central nervous system tumours (including pinealoblastoma, medulloepithelioma, CNS neuroblastoma, ganglioneuroblastoma)

Inclusion criteria

• {"criterion_text":"- First diagnosed, previously untreated, patients with high-risk medulloblastoma (according to WHO 2016 classification): metastatic; anaplastic or large cell; partially resected (residual volume on two floors> 1.5cm2) according to WHO classification (2016); N-MYC C-MYC amplified\n- Written informed consent obtained from the patient/parents or legal representative\n- Availability for treatment and ability to be compliant with the protocol\n- Patients with other embryonic tumors (WHO classification 2016) - embryonic tumor with C19MC-altered multilayer rosettes; embryonic tumor with multilayer rosettes not otherwise specified, medulloepithelioma, CNS neuroblastoma, CNS ganglioneuroblastoma, CNS embryonic tumor not otherwise specified, CNS embryonic tumor with rhabdoid features - of first diagnosis, previously untreated (with chemo and radiotherapy) and treated only surgically\n- First diagnosed Pinealoblastomas (WHO 2016), previously untreated (with chemo and radiotherapy) and treated only surgically\n- Males and females aged >3 years and <21 years at time of the diagnosis\n- Life expectancy > or = 12 months\n- Karnofsky/Lansky > or = 40 %\n- Adequate hematological function (leucocyte > or = 2.0 x 10^9/l - Hemoglobin > or = 10 g/dl - platelet > or = 50 x 10^9/l)\n- Adequate liver function (total bilirubin < or = 2.5 x ULN - ALT/AST < or = 5.0 x ULN)\n- Adequate renal function (serum creatinine < or = 1.5 x ULN)"}

Exclusion criteria

• {"criterion_text":"- Any disease or condition that contraindicates the use of the study treatment (es. serious mental retardation, brain palsy, congenital syndrome, cardiomyopathy, psychosocial problems)\n- Other contraindications reported in the SmPC\n- Administration of a first-line chemotherapy cycle at the same time as the start of the study\n- Concomitant partecipation to other clinical trial\n- Pregnancy or breastfeeding\n- Inadequate contraception by patients of both sexes\n- Hypersensibility to active principles or excipients\n- Severe bone-marrow depression\n- Concomitant administration of live attenuated vaccines\n- Inability to comply for study follow-up"}

Primary endpoints

• {"endpoint_text":"- Percentage of subjects with neurological symptoms >=G3, evaluated according to CTCAE 4.0, not present at the beginning of postoperative chemoradiotherapy and not related to the recurrence / progression of the disease - Percentage of subjects with SAE - Percentage of subjects with SAE leading to withdrawal from the study - Mortality due to adverse events","definition_or_measurement_approach":"Neurological symptoms graded by CTCAE v4.0; Serious adverse events (SAE) recorded and classified; SAE leading to withdrawal and mortality due to adverse events tracked."}

Secondary endpoints

• {"endpoint_text":"- Progression free survival (PFS) defined as time frame between the date of the enrolment and the date tumour progression or death\n- Overall survival (OS) defined as time frame between the date of the enrolment and the death to any cause\n- Difference in scoring for neuropsychological (Griffiths-III, WPPSI-III, WISC-IV, WAIS-IV, Rey, Nepsy-2) and psycological (PedsQL, CBCL) scales\n- Rate of treatment response: CR, complete response the complete disappearance of all radiographic and/or cytological evidence of tumor; PR, partial response >= 50% reduction in the product of the perpendicular diameters of the tumor with negative cytology - SD, stable disease <= 25%reduction in tumor size; PD, progressive disease >= 25% increase in tumor size or the appearance of tumor in previously uninvolved areas\n- Frequency of endocrinological and visual sequelae","definition_or_measurement_approach":"PFS: time from enrolment to tumour progression or death; OS: time from enrolment to death from any cause; Neuropsychological and psychological outcomes measured by specified scales (Griffiths-III, WPPSI-III, WISC-IV, WAIS-IV, Rey, Nepsy-2, PedsQL, CBCL); Response rates defined with CR, PR, SD, PD criteria as specified; Endocrinological and visual sequelae frequency assessed clinically."}

Italy

Earliest CTIS Part Ii Submission Date

22-11-2024

Latest Decision Or Authorization Date

27-01-2025

Processing Time Days

66

Number Of Sites

1

Number Of Participants

18

Primary sponsor

Full Name

Azienda Ospedaliera Universitaria Meyer IRCCS

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Italy