ETOPOSIDE for Retinoblastoma

Inclusion criteria

• {"criterion_text":"- Overall study inclusion criteria: Patients not previously treated by chemotherapy or radiotherapy for this tumour or another cancer."}
• {"criterion_text":"- Study 2 inclusion criteria: Patients affected by bilateral retinoblastoma very asymmetric with a group D eye that can be treated by intraarterial chemotherapy by Melphalan and the other amenable to local treatments without chemotherapy."}
• {"criterion_text":"- Study 2 inclusion criteria: Children from 6 months to 6 years old."}
• {"criterion_text":"- Study 3 inclusion criteria: Children affected of bilateral group D retinoblastoma or on the only eye amenable to conservative treatment."}
• {"criterion_text":"- Overall study inclusion criteria: Possible long term follow-up."}
• {"criterion_text":"- Overall study inclusion criteria: Written informed consent of the parents or the legal representative."}
• {"criterion_text":"- Overall study inclusion criteria: Patients having social security cover."}
• {"criterion_text":"- Overall study inclusion criteria: No contra-indications to the study treatments."}
• {"criterion_text":"- Studies 1 and 3 inclusion criteria : Children from 0 to 6 years old."}
• {"criterion_text":"- Study 1 inclusion criteria :\tPatients affected by unilateral retinoblastoma groups A, B (according to the age), group C (according to the age and vitreous seeding), or bilateral retinoblastoma groups A, B, C (excluding the eyes with macular threat and bilateral group D eyes or on the only remaining eye) amenable to a conservative treatment (at least on one eye in bilateral disease) but needing initial chemotherapy because of the location, the size of the lesion (more than 4 mm of diameter), a vision threat or risks of intravitreal relapse making those patients not amenable to chemothermotherapy first line."}
• {"criterion_text":"- Study 1 inclusion criteria : Patients less than six months of age with unilateral retinoblastoma groups B, C, D, or bilateral very asymmetric with one eye group D and the other amenable to local treatments without chemotherapy."}
• {"criterion_text":"- Study 2 inclusion criteria: Patients affected by unilateral or bilateral retinoblastoma group B (according to the age), group C (according to the age and vitreous seeding), or group D."}
• {"criterion_text":"- Overall study inclusion criteria: Patients not previously treated by chemotherapy or radiotherapy for this tumour or another cancer."}

Exclusion criteria

• {"criterion_text":"- Overall study non-inclusion criteria: Patients older than 6 years old."}
• {"criterion_text":"- Study 1 non-inclusion criteria: Patients for whom a local treatment is possible without initial chemotherapy (tumour smaller than 4 mm and located far from optic nerve head or macula)."}
• {"criterion_text":"- Study 1 non-inclusion criteria: Patients with an unilateral group D with massive tumour or group E eyes needing enucleation first line or after initial chemotherapy (in case of buphthalmia or suspected optic nerve invasion or extrascleral extension)."}
• {"criterion_text":"- Study 1 non-inclusion criteria: Patients affected by bilateral retinoblastoma very asymmetric with a group D eye that can be treated by intraarterial chemotherapy by Melphalan and the other amenable to local treatments without chemotherapy."}
• {"criterion_text":"- Study 1 non-inclusion criteria: Patients with bilateral retinoblastoma and bilateral group D eyes or on the only remaining eye or presenting a bilateral macular threat requiring conservative treatment by a 6 cycles, three drugs regimen."}
• {"criterion_text":"- Study 2 non-inclusion criteria: Patients with a unilateral group D (extensive) or B or C or D but covering the optic nerve head or group E eyes for which enucleation is warranted first line or after chemotherapy (in case of buphthalmia or suspected optic nerve invasion or extrascleral extension)."}
• {"criterion_text":"- Study 2 non-inclusion criteria: Patients with unilateral group D eye with tumour volume of more than 50% of eye volume, for whom a massive choroidal invasion could be associated (on clinical or imaging criteria) and for which enucleation is warranted."}
• {"criterion_text":"- Study 3 non-inclusion criteria: Patients for whom a local treatment is possible without chemoreduction (tumour smaller than 4 mm, distant from macula and from optic nerve head)."}
• {"criterion_text":"- Study 3 non-inclusion criteria: Patients affected by bilateral retinoblastoma very asymmetric with a group D eye that can be treated by intraarterial chemotherapy by Melphalan and the other amenable to local treatments without chemotherapy."}
• {"criterion_text":"- Study 3 non-inclusion criteria: Patients with bilateral retinoblastoma without macular threat or groups A, B, C than can be treated with chemoreduction by VP16 and Carboplatin then chemothermotherapy without laser treatment at day 8."}
• {"criterion_text":"- Overall study non-inclusion criteria: Patients with extraocular retinoblastoma."}
• {"criterion_text":"- Overall study non-inclusion criteria: Patients with a disease being a contra-indication to chemotherapy."}
• {"criterion_text":"- Overall study non-inclusion criteria: Patients anteriorly treated by chemotherapy."}
• {"criterion_text":"- Overall study non-inclusion criteria: Patients anteriorly treated by external beam irradiation."}
• {"criterion_text":"- Overall study non-inclusion criteria: Patients anteriorly treated for another cancer."}
• {"criterion_text":"- Overall study non-inclusion criteria: Follow-up not possible due to geographic distance from the center or for social or psychological reasons.."}
• {"criterion_text":"- Overall study non-inclusion criteria: Parents not having accepted the therapeutic strategy after explanations by the investigator."}
• {"criterion_text":"- Overall study non-inclusion criteria: Contra-indication to the use of one of the drugs used in the study."}

Primary endpoints

• {"endpoint_text":"- For the 3 studies: ocular preservation (absence of secondary enucleation) and absence of external beam radiotherapy at 18 months.","definition_or_measurement_approach":"Ocular preservation defined as absence of secondary enucleation and absence of external beam radiotherapy assessed at 18 months follow-up."}

Secondary endpoints

• {"endpoint_text":"- Evaluation or the risk of relapse by fundus examination under general anaesthesia until the age of 4 then without general anaesthesia.","definition_or_measurement_approach":"Tumor relapse assessed prospectively by fundus examination under general anaesthesia until age 4, thereafter by fundus exam without general anaesthesia."}
• {"endpoint_text":"- Prospective evaluation of the ocular and general side effects (short, medium and long term) of the intravenous chemotherapy, intra-arterial chemotherapy, combined to the local treatment as well as the intra-vitreal injections of melphalan.","definition_or_measurement_approach":"Prospective collection and evaluation of ocular and systemic adverse events across short-, medium- and long-term follow-up for intravenous, intra-arterial, combined local treatments and intravitreal melphalan."}
• {"endpoint_text":"- Evaluation of the response to intra-vitreal chemotherapy by melphalan.","definition_or_measurement_approach":"Response to intravitreal melphalan measured clinically by ocular/tumour response assessments after intravitreal injections."}
• {"endpoint_text":"- Study of the radiation doses received during intra-arterial procedures.","definition_or_measurement_approach":"Dosimetric measurement of radiation exposure (scopy) during intra-arterial procedures."}
• {"endpoint_text":"- Evaluate the number of patients presenting with another tumour.","definition_or_measurement_approach":"Count and report incidence of second primary tumours during long-term follow-up."}

France

Earliest CTIS Part Ii Submission Date

13-08-2024

Latest Decision Or Authorization Date

05-09-2024

Processing Time Days

23

Number Of Sites

27

Number Of Participants

133

Primary sponsor

Full Name

Institut Curie

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France