Sirolimus for Diffuse intrinsic pontine glioma

Inclusion criteria

• {"criterion_text":"- Age 3 to 18 years, inclusive\n- Informed consent signed by parents or legal guardian of minor and by patient over 13 years of age) for the study diagnostic and therapeutic procedures, including molecular -based diagnostic and prognostic tests using NGS method\n- Diagnosis of diffuse intrinsic pontine glioma (DIPG) based on clinical and radiological criteria\n- Histological diagnosis of glioma WHO grade III and IV confirmed by reference pathologist"}

Exclusion criteria

• {"criterion_text":"- Any severe comorbid disease (eg kidney insufficiency, immunological deficiencies, HIV infection)\n- Clinically significant cardiovascular condition ( eg arrythmia)\n- Previous (< 5 yrs ) diagnosis of malignant neoplasm\n- Active infection requiring systemic treatment\n- Known allergy to the studied products\n- Concomitant use of CYP3A or PgP inhibitors\n- Radiological evidence of active intracranial hemorrhage (excluding receding post biopsy or focal bleeding)\n- Major surgery in the past 28 days"}

Primary endpoints

• {"endpoint_text":"- Overall survival (OS) from the time of diagnosis until last visit or death","definition_or_measurement_approach":"Overall survival measured from time of diagnosis until last visit or death (as stated)"}
• {"endpoint_text":"- Safety assessment of sirolimus administered during radiotherapy and in combination with trametinib as adjunctive treatment after irradiation","definition_or_measurement_approach":"Safety assessment as stated in endpoint (specific safety measurement approach not detailed in available data)"}

Secondary endpoints

• {"endpoint_text":"- Progression free survival from diagnosis to progression","definition_or_measurement_approach":"Progression-free survival measured from diagnosis to documented progression (as stated)"}
• {"endpoint_text":"- Response rate assessed at time points specified in the protocol (MRI every 2 months from commnecement of systemic treatment)","definition_or_measurement_approach":"Response rate assessed at protocol-specified time points; MRI every 2 months from start of systemic treatment (as stated)"}
• {"endpoint_text":"- Quality of life assessment (using PedQoL- pediatric quality of life inventory) at time points specified in the protocol","definition_or_measurement_approach":"Quality of life measured using PedQoL pediatric quality of life inventory at protocol-specified time points (as stated)"}
• {"endpoint_text":"- Comparison of safety in 2 groups of patients; treated with sirolimus alone or in combination with trametinib","definition_or_measurement_approach":"Comparison of safety between patients treated with sirolimus alone versus sirolimus plus trametinib (specific safety endpoints/measures not detailed in available data)"}

Poland

Earliest CTIS Part Ii Submission Date

08-11-2024

Latest Decision Or Authorization Date

18-12-2024

Processing Time Days

40

Number Of Sites

2

Number Of Participants

100

Primary sponsor

Full Name

Instytut Pomnik Centrum Zdrowia Dziecka

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Poland

Third parties

• {"country":"Poland","full_name":"Warszawski Uniwersytet Medyczny","duties_or_roles":"code 4","organisation_type":"Educational Institution"}
• {"country":"Poland","full_name":"Neuca S.A.","duties_or_roles":"Labelling","organisation_type":"Pharmaceutical company"}
• {"country":"Poland","full_name":"Cefea Sp. z o.o. S.K.","duties_or_roles":"Labelling","organisation_type":"Pharmaceutical company"}