Regorafenib for Ewing's sarcoma | Osteosarcoma | Refractory primary bone tumors

Inclusion criteria

• {"criterion_text":"- Age> 9 years and ≤ 21 years at the time of inclusion for the study."}
• {"criterion_text":"- Ewing's sarcoma or osteosarcoma confirmed by histopathological examination and the tests performed so far."}
• {"criterion_text":"- Treatment failure identified no earlier than 30 days prior to study treatment initiation (at least one subsection must be met for the patient to meet this criterion): a. progression on treatment of I or another line or b. relapse."}
• {"criterion_text":"- Giving written, informed consent to participate in the study prior to the commencement of the procedures included in the study protocol, including treatment with regorafenib in accordance with the current legal regulations."}
• {"criterion_text":"- Life expectancy of at least 12 weeks from signing the informed consent."}
• {"criterion_text":"- Possibility of swallowing the tablet."}
• {"criterion_text":"- Consent to use effective contraception throughout the period of regorafenib treatment and at least 2 years after its discontinuation in patients in puberty."}

Exclusion criteria

• {"criterion_text":"- Failure to meet any of the inclusion criteria."}
• {"criterion_text":"- Patients with heart defects and / or cardiac arrhythmias requiring permanent treatment with antiarrhythmic drugs."}
• {"criterion_text":"- Other acute or chronic medical conditions, behaviors, or abnormal laboratory values that may increase the risk of participating in this clinical trial or taking the study medication, or may affect the interpretation of the study results, or, in the investigator's opinion, may cause that the patient should not be enrolled in the study."}
• {"criterion_text":"- Prior treatment with regorafenib."}
• {"criterion_text":"- Pregnancy and breastfeeding."}
• {"criterion_text":"- Known hypersensitivity to the drug or any of its ingredients."}
• {"criterion_text":"- Taking medications that cannot be used while under regorafenib treatment."}
• {"criterion_text":"- Persistent toxicity related to previous therapy, preventing drug incorporation."}
• {"criterion_text":"- Diagnosis of other neoplastic disease prior to inclusion in the study."}
• {"criterion_text":"- Patients with uncontrolled hypertension."}
• {"criterion_text":"- Patients with diseases related to the coagulation disorders."}

Primary endpoints

• {"endpoint_text":"- EFS - (Event-Free Survival) event-free survival - will be measured from randomization to the occurrence of: death, assertion of disease progression or recurrence, finding a secondary neoplasm.","definition_or_measurement_approach":"Measured from randomization to occurrence of death, documented disease progression or recurrence, or diagnosis of a secondary neoplasm."}
• {"endpoint_text":"- Determination of the dose of the test substance in patients between 9 and 18 years at which exposure to the drug similar to that recommended for adults will be achieved.","definition_or_measurement_approach":"Determination of dose in patients aged 9–18 years that achieves drug exposure similar to adult recommended exposure (pharmacokinetic based dose determination)."}
• {"endpoint_text":"- To evaluate the safety of regorafenib by analyzing adverse events (AEs) including adverse events of special importance.","definition_or_measurement_approach":"Analysis of adverse events (AEs), including AEs of special importance, to evaluate safety."}
• {"endpoint_text":"- Assessment of the safety of regorafenib through the analysis of recorded vital signs, laboratory test results, echocardiography, and ECG.","definition_or_measurement_approach":"Safety assessment via analysis of recorded vital signs, laboratory results, echocardiography and ECG data."}

Secondary endpoints

• {"endpoint_text":"- PFS (Progression-Free Survival) - progression-free survival - will be measured from randomization to finding disease progression in imaging studies.","definition_or_measurement_approach":"Measured from randomization to documented disease progression on imaging."}
• {"endpoint_text":"- OS (Overall Survival) - will be measured from randomization to death due to neoplastic disease.","definition_or_measurement_approach":"Measured from randomization to death due to neoplastic disease."}
• {"endpoint_text":"- ORR (Overall Response Rate) - Percentage of patients who achieved a protocol-defined response to treatment.","definition_or_measurement_approach":"Proportion of patients achieving protocol-defined response to treatment."}
• {"endpoint_text":"- Time to reach the target serum concentration of the test substance.","definition_or_measurement_approach":"Time to reach target serum concentration (pharmacokinetic measurement)."}
• {"endpoint_text":"- Maximum serum concentration at steady state Cmaxs.","definition_or_measurement_approach":"Measurement of maximum serum concentration at steady state (Cmaxs)."}
• {"endpoint_text":"- Steady-state trough serum concentration Cminss.","definition_or_measurement_approach":"Measurement of trough serum concentration at steady state (Cminss)."}
• {"endpoint_text":"- Casual steady-state serum concentration Css.","definition_or_measurement_approach":"Measurement of casual/spot serum concentration at steady state (Css)."}
• {"endpoint_text":"- Exposure to Ctau.","definition_or_measurement_approach":"Measurement of exposure metric Ctau (pharmacokinetic exposure)."}
• {"endpoint_text":"- Time to steady-state concentration of the test substance in the serum.","definition_or_measurement_approach":"Time from dosing to achievement of steady-state serum concentration."}

Methods

• Cooperation with patient organization Stowarzyszenie Pomocy Chorym na Mięsaki i Czerniaki Sarcoma to accelerate recruitment in Poland (patient-organization supported recruitment).

Poland

Earliest CTIS Part Ii Submission Date

24-07-2024

Latest Decision Or Authorization Date

06-08-2024

Processing Time Days

13

Number Of Sites

2

Number Of Participants

30

Primary sponsor

Full Name

Instytut Matki I Dziecka

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Poland

Third parties

• {"country":"Poland","full_name":"Narodowy Instytut Onkologii Im. Marii Sklodowskiej-Curie Panstwowy Instytut Badawczy","duties_or_roles":"NGS genetic tests","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Poland","full_name":"Masha Regulatory Service Anna Jelitto","duties_or_roles":"Project Management (codes: 1,12,15,8)","organisation_type":"Industry"}
• {"country":"Poland","full_name":"Sieć Badawcza Łukasiewicz – Instytut Chemii Przemysłowej im. Prof. Ignacego Mościckiego","duties_or_roles":"pharmacokinetics; study support (code: 4)","organisation_type":"Industry"}
• {"country":"Poland","full_name":"Stowarzyszenie Pomocy Chorym na Mięsaki i Czerniaki Sarcoma","duties_or_roles":"patients organization - cooperation within the acceleration of recruitment","organisation_type":"Health care"}
• {"country":"Poland","full_name":"Uniwersytet Przyrodniczy we Wrocławiu","duties_or_roles":"tumor cells culture","organisation_type":"Educational Institution"}