NAXITAMAB for Ewing's sarcoma

Inclusion criteria

• {"criterion_text":"- Histologically proven Ewing sarcoma of the bone or soft tissues."}
• {"criterion_text":"- Consent to the use of effective contraception throughout the period of the study and a minimum of 1 year after discontinuation of study treatment in patients at puberty and sexual maturity."}
• {"criterion_text":"- Subject's archival tumour sample (formalin-fixed, paraffinembedded; FFPE) available for evaluation of GD2 expression."}
• {"criterion_text":"- Documented disease progression (during or after completion of at least one line treatment) or any subsequent recurrence."}
• {"criterion_text":"- GD2 positive tumor assessed by IHC."}
• {"criterion_text":"- Age ≥ 2 years and ≤ 21 years."}
• {"criterion_text":"- Life expectancy of at least 12 weeks from the time informed consent was signed."}
• {"criterion_text":"- Previous systemic anticancer treatment completed ≥ 3 weeks, major surgery ≥ 2 weeks, and radiation therapy ≥ 4 weeks prior to study enrollment."}
• {"criterion_text":"- Recovered from adverse effects of prior surgery, radiotherapy, or anti-neoplastic therapy at the discretion of the investigator."}
• {"criterion_text":"- Signing of informed consent for trial participation (including for naxitamab treatment) according with current legal regulations."}

Exclusion criteria

• {"criterion_text":"- Failure to meet any of the inclusion criteria."}
• {"criterion_text":"- Requirement, or likely requirement, for corticosteroids at doses >10 mg prednisolone (or equivalent) per day or other immunosuppressive agents."}
• {"criterion_text":"- Diagnosis of other malignancies before study inclusion."}
• {"criterion_text":"- Planning to become pregnant (while being treated with IT or naxitamab), pregnancy or breastfeeding."}
• {"criterion_text":"- Other acute or persistent disorders, behaviors or abnormal laboratory test results, which might increase the risk related to the participation in this clinical trial or to taking the study drug, or which might influence the interpretation of the study results, or which, in the investigator's opinion, disqualify a patient from participating in the trial."}
• {"criterion_text":"- Not eligible to IT."}
• {"criterion_text":"- Previous treatment with an anti-GD2 antibody."}
• {"criterion_text":"- Hypersensitivity to the study drugs or any of their ingredients (covers IT and naxitamab)."}
• {"criterion_text":"- Simultaneous treatment with other drugs which might interact with naxitamab or IT regimen."}
• {"criterion_text":"- Persistent toxicity related to prior therapy, making it impossible to treat with naxitamab."}
• {"criterion_text":"- Significant cardiac conduction abnormalities, including known familial prolonged QT syndrome, or screening QTc >480 msec."}
• {"criterion_text":"- Symptoms of congestive heart failure or left ventricular ejection fraction <50%."}
• {"criterion_text":"- Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and/or chronic oxygen requirement. In addition, room air pulse oximetry < 94% and/or abnormal pulmonary function tests if these assessments are clinically indicated."}

Primary endpoints

• {"endpoint_text":"- Safety and tolerability will be assessed based on an analysis of adverse events on all enrolled subjects. These events will be divided according to severity, seriousness, affected organ or system and analyzed for:","definition_or_measurement_approach":"Assessment based on analysis of adverse events in all enrolled subjects, divided by severity, seriousness and system/organ."}
• {"endpoint_text":"- number of serious adverse events (SAE)","definition_or_measurement_approach":"Count of serious adverse events (SAE)."}
• {"endpoint_text":"- the number of adverse events (AE), including events of particular importance to the incidence and severity of adverse events occurring during treatment (TEAE) (encoded according to the preferred term and class of organ systems using the Medical Dictionary for Regulatory Activities (MedDRA); these events will be estimated according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v5.0)","definition_or_measurement_approach":"AE/TEAE encoded using MedDRA preferred terms and organ system classes; severity graded according to NCI CTCAE v5.0."}
• {"endpoint_text":"- the result of a medical examination with the analysis of recorded vital signs","definition_or_measurement_approach":"Medical examination results including analysis of recorded vital signs."}
• {"endpoint_text":"- assessment of laboratory abnormalities according to NCI CTCAE v5.0","definition_or_measurement_approach":"Laboratory abnormalities assessed and graded according to NCI CTCAE v5.0."}
• {"endpoint_text":"- This primary end point has been redacted following transparency rules and protection of confidential information.","definition_or_measurement_approach":"Redacted."}

Secondary endpoints

• {"endpoint_text":"- To assess the efficacy of the use of naxitamab in combination with standard IT chemotherapy versus chemotherapy alone:","definition_or_measurement_approach":"Comparison of naxitamab + standard IT chemotherapy versus standard chemotherapy alone (efficacy assessment)."}
• {"endpoint_text":"- EFS (Event-Free Survival) - from randomization to the date of disease progression, recurrence, second malignancy, death or to date of last follow-up for patients without events,","definition_or_measurement_approach":"EFS measured from randomization to progression, recurrence, second malignancy, death, or last follow-up."}
• {"endpoint_text":"- PFS (Progression-Free Survival) - from randomization to progression of the disease,","definition_or_measurement_approach":"PFS measured from randomization to radiographic or clinical disease progression."}
• {"endpoint_text":"- ORR (Overall Response Rate) - defined as the proportion of patients with a best overall response of complete response (CR) or partial response (PR) according WHO criteria,","definition_or_measurement_approach":"ORR defined as proportion of patients achieving CR or PR per WHO criteria."}
• {"endpoint_text":"- OS (Overall Survival) - from randomization to subject's death.","definition_or_measurement_approach":"OS measured from randomization to death from any cause."}

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

24

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":"Masha Regulatory Service Anna Jelitto","duties_or_roles":"sponsorDuties codes: 1, 10, 12, 8","organisation_type":"Industry"}