💬In plain words
What the trial is, and whether it could suit you
Neuroblastoma (NB) is a malignant neoplasm of the sympathetic nervous system, occurring in 1 in 8,000 live births, accounting for 6-10% of all childhood malignant neoplasms and responsible for 12-15% of mortality -, making it the most common and life-threatening extracranial tumor in childhood.
Patients with stage 4 high-risk NB is the subgroup with the poorest prognosis. Within this group, two subgroups with an extremely unfavorable disease course are distinguished: patients with a poor response to the induction phase of therapy (refractory disease) and patients with relapsed or progressive disease.
Nowadays, 10-15% of patients show a poor end-induction response, whereas achieving a good end-induction response associated with better long-term survival. Improvement of the response to induction therapy may contribute to better treatment outcomes in newly diagnosed high-risk NB patients
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
Inclusion Criteria:
* Signed voluntary informed consent to participate in the clinical trial
* Histologically verified diagnosis of neuroblastoma or ganglioneuroblastoma
* Patients stratified to the high-risk group according to the criteria of the German Society of Pediatric Oncology and Hematology (GPOH) - NB 2004, aged from 18 months to 18 years, and meeting the following conditions:
1. Arm A: Refractory disease - patients who have completed the induction phase of therapy (6 cycles of N5/N6) with a poor response to therapy (MR, SD), with the exception of PD
2. Arm В: Relapsed/progressive disease - patients who develop any new tumor lesions (after having previously achieved СR), or any new tumor lesion; an increase of \>25% in any previously existing measurable lesion; or newly detected bone marrow involvement by NB cells in cases where the bone marrow had previously been free of involvement
* Performance status ≥ 70% (Lansky or Karnofsky scale) at the time of determining the indication for chemoimmunotherapy combined with NK cell therapy.
* Expected life expectancy ≥ 12 weeks.
* No signs of drug-induced neuropathy or neuropathic pain.
* Adequate liver function: alanine aminotransferase (ALT) / aspartate aminotransferase (AST) activity \< 5 × upper limit of normal (ULN).
* Adequate renal function: creatinine clearance or glomerular filtration rate (GFR) \> 60 mL/min/1.73 m².
* Coagulation parameters: prothrombin index (PTI) 70-120%; activated partial thromboplastin time (APTT) \< 36 seconds.
* No clinical signs of heart failure; left ventricular ejection fraction (LVEF) ≥ 55%.
* Adequate respiratory function (oxygen saturation by pulse oximetry \> 94% on room air, no dyspnea at rest), and no pathological findings on chest X-ray.
* Completion of comprehensive assessment to evaluate the extent of the tumor process.
Exclusion Criteria:
* Lack of a signed voluntary informed consent form for participation in the clinical study.
* Absence of comprehensive pre-treatment assessment results at the time of initiation of specific therapy.
* Patients with NBL or ganglioneuroblastoma stratified to low or intermediate-risk group
* Good response (PR, VGPR, CR) or PD at the end of the induction phase of therapy (applicable only to patients receiving therapy within the framework of the intensified induction phase).
* Progressive or relapsed disease with central nervous system involvement and/or leptomeningeal involvement.
* History of acute intolerance reactions to the main chemotherapeutic and immunobiological agents and supportive care drugs used in this clinical trial protocol.
* Presence of complications of the underlying disease and comorbidities that preclude treatment within this protocol, including severe type I hypersensitivity reactions in the medical history.
* Requirement for concomitant medications with known cross pharmacodynamic interactions with the drugs used in this clinical trial protocol.
* Presence of ultrasonographic signs of heart failure (LVEF ≤ 55%), clinical and laboratory signs of chronic kidney disease of stage ≥ III, or kidney injury of grade I, F or L according to the standardized RIFLE criteria for acute kidney injury (an acronym for "risk, injury, failure, loss, end-stage").
* Pregnancy, due to the high teratogenicity and toxicity of the drugs used in this clinical trial protocol. Female patients of childbearing potential are required to undergo pregnancy testing.
* Mental illness of the patient or legal guardians that makes it impossible to understand the nature of the study and compromises adherence to medical prescriptions and sanitary-hygienic requirements.