Safety evaluation and initial efficacy evaluation will be conducted in a group of patients as a non-commercial, open-label clinical trial of dinutuximab beta (Qarziba) phase IIa.
The investigational medicinal product will be dinutuximab beta (anti-GD2 antibodies against GD2 disialoganglioside on NBL cells) at a dose of 10 mg / m2 / day by continuous infusion for 5 days in combination with irinotecan / temozolomide, topotecan / temozolomide or N5 / N6 chemotherapy GPOH protocol.
The study group will be patients with recurrent / progression of NBL or disease resistant to first-line treatment, for whom there are currently no standards of management, and the treatment methods used so far do not give a chance to achieve a permanent remission of the disease. After diagnosis of relapse / progression or resistance to treatment, it is permissible to administer 2 cycles of standard chemotherapy prior to enrollment in the study.
The study plans to recruit 20 patients who will receive 5-7 cycles of DB with chemotherapy. The choice of an appropriate chemotherapy regimen will depend on the patient's prior treatment and tolerance.
The safety assessment will be conducted based on the registration of the types and frequency of adverse reactions identified on the basis of the registration of clinical parameters, symptoms and / or diseases reported by the patient or identified during the intervention and / or follow-up visits, abnormal laboratory and / or imaging test results.
The initial assessment of the effectiveness will consist in comparing the percentage of objective responses obtained and the annual EFS and PFS (imaging tests, including scintigraphy, bone marrow examination and tumor markers). The study also included an exploratory evaluation of the usefulness of immunological, genetic and other studies.
Diagnosis of NBL according to international criteria (International Neuroblastoma Risk Group, INRG).
Patients 1-18 years of age with HR-NBL with primary refractory disease, disease progression or recurrence.
Adequate function of vital organs (if abnormal, dysfunction below grade 4 according to the CTC AE WHO classification, except for disorders defined in the
Life expectancy ≥6 months.
Obtaining the informed written consent of the patient and/or statutory representative for the treatment.
Female patients of childbearing potential must consent to the use of effective contraception; Breastfeeding patients must consent to the termination of breastfeeding.
Patients who have previously received immunotherapy with DB or other anti-GD2 specific antibodies may be eligible for this study.
Single group
Chemoimmunotherapy (Dinutuximab beta in combination with chemotherapy)
Combination productDinutuximab beta in combination with chemotherapy.
Number of cycles aborted due to toxicity.
Number of cycles in which treatment interruptions due to the occurrence of side effects will be longer than provided for in the treatment protocol.
Number of episodes of Capillary Leak Syndrome, regardless of severity.
Number of episodes of cytokine release syndrome, regardless of severity.
Percentage of patients with complete remission assessed during the study and at the last visit.
Percentage of patients with partial remission assessed during the study and at the last visit.
Percentage of patients with disease stabilization assessed during the examination and at the last visit.
Percentage of patients PFS assessed during the study.
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