Recurrent or progression of neuroblastoma or neuroblastoma refractory to first-line treatment MedDRA version: 20.0 Level: PT Classification code 10029260 Term: Neuroblastoma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 21.0 Level: PT Classification code 10066595 Term: Neuroblastoma recurrent System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) MedDRA version: 20.0 Level: LLT Cla
Conditions
Interventions
Trade Name: Qarziba 4,5 mg/ml concentrate for solution for infusion
Product Name: DINUTUXIMAB BETA
Pharmaceutical Form: Concentrate for solution for infusion
Sponsors
Jagiellonian University Medical College
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Diagnosis of NBL according to international criteria (International Neuroblastoma Risk Group, INRG). 2. Patients 1-18 years of age with HR-NBL with primary refractory disease, disease progression or recurrence. 3. Adequate function of vital organs (if abnormal, dysfunction below grade 4 according to the CTC AE WHO classification, except for disorders defined in the exclusion criteria). 4. Life expectancy =6 months. 5. Obtaining the informed written consent of the patient and / or statutory representative for the treatment. 6. Female patients of childbearing potential must consent to the use of effective contraception; Breastfeeding patients must consent to the termination of breastfeeding. 7. Patients who have previously received immunotherapy with DB or other anti-GD2 specific antibodies may be eligible for this study. Are the trial subjects under 18? yes Number of subjects for this age range: 28 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients with toxicities of =3 CTCAE WHO grade, except hearing impairment, hematological disorders, liver and kidney disorders. 2. Patients with neurological toxicities of =2 CTCAE WHO grade. 3. Active life-threatening infection until stabilization of the patient's condition. 4. Pregnancy and / or lactation. 5. Sexually active patients who refuse to use an effective method of contraception. 6. Current treatment with experimental drugs or use of such treatment within 2 weeks before signing the informed consent to participate in the study. 7. Radiotherapy within 3 weeks prior to the start of the study. 8. Participation in another clinical trial within 6 months before signing the informed consent to participate in the trial (not applicable to clinical trials in 1st line of treatment in HR-NBL). 9. Lack of informed written consent to treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To test the safety and efficacy of dinutuximab beta therapy in combination with chemotherapy for the treatment of patients with relapsed or progressive neuroblastoma and for first-line treatment of neuroblastoma refractory to standard therapy. The primary objective of the study is to evaluate the safety profile of 20 patients receiving dinutuximab beta in combination with chemotherapy compared to historical data from two reference groups: - group 1 for patients treated with chemotherapy without immunotherapy - group 2 for patients treated with immunotherapy without chemotherapy in patients with relapsed or progressive or primary resistance to treatment. The reference groups will consist of 2 groups of 10 patients who meet the inclusion criteria for the target group, but are treated only with chemotherapy (reference group 1) or only with immunotherapy (reference group 2) at the Department of Pediatric Oncology and Hematology in Krakow. ;Secondary Objective: The secondary objective of the study is an initial efficacy assessment as a percentage of patients: 1.complete remission assessed during the study and at the last visit, 2.with partial remission assessed during the study and at the last visit, 3.with disease stabilization assessed during the examination and at the last visit, 4. PFS assessed during the study, 5. ESF assessed during the study. In addition, the tertiary and exploratory objective of the study is to evaluate the usefulness of laboratory parameters in predicting response to dinutuximab beta therapy. Tertiary goals will not be subject to formal analysis.;Primary end point(s): The following safety endpoints will be assessed: 1.number of cycles aborted due to toxicity, 2.number of cycles in which treatment interruptions due to the occurrence of side effects will be longer than provided for in the treatment protocol, 3.number of episodes of Capillary Leak Syndrome, regardless of severity 4.number of episodes of cytokine release syndrome, r | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Percentage of patients: 1.with complete remission assessed during the study and at the last visit, 2.with partial remission assessed during the study and at the last visit, 3.with disease stabilization assessed during the examination and at the last visit, 4. PFS assessed during the study, 5. ESF assessed during the study.;Timepoint(s) of evaluation of this end point: During the study and at the last visit - 12 months after end of treatment, | — |
Countries
Poland
Contacts
Public ContactClinical Trial Office
Jagiellonian University Medical College
Outcome results
None listed