A Phase 1 interventional study of Chemoimmunotherapy (Dinutuximab beta in combination with chemotherapy) in High-Risk Neuroblastoma, sponsored by Jagiellonian University. Recruiting at 1 site in Poland. Open to participants aged 1 Year to 18 Years. Per ClinicalTrials.gov, last updated 2022-03-09.
Sponsored by Jagiellonian University · Phase 1, Interventional, and Treatment
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.
Neuroblastoma (NBL) accounts for 8-10% of all childhood malignancies. It is the most common solid tumor outside the central nervous system in people \<18 years of age. It occurs in 6-11 / 1 million children annually. In Poland, NBL is diagnosed annually in 60-70 patients, in 1/3 high-risk disease (HR). In 90% of children, NBL is diagnosed before the age of 5. The diagnosis is made on the basis of the histopathological assessment of the tumor tissue or the presence of NBL cells in the bone marrow together with elevated levels of catecholamines or their metabolites in the urine. The prognostic factors include the patient's age at diagnosis, stage of disease, tumor histopathology, DNA ploidy, MYCN gene status, chromosomal changes, and initial response to therapy. Due to the different course of the disease, the therapeutic decision is made after determining the risk group based on international criteria (International Neuroblastoma Risk Group Stage System, INRGSS and International Neuroblastoma Staging System, INSS). In the lowest-risk group, management is limited to observation or surgery, and in the intermediate-risk group, only standard low- and intermediate-intensity chemotherapy or combined with radiation therapy and surgery is performed. In contrast, HR-NBL uses intensive combination therapy, including standard induction chemotherapy, surgery, high-dose chemotherapy (HD-CHT) and autologous hematopoietic stem cell transplantation (auto-HSCT), radiotherapy and maintenance therapy with 13-cis retinoic acid and targeted immunotherapy with anti-GD2 antibodies. Treatment outcomes in NBL vary from spontaneous tumor regression in some infants to an OS rate of \<50% despite intensive combination therapy in the HR-NBL group.
625 studies on the registry are indexed under Neuroblastoma; 123 are open to participants now.
This study's planned enrollment of 20 is below the median of 32 across 475 interventional studies indexed under Neuroblastoma.
Browse Neuroblastoma studies →Jagiellonian University is the lead sponsor of 156 studies on the registry; 34 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Chemoimmunotherapy arm.
Combination Product: Chemoimmunotherapy (Dinutuximab beta in combination with chemotherapy)
Dinutuximab beta in combination with chemotherapy.
Number of cycles aborted due to toxicity.
Time frame: 12 months after end of treatment.
Number of cycles in which treatment interruptions due to the occurrence of side effects will be longer than provided for in the treatment protocol.
Time frame: 12 months after end of treatment.
Number of episodes of Capillary Leak Syndrome, regardless of severity.
Time frame: 12 months after end of treatment.
Number of episodes of cytokine release syndrome, regardless of severity.
Time frame: 12 months after end of treatment.
Number of episodes of allergic reactions in CTCAE grade 3 and 4 (version in force at that time).
Time frame: 12 months after end of treatment.
Number of hematological toxicities in grade 3 and 4 CTCAE (version in force at that time).
Time frame: 12 months after end of treatment.
Number of neurological toxicity episodes, regardless of severity.
Time frame: 12 months after end of treatment.
The percentage of patients with pupil disorders and / or visual disturbances.
Time frame: 12 months after end of treatment.
Proportion of patients with renal or hepatic impairment in CTCAE grade 3 and 4 (version in force at that time).
Time frame: 12 months after end of treatment.
Other side effects in grade 3 and 4 CTCAE (version in force at the time)3 and 4 (version in force at that time).
Time frame: 12 months after end of treatment.
Percentage of patients with complete remission assessed during the study and at the last visit.
Time frame: 12 months after end of treatment.
Percentage of patients with partial remission assessed during the study and at the last visit.
Time frame: 12 months after end of treatment.
Percentage of patients with disease stabilization assessed during the examination and at the last visit.
Time frame: 12 months after end of treatment.
Percentage of patients PFS assessed during the study.
Time frame: 12 months after end of treatment.
Percentage of patients ESF assessed during the study.
Time frame: 12 months after end of treatment.
Plan to share: Undecided
No publications or documents are linked to this record.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Jagiellonian University