Neuroblastoma is one of the most common solid childhood tumours, and a major cause of cancer-related death in children. More than 1200 children/young adults a year are diagnosed in USA and Europe. Around 600 of these cases are considered high-risk, which means the cancer is more difficult to treat successfully. Despite improvements in survival over recent decades, a significant proportion of patients with high-risk neuroblastoma have disease that does not respond to standard treatments (refractory neuroblastoma) or comes back after completion of standard frontline treatment (relapsed neuroblastoma). Therefore, there is a need to develop new treatment strategies and test new drugs to improve outcomes for children with neuroblastoma. Aims Of The BEACON2 Trial * To improve survival for patients with relapsed neuroblastoma by developing new treatment combinations * To evaluate new treatment combinations in relapsed neuroblastoma, within a phase I/II trial that can impact clinical practice, while also allowing dose confirmation for new promising combinations * To evaluate the safety, activity, efficacy and impact on quality of life of these new treatment combinations in relapsed neuroblastoma patients * To improve our understanding of relapsed neuroblastoma biology and advance the development of targeted therapies using biomarkers, by conducting a comprehensive biomarker sample collection. Trial Design BEACON2 is a randomised phase I/phase II, open label, international trial. The trial will have two tiers: Tier 1 will be the main randomisation for two treatment arms initially. Participants will be randomised at trial entry to receive one of the available regimens, treatment A or treatment B. Tier 2 will include smaller dose expansion/confirmation cohorts for more novel experimental treatment combinations (Arm C and future arms), with the potential for them to be moved to Tier 1. Current Tier 1 (Randomisation Tier) Treatment Arms in the BEACON2 Trial: Arm A: dbIT Treatment with dinutuximab beta, irinotecan, and temozolomide, 3 weekly x12 cycles Arm B: BIT Treatment with bevacizumab, irinotecan, and temozolomide, 3 weekly x12 cycles Current Tier 2 (Registration Only Tier) Treatment Arms in the BEACON2 Trial: Arm C: dbBIT Treatment with dinutuximab beta, bevacizumab, irinotecan, and temozolomide, 3 weekly x12 cycles Patient Population and Sample Size Patients aged ≥1 years of age with relapsed neuroblastoma. For each arm in Tier 1, up to 75 patients will be recruited to complete phase 2 investigations. For each arm in Tier 2, 10 patients will be recruited to complete phase I investigations. Approximately 160 participants are initially planned, 75 in each arm of Tier 1 and 10 participants for one dose-confirmation cohort in Tier 2. The study is expected to recruit patients for 3 years, and then finish patient follow-up after an additional 5 years. Translational Sub-study / Biological Studies It is standard of care for patients diagnosed with relapsed neuroblastoma to: * Have had a tumour sample collected at point of initial diagnosis (either during biopsy or surgery) * Have bloods collected before they start and during treatment for their relapsed neuroblastoma * Have a bone aspirate/trephine procedure in order to help confirm relapse. These samples provide very important opportunities for further research, and the study investigators would like to make full use of these opportunities by collecting the analysis already performed on these samples and collect some additional samples (at the same time as the standard ones) to learn and understand more about neuroblastoma and its treatment. Samples will undergo research analysis at the national SIOPEN reference laboratories.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
160
Bevacizumab
Dinutuximab beta
Irinotecan
Topotecan
Temozolomide capsule
Temozolomide liquid suspension
Sydney Children's Hospital (SCH)
Sydney, Australia
NOT_YET_RECRUITINGSt. Anna Children´s Hospital
Vienna, Austria
NOT_YET_RECRUITINGCliniques Universitaires Saint-Luc (CUSL)
Brussels, Belgium
NOT_YET_RECRUITINGStarship Children's Hospital (SSH)
Auckland, New Zealand
NOT_YET_RECRUITINGRoyal Aberdeen Children's Hospital
Aberdeen, United Kingdom
RECRUITINGRoyal Belfast Hospital for Sick Children
Belfast, United Kingdom
NOT_YET_RECRUITINGBirmingham Children's Hospital
Birmingham, United Kingdom
RECRUITINGBristol Royal Hospital for Children
Bristol, United Kingdom
RECRUITINGAddenbrookes Hospital
Cambridge, United Kingdom
RECRUITINGChildren's Hospital for Wales
Cardiff, United Kingdom
RECRUITING...and 13 more locations
Progression-Free Survival time
• Progression-Free Survival time (as per International Neuroblastoma Response Criteria (PD Progressive disease, SD Stable disease, MR Minor response, PR Partial response, CR Complete response) INRC 2017) - for Tier 1 (randomised comparison)
Time frame: From date of randomization until the date of first documented failure event (progression or death), assessed up to 60 months.
Occurrence of dose-limiting toxicities to definition of a safe and tolerable combination regimen
Tier 2 (dose expansion-confirmation cohorts): Definition of a safe and tolerable (i.e. \</= 3 dose-limiting toxicities) combination regimen
Time frame: From date of randomization until the date of first documented failure event (progression or death), assessed up to 60 months.
Best objective response
Best objective response (complete and partial response) per the INRC 2017 during trial treatment (12 cycles)
Time frame: At the end of the last treatment cycle, up to 12 cycles (each cycle is 21 days)
Clinical benefit
Clinical benefit (complete, partial and minor response and stable disease) per the INRC 2017 (International Neuroblastoma Response Criteria (PD Progressive disease, SD Stable disease, MR Minor response, PR Partial response, CR Complete response))
Time frame: At the end of the last treatment cycle, up to 12 cycles (each cycle is 21 days)
Time response to progression
Time between Response to Progression (i.e. Duration of Response) for patients who respond
Time frame: From date of response (Complete Response, Partial Response or Minor Response as per INRC) until the date of first documented failure event (progression or death), assessed up to 60 months.
Overall Survival time
Overall Survival time
Time frame: From date of trial entry until the date of first documented failure event (progression or death), assessed up to 60 months.
Quality of life of patients measured by Peds-QL questionnaires
Quality of life of patients measured by Pediatric Quality of Life Inventory (Peds-QL) questionnaires. 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0), so that higher PedsQL 3.0 scores indicate better Health Related Quality of Life (HRQOL).
Time frame: At the end of the last treatment cycle, up to 12 cycles (each cycle is 21 days)
Incidence and Severity of AEs
Incidence and Severity of AEs
Time frame: From date of trial entry until the date of last follow up, assessed up to 60 months.
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