Neuroblastoma is the most common type of solid cancer found outside the brain in young children. Generally, it affects children younger than 5 years old, with the average age when it is found being just 2 years. Most patients have 'high-risk' disease, with spread of the disease to different sites (metastases). This multinational study aims to find out how effective and safe the treatment of a monoclonal anti-GD2 antibody hu14.18K322A (daretabart) is when used together with chemotherapy to treat children and young people who have high-risk neuroblastoma.
This is a Phase 2/3, open label, single arm study, evaluating how well hu14.18K322A works and how safe it is in combination with chemotherapy, in relapsed and refractory high-risk neuroblastoma (HRNB) patients. The study is in 2 parts. The first part of the study will test two doses of hu14.18K322A to give with chemotherapy in 12 patients to confirm the best dose. Once the best dose is found, Part 2 of the study will recruit up to an additional 66 participants with relapsed disease and 66 participants with refractory disease. Children and young people who take part in the study must be between 18 months and 18 years old at the time of consent and have confirmed HRNB that has spread to other parts of the body. It is expected that participants will receive up to 12 cycles of treatment. Once the treatment has finished, participants will be followed up to study long-term effects.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
144
21-day cycle for a maximum of 12 cycles
Children's Hospital Colorado Anschutz Medical
Aurora, Colorado, United States
RECRUITINGUniversity of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States
RECRUITINGThe Children's Hospital at Montefiore
The Bronx, New York, United States
RECRUITINGDuke University Medical Center
Durham, North Carolina, United States
RECRUITINGChildren's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
RECRUITINGThe Hospital for Sick Children
Toronto, Ontario, Canada
RECRUITINGUniversity Hospital Vall d'Hebron
Barcelona, Spain
RECRUITINGHospital Sant Joan de Deu
Barcelona, Spain
RECRUITINGSouthampton Children's Hospital
Southampton, Hampshire, United Kingdom
RECRUITINGOverall response rate (ORR)
Proportion of participants treated with hu14.18K322A in combination with chemotherapy who achieve a complete response (CR) or partial response (PR) according to the International Neuroblastoma Response Criteria (INRC)
Time frame: Assessed at end of treatment (up to 12 months)
Metastatic complete response rate (mCRR)
Proportion of participants treated with hu14.18K322A in combination with chemotherapy who achieve an overall metastatic complete response (mCR) response
Time frame: Assessed at end of treatment (up to 12 months)
Overall survival (OS)
Time frame: Up to 3 years
Overall Metastatic Response Rate (mORR)
Time frame: Assessed at end of treatment (up to 12 months)
Duration of Response (DOR)
Time frame: From the time of first response until disease progression, discontinuation from study, start of new therapy, or death
Progression-Free Survival (PFS)
Time frame: From start of Cycle 1 until the first occurrence of progression followed for up to 3 years
Event-Free Survival (EFS)
Time frame: From start of Cycle 1 until the first occurrence of progression, secondary malignancy, or death due to any cause followed for up to 3 years
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