EPG-HU1418-201
Research type
Research Study
Full title
A Phase 2/3 Study to Characterize and Evaluate the Efficacy, Safety, and Tolerability of hu14.18K322A Treatment Given in Combination with Chemotherapy in Participants with High-Risk Neuroblastoma
IRAS ID
1013435
Contact name
Patricia Marosics
Contact email
Sponsor organisation
Renaissance Pharma Ltd. (part of the Essential Pharma Group)
Research summary
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 hu14.18K322A is when used together with chemotherapy to treat children and young people who have high-risk neuroblastoma. The study is designed so that two groups will be included initially, one group whose disease got better with prior treatment but has since come back (relapsed) and one group whose disease did not get better with initial treatment (refractory). Each group will receive hu14.18K322A and chemotherapy to study how well the combined treatment works and how safe it is. The study is "open-label," meaning everyone knows what treatment is being given. The first part of the study will focus on finding the best dose of hu14.18K322A to give with chemotherapy. In this part, up to 12 participants (6 with relapsed disease and 6 with refractory disease) will be placed into one of two groups, to test two different doses of hu14.18K322A with the same chemotherapy drugs. An independent group of experts will carefully review safety information and side effects after the first cycle of treatment to decide which dose of hu14.18K322A should be used for the rest of the study. Children and young people who take part in the study must be between 18 months and 18 years old and have confirmed high-risk neuroblastoma that has spread to other parts of the body. Up to 72 participants with relapsed disease and 72 participants with refractory disease may be enrolled. It is expected that participants will receive up to 12 cycles (a cycle is 21 days) of treatment. Once the treatment has finished, participants will be followed up to study any long-term effects.
REC name
North of Scotland Research Ethics Committee 1
REC reference
26/NS/0025
Date of REC Opinion
27 Apr 2026
REC opinion
Further Information Favourable Opinion