The multi-center, prospective randomized Phase III clinical trial is expected to enroll approximately 100 evaluable children with hepatoblastoma, an orphan disease, being treated with cisplatin. Patients will be randomized to receive either cisplatin alone, a platinum-based drug associated with frequent hearing loss used to treat hepatoblastoma, or cisplatin plus sodium thiosulfate (STS).
The study, which will be coordinated through the Children’s Cancer and Leukaemia Group (CCLG) in UK, will compare the level of hearing loss (ototoxicity) associated with cisplatin alone versus the combination of cisplatin plus STS, as well as the safety, tolerability and anti-tumor activity in both arms of the study.
Penelope Brock, consultant in paediatric oncology, Great Ormond Street Hospital for children in UK, said: “Hearing loss remains a very significant yet potentially preventable side effect of contemporary cancer treatment. Cisplatin, for instance, continues to be an important treatment option in childhood cancers but can cause permanent hearing loss; such hearing loss can lead to significant long term social and behavioral issues for these children. Drugs such as STS that protect against this hearing loss, without interfering with the effectiveness of the chemotherapy, could greatly improve the quality of these children’s lives.”