Safety and efficacy of oral cancer vaccine B440 in patients with PD-1/PD-L1 inhibitor-resistant advanced urothelial cancer: a study protocol for a phase 1 multicenter, open-label, single-arm clinical trial

• Published in: BMC cancer Vol. 25; no. 1; pp. 195 - 11
• Main Authors: Furukawa, Junya, Kakei, Yasumasa, Murakami, Sae, Kita, Hiroshi, Ueki, Hideto, Hara, Takuto, Teishima, Jun, Hinata, Nobuyuki, Miyake, Hideaki, Fujisawa, Masato, Shirakawa, Toshiro
• Format: Journal Article
• Language: English
• Published: London BioMed Central 04.02.2025; BMC
• Subjects: Administration, Oral; Adult; advanced urothelial cancer; Aged; Angina pectoris; Antibodies; Antigens; B440; B7-H1 Antigen - antagonists & inhibitors; Biomedical and Life Sciences; Biomedicine; Bladder cancer; Breastfeeding & lactation; Cancer Research; Cancer therapies; Cancer vaccines; Cancer Vaccines - administration & dosage; Cancer Vaccines - adverse effects; Cancer Vaccines - therapeutic use; Clinical trial; Clinical trials; Clinical Trials, Phase I as Topic; Creatinine; Drug Resistance, Neoplasm; Drugs; Female; Health Promotion and Disease Prevention; Humans; ICI; Immune Checkpoint Inhibitors - pharmacology; Immune Checkpoint Inhibitors - therapeutic use; Immune system; Immunity (Disease); Immunotherapy; Japan; Male; Medicine/Public Health; Metastasis; Middle Aged; Multicenter Studies as Topic; Oncology; Oral cancer; Patients; PD-1 protein; PD-L1 protein; Phase 1; Programmed Cell Death 1 Receptor - antagonists & inhibitors; Protein expression; Proteins; Registration; Response rates; Study Protocol; Surgical Oncology; Toxicity; Treatment Outcome; Urologic Neoplasms - drug therapy; Urologic Neoplasms - immunology; Urologic Neoplasms - pathology; Urothelial cancer; Vaccines; WT-1 protein; Japan; B440; Clinical trial; WT-1 protein; Phase 1; advanced urothelial cancer; ICI
• ISSN: 1471-2407; 1471-2407
• DOI: 10.1186/s12885-025-13514-4

Background This is a multicenter, open-label, single-arm clinical trial to evaluate the safety and efficacy of oral cancer vaccine B440 in patients with PD-1/PD-L1 inhibitor-resistant advanced urothelial cancer. Methods The trial will be performed at three university hospitals in Japan. The target number of patients will be 12. The patients will be treated orally with B440 once daily for 5 days followed by 2 days for four consecutive courses (4 weeks, 20 treatments). The low-dose group will receive 800 mg (4 capsules) per dose and the high-dose group will receive 1,600 mg (8 capsules) per dose. The primary outcome will be the number and incidence of DLT cases the start of treatment and Day 28. Secondary outcomes are the presence or absence of a response, the best overall response and PFS. Discussion If this trial shows B440 to be safe and effective, it may lead to a late phase randomized controlled trial in advanced urothelial cancer. Ultimately, we hope to provide a new treatment option for such patients. Trial registration Japan Registry of Clinical Trials (jRCT) identifier: jRCT2051220143. Registered on December 27, 2022.