Phase I Safety and Tolerability of Intravesical VAX014 for Instillation in Subjects with Non-Muscle Inva (H-43665)
Phase 1, open-label, study of intravesical VAX014 in subjects with Non-Muscle Invasive Bladder Cancer (NMIBC) conducted in two phases; a dose escalation phase (Phase 1a) followed by an expansion phase at the RP2D (Phase 1b).
In the interest of safety, all subjects in the Phase 1a portion of the study will be staggered by at least one week from the prior subject.
The 1a dose escalation phase of the study will utilize a conventional 3+3 oncology study design in order to define an MTD (or maximal practical dose - MPD) and a Recommended Phase 2 Dose (RP2D). Subjects will receive intravesical VAX014 based on their assigned cohort. Each cohort will consist of 3 to 6 subjects.
The 1b phase of the study will evaluate the RP2D administered to 10 additional subjects.
Both phases of the study will use a Window of Opportunity (WOO) study design wherein subjects with a single low-grade Ta lesion measuring ≥ 5 mm and ≤ 15 mm in largest diameter (either at presentation or remaining following resection of up to 4 similar lesions) will receive weekly intravesical VAX014 for 6 weeks prior to undergoing a scheduled cystoscopy. At the time of cystoscopy, the single low-grade Ta lesion, if remaining, will be measured and resected. If the lesion has resolved, then a biopsy will be taken at the location of the lesion.
Resected specimens and any biopsy material collected during the course of the study will be archived for potential assessment of biomarkers (e.g., alpha 3 and alpha 5 integrin expression).
1. Age 18 or more years
2. Pathologically confirmed low-risk, Ta Grade 1 (TaG1) urothelial carcinoma (UC) of the urinary bladder1.