Skip to content
Bending the Curve Trial by Trial
?

Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial

Phase 3N = 157NCT02773849

Population
Adults with BCG-unresponsive non-muscle-invasive bladder cancer, ECOG status 2 or less; 157 enrolled and treated, 151 in the per-protocol efficacy analyses. Excluded: upper urinary tract disease, prostatic urethral urothelial carcinoma, lymphovascular invasion, micropapillary disease, hydronephrosis.
Design
Phase 3, multicentre, open-label, single-arm, repeat-dose study at 33 US centres. A single intravesical 75 mL dose of nadofaragene firadenovec (3 x 10^11 viral particles per mL), repeated at months 3, 6 and 9 in the absence of high-grade recurrence. Primary endpoint: complete response at any time in patients with carcinoma in situ (with or without a high-grade Ta or T1 tumour); null hypothesis a complete response rate below 27%. Efficacy analysed in the per-protocol population. The FDA approval letter calls the study CS-003.
Primary endpoint
Complete response in carcinoma in situ
Arms
  • Nadofaragene firadenovec (experimental, n = 157): 75 mL intravesical dose (3 x 10^11 viral particles per mL), repeated at months 3, 6 and 9 if no high-grade recurrence
People
Boorjian SA
Show the numbers
Results reported for Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial
EndpointAnalysisNadofaragene firadenovecControlEffect
Complete response in carcinoma in situPer-protocol efficacy population: 103 patients with carcinoma in situ (with or without a high-grade Ta or T1 tumour) strictly meeting the BCG-unresponsive definition; not described as intention-to-treatComplete response within 3 months of the first dose (the primary endpoint was complete response at any time)primaryBoorjian 202153.4%—55 of 103 patients; the abstract gives no confidence interval.
Complete response maintained at 12 months55 patients with a complete responseprimaryBoorjian 202145.5%—25 of 55 patients; the abstract gives no confidence interval.
Results reported for Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial
EndpointAnalysisNadofaragene firadenovecControlEffect
Complete response in carcinoma in situPer-protocol efficacy population: 103 patients with carcinoma in situ (with or without a high-grade Ta or T1 tumour) strictly meeting the BCG-unresponsive definition; not described as intention-to-treatComplete response within 3 months of the first dose (the primary endpoint was complete response at any time)primaryBoorjian 202153.4%—55 of 103 patients; the abstract gives no confidence interval.
Complete response maintained at 12 months55 patients with a complete responseprimaryBoorjian 202145.5%—25 of 55 patients; the abstract gives no confidence interval.
Primary publication: Boorjian SA, Alemozaffar M, Konety BR, Shore ND, Gomella LG, Kamat AM, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol. 2021;22(1):107-117. PubMedDOI

Appears in

Sources

  1. Boorjian SA, Alemozaffar M, Konety BR, Shore ND, Gomella LG, Kamat AM, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol. 2021;22(1):107-117.

    PubMedDOI