Ferring Pharmaceuticals, Inc
Background
Approximately one-third of patients with non–muscle-invasive bladder cancer (NMIBC) initially responding to Bacillus Calmette-Guérin (BCG) experience recurrence and/or progression in the first year after treatment.1 Local, effective, bladder-preserving options are needed for patients with BCG-unresponsive NMIBC. Nadofaragene firadenovec-vncg is the first FDA-approved intravesical gene therapy for treatment of high-risk BCG-unresponsive NMIBC with carcinoma in situ (CIS) ± papillary tumors. In a single-arm, multicenter, open-label, repeat-dose, phase 3 study, 53.4% of patients (55/103) with CIS ± high-grade Ta/T1 BCG-unresponsive NMIBC achieved complete response (CR) 3 months after the first instillation of nadofaragene firadenovec.2 The safety profile of nadofaragene firadenovec was manageable, with 103 (66%) patients reporting mild (grade 1/2) study drug-related adverse events (AEs). Six (3.8%) patients had grade 3/4/5 study drug-related AEs. ABLE-41 (NCT06026332) is an observational study evaluating the effectiveness, overall experiences, patterns of use, and safety in patients treated with nadofaragene firadenovec in a US real-world setting.
Methods
This noninterventional study includes approximately 50 urology sites in the U.S. with anticipated enrollment of 800 patients. Adults ≥18 years with prescribed and scheduled treatment with nadofaragene firadenovec per physician discretion or those who received their first instillation of nadofaragene firadenovec per physician discretion after September 5, 2023, but before site activation are eligible to enroll. The primary objective is to assess the effectiveness of nadofaragene firadenovec measured as the CR rate as determined by investigator. Secondary outcomes include: nadofaragene firadenovec patterns of use; duration of CR, recurrence-free survival, cystectomy-free survival, progression-free survival, overall survival, and bladder cancer–specific mortality; patient, caregiver and physician experiences; adjunctive use of molecular markers; and safety. Patient and caregiver experiences will be assessed using the respective EuroQol 5 Dimension 5 Level questionnaire and Work Productivity and Activity Impairment questionnaire, adapted for caregiving. Patients and caregivers will be surveyed before all nadofaragene firadenovec administrations. Physicians will be surveyed 1, 12, and 24 months after first patient first instillation. All AE data will be collected starting from index date. The estimated follow-up period is 24 months, until study discontinuation, or withdrawal. Final results from this large, prospective, multi-institutional, real-world registry providing early use and outcomes of nadofaragene firadenovec are expected December 2026. References: 1. Sylvester RJ et al. Eur Urol. 2010;57:766-73; 2. Boorjian SA et al. Lancet Oncol. 2021;22:107-17. Clinical trial information: NCT06026332.