NCT06545955

A Trial to Evaluate Intravesical Nadofaragene Firadenovec Alone or in Combination With Chemotherapy or Immunotherapy in Participants With High-grade BCG Unresponsive Non-muscle Invasive Bladder Cancer

A Phase 3, Randomised, Multi-center, Open Label Trial to Evaluate the Safety and Efficacy of Intravesical Nadofaragene Firadenovec Alone or in Combination With Chemotherapy or Immunotherapy in Participants With High-grade Bacillus Calmette-Guerin Therapy (BCG) Unresponsive Non-muscle Invasive Bladder Cancer (NMIBC)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 250 (estimated)
• Sponsor: Ferring Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The pivotal phase 3 trial (rAd-IFN-CS 003) evaluating the efficacy of nadofaragene firadenovec showed that 55 (53.4%) of 103 subjects with CIS ± high-grade Ta/T1 achieved a complete response (CR) at 3 months. In this trial, the safety and efficacy of intravesical instillation of nadofaragene firadenovec alone or in combination with chemotherapy or immunotherapy will be evaluated in participants with NMIBC CIS (± high-grade Ta/T1).

Conditions

• Non-muscle Invasive Bladder Cancer With Carcinoma in Situ

Interventions

Type	Name	Description
DRUG	Nadofaragene Firadenovec	vector-based gene therapy for NMIBC treatment to potentiate durable therapeutic responses by interferon (IFN) alfa-2b (IFN-α2b) amplification. It is a non-replicating recombinant adenovirus serotype 5 vector containing a transgene encoding the human IFN-α2b gene.
DRUG	Gemcitabine	Intravesical Gemcitabine chemotherapy, used in combination with Docetaxel.
DRUG	Docetaxel	Intravesical Docetaxel chemotherapy, used in combination with Gemcitabine.
DRUG	Pembrolizumab	Pembrolizumab is an FDA approved immune checkpoint inhibitor which restores the anti-tumour immune response by blocking the programmed cell death protein 1 (PD-1). Pembrolizumab is administered via intravenous (IV) infusion.

Timeline

• Start date: 2024-10-01
• Primary completion: 2028-06-30
• Completion: 2030-12-31
• First posted: 2024-08-09
• Last updated: 2026-03-04

Locations

64 sites across 7 countries: United States, Canada, Czechia, France, Poland, South Korea, Spain

Regulatory

• FDA-regulated drug study