Toripalimab ± Sequential Intravesical Gemcitabine-Mitomycin C for BCG-Unresponsive/-Intolerant Hi… (NCT07189793) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Toripalimab ± Sequential Intravesical Gemcitabine-Mitomycin C for BCG-Unresponsive/-Intolerant High-Risk NMIBC: Open-Label Randomized Phase 2 Study
China106 participantsStarted 2025-10
Plain-language summary
This open-label, randomised, multicentre, phase 2 study (OHAI-NMIBC-01) compares toripalimab plus sequential intravesical gemcitabine followed by mitomycin C (GEM→MMC) with toripalimab alone in adults with BCG-unresponsive or BCG-intolerant high-risk non-muscle-invasive bladder cancer (HR-NMIBC). Two prespecified cohorts are analysed: (1) CIS cohort (CIS with/without Ta/T1) and (2) non-CIS cohort (high-risk Ta/T1 without CIS). In the combination arm, intravesical GEM→MMC is given weekly for 6 weeks (induction) and, for patients without recurrence at the first tumour assessment (\~month 3), monthly maintenance continues up to 24 months or until progression/unacceptable toxicity; toripalimab IV every 3 weeks starts during the first intravesical cycle and continues up to 24 months or until progression/unacceptable toxicity. The monotherapy arm receives toripalimab IV every 3 weeks up to 24 months or until progression/unacceptable toxicity. Cystoscopy and urine cytology are performed every 3 months; imaging every 24 weeks. Primary endpoints are 3-month complete response (CR) rate in the CIS cohort and median recurrence-free survival (RFS) in the non-CIS cohort. Secondary endpoints include landmark CR, PFS and OS, RFS/HG-RFS landmarks in the non-CIS cohort, and safety (CTCAE v5.0). Exploratory analyses will assess outcomes by protocol-defined PD-L1 status. Approximately 106 participants will be enrolled at multiple sites in China.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Age ≥18 years; sex: all; signed written informed consent by the participant or legally authorised representative.
✓. Histologically confirmed high-risk non-muscle-invasive bladder cancer (HR-NMIBC), defined as any T1, high-grade Ta, and/or carcinoma in situ (CIS).
✓. BCG-intolerant (unable to continue BCG because of severe adverse reactions) or meeting at least one definition of BCG treatment failure:
✓. Persistent or recurrent CIS within 12 months after completion of adequate BCG (with or without concomitant NMIBC);
✓. Recurrent high-grade Ta/T1 within 6 months after completion of adequate BCG;
✓. High-grade T1 at the first evaluation after BCG induction (\~3 months);
✓. Ta high-grade and/or CIS present or recurrent at \~3 months after receiving ≥5 BCG instillations.
✓. ECOG performance status 0-2.
Exclusion criteria
✕. Muscle-invasive bladder cancer (T2-T4).
What they're measuring
1
Complete Response (CR) Rate at Month 3 in the CIS Cohort
Timeframe: Baseline to Month 3
2
Median Recurrence-Free Survival (RFS) in the Non-CIS Cohort
Timeframe: From randomisation/start of treatment to first event, assessed up to 24 months
Trial details
NCT IDNCT07189793
SponsorFirst Affiliated Hospital of Wenzhou Medical University
✕. Low-grade (LG) recurrence during or after BCG therapy.
✕. Concomitant upper tract urothelial carcinoma, or lymph-node/distant metastasis.
✕. Indwelling ureteral stent or known vesicoureteral reflux.
✕. Contraindications to intravesical instillation, including within 2 weeks after TURBT, bladder perforation, symptomatic urinary tract infection, or gross haematuria.
✕. Known hypersensitivity or contraindication to gemcitabine, mitomycin C, or toripalimab.
✕. Systemic chemotherapy, small-molecule targeted therapy, or radiotherapy within 2 weeks before first study treatment.