Urinary Bladder NeoplasmsPhase 2OHAI-NMIBC-01NCT07189793

Toripalimab ± Sequential Intravesical Gemcitabine-Mitomycin C for BCG-Unresponsive/-Intolerant High-Risk NMIBC: Open-Label Randomized Phase 2 Study

A Phase 2 study testing Toripalimab, Gemcitabine (GEM) and Mitomycin C (MMC) in people with Urinary Bladder Neoplasms and Carcinoma, Transitional Cell, run by First Affiliated Hospital of Wenzhou Medical University at 1 site in China.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Urinary Bladder Neoplasms, Carcinoma, Transitional Cell, Carcinoma in Situ of Bladder
What is being tested?: Toripalimab, Gemcitabine (GEM) and Mitomycin C (MMC)
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 106 participants
Where: 1 siteChina
Study dates: Dec 2026to Oct 2028
Phase: Phase 2
Are there results?: Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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);

Show all 12Show fewer

Ta high-grade and/or CIS present or recurrent at ~3 months after receiving ≥5 BCG instillations.
Adequate BCG, for the purposes of this protocol, is defined as receipt of at least 5 of 6 induction instillations (maintenance not required).
ECOG performance status 0-2.
Adequate organ function per protocol laboratory criteria.
No intravesical chemotherapy or immunotherapy between the most recent cystoscopy/TURBT and study start; a single immediate postoperative intravesical chemotherapy at the time of the most recent cystoscopy/TURBT is allowed during screening per local practice.
Willing and able to comply with study procedures.

Who cannot join

Muscle-invasive bladder cancer (T2-T4).
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.

Show all 13Show fewer

Systemic chemotherapy, small-molecule targeted therapy, or radiotherapy within 2 weeks before first study treatment.
Prior immune checkpoint inhibitor therapy.
Pregnant, planning pregnancy, or breastfeeding women.
Ongoing acute or chronic systemic infection, or history of active tuberculosis.
Other malignancy requiring active treatment.
Any condition that, in the investigator's judgment, makes participation not in the patient's best interest or could confound study results.
Study Population Adults with BCG-unresponsive or BCG-intolerant HR-NMIBC treated at participating centres in China.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Qi Lin, MM contact

Wei Chen, MD contact

China1 site

The First Affiliated Hospital of Wenzhou Medical University
Wenzhou, Zhejiang, 325000
Hang Huang, MD, +8613738301029, Huanghang163@163.com

Other trials in China

About this study

Official title: Toripalimab With Sequential Intravesical Gemcitabine-Mitomycin C Versus Toripalimab Alone for the Treatment of BCG-Unresponsive/-Intolerant High-Risk Non-Muscle-Invasive Bladder Cancer: An Open-Label, Randomized, Multicenter, Phase 2 Study

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.

Read the detailed description

Rationale and Objectives. A substantial proportion of HR-NMIBC patients are BCG-unresponsive or BCG-intolerant and face early high-grade recurrence and consideration of cystectomy. This trial evaluates whether adding sequential intravesical chemotherapy (gemcitabine followed by mitomycin C in the same visit) to systemic PD-1 blockade (toripalimab) improves outcomes versus toripalimab alone.

Design. Prospective, open-label, randomised (1:1), parallel-group, phase 2 study at multiple centres in China. Treatment continues until recurrence/progression, unacceptable toxicity, withdrawal, or completion of 24 months.

Interventions.

Arm A (Combination): Toripalimab 240 mg IV every 3 weeks (Q3W) up to 24 months plus intravesical GEM→MMC: gemcitabine 1,000 mg retained ~60 min and drained, then mitomycin C 40 mg retained ~60 min; administered weekly for 6 weeks (induction) and then maintenance every 4 weeks (Q4W) up to month 24 if no high-grade recurrence.

Arm B (Monotherapy): Toripalimab 240 mg IV Q3W up to 24 months.

Assessments. Cystoscopy and urine cytology at months 3, 6, 9, 12 and every 3 months thereafter; abdominopelvic/upper-tract CT or MRI every 24 weeks; routine laboratory tests prior to dosing/instillation. (Exploratory tissue/urine/blood sampling may be performed per protocol.)

Primary Endpoints.

CIS cohort: CR rate at month 3 (proportion). CR is met by any of the following protocol-specified scenarios indicating no high-grade bladder disease: (a) negative urine cytology and negative cystoscopy; (b) negative cytology with cystoscopic lesions that are benign or low-grade Ta on biopsy; or (c) negative cystoscopy with positive cytology attributed to tumour in the upper tract or prostatic urethra and random bladder biopsies negative.

Non-CIS cohort: Median RFS, defined as time from start of treatment to high-grade Ta recurrence, any T1, or new CIS. Recurrence requires cystoscopic suspicion confirmed by histopathology; if cytology becomes positive prior to histologic confirmation, the recurrence date is set at the first positive cytology once recurrence is subsequently confirmed.

Secondary Endpoints.

CIS cohort: CR rates at months 6, 12, 18, and 24; PFS rates at the same landmarks (progression defined as lamina propria invasion from Ta/CIS to T1, progression to ≥T2, or new nodal/distant metastasis); OS at month 24, EOT+6 months, and EOT+12 months; incidence of adverse events (AEs).

Non-CIS cohort: RFS rates at months 6, 12, 18, and 24; high-grade RFS (HG-RFS) rates at the same landmarks (high-grade recurrence defined as Tis or Ta/T1 high-grade, or muscle-invasive disease at TURBT or cystectomy); PFS rates at months 6/12/18/24; OS at month 24, EOT+6 months, and EOT+12 months; AE incidence.

Exploratory Endpoints.

CIS cohort: 3-month CR rate in PD-L1-positive and PD-L1-negative subgroups (per protocol-specified assay and criteria; no prespecified CPS threshold).

Non-CIS cohort: Median RFS in PD-L1-positive and PD-L1-negative subgroups (recurrence definitions as above).

Sample Size and Oversight. Approximately 106 participants will be randomised 1:1. Safety will be monitored throughout; a Data Monitoring Committee oversees participant protection and study conduct.

Conditions: Urinary Bladder Neoplasms, Carcinoma, Transitional Cell, Carcinoma in Situ of Bladder, Non-Muscle-Invasive Bladder Cancer
Being tested: Toripalimab, Gemcitabine (GEM), Mitomycin C (MMC)
Keywords: NMIBC, High-Risk NMIBC, BCG-Unresponsive, BCG-Intolerant, BCG Failure, Carcinoma in Situ (CIS), Intravesical Therapy, Ta, T1, Gemcitabine, Mitomycin C, GEM-MMC, Toripalimab, PD-1 Inhibitor, Bladder-Sparing
Medical subject headings: Urinary Bladder Neoplasms, Carcinoma, Transitional Cell, Non-Muscle Invasive Bladder Neoplasms, Carcinoma in Situ

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ToripalimabDrug
Also called JS001, Tuoyi
PD-1 inhibitor administered intravenously every 3 weeks (Q3W) for up to 24 months. Starts during the first intravesical treatment cycle.
Gemcitabine (GEM)Drug
Also called GEM
Intravesical instillation as part of a sequential regimen with mitomycin C: weekly for 6 weeks (induction); if no recurrence at first tumour assessment (~month 3), maintenance instillations continue monthly up to 24 months.
Mitomycin C (MMC)Drug
Intravesical instillation immediately after intravesical gemcitabine in the same visit (sequential regimen): weekly for 6 weeks (induction); if eligible, monthly maintenance up to 24 months.

Study groups in NCT07189793
Study group	Role	What they receive
Combination: Toripalimab + Intravesical GEM-MMC	Experimental	Toripalimab IV Q3W starting with the first intravesical cycle, plus sequential intravesical gemcitabine followed by mitomycin C in the same visit. Induction weekly ×6; if no recurrence at first tumour assessment (~month 3), monthly maintenance up to 24 months. Receives: Drug: Toripalimab; Drug: Gemcitabine (GEM); Drug: Mitomycin C (MMC)
Monotherapy: Toripalimab Alone	Active comparator	Toripalimab IV every 3 weeks (Q3W) up to 24 months or until disease progression, unacceptable toxicity, or withdrawal; no intravesical chemotherapy. Receives: Drug: Toripalimab

What is being measured

The primary outcome is the main question the study is designed to answer.

Complete Response (CR) Rate at Month 3 in the CIS Cohort
Proportion of participants in the CIS cohort achieving CR at month 3. CR is met by any of the following protocol-specified scenarios indicating no high-grade bladder disease: (a) negative urine cytology and negative cystoscopy; (b) negative cytology with cystoscopic lesions that are benign or low-grade Ta on biopsy; or (c) negative cystoscopy with positive cytology attributed to tumour in the upper tract or prostatic urethra and random bladder biopsies negative.
Measured: Baseline to Month 3
Median Recurrence-Free Survival (RFS) in the Non-CIS Cohort
RFS is time to high-grade Ta recurrence, any T1, or new CIS, whichever occurs first. Recurrence requires cystoscopic suspicion confirmed by histopathology; if cytology becomes positive before histologic confirmation, the recurrence date is set at the first positive cytology once recurrence is subsequently confirmed. Death from any cause before documented recurrence is counted as an event.
Measured: From randomisation/start of treatment to first event, assessed up to 24 months

Secondary outcomes (9)

CR Rates at Months 6, 12, 18, and 24 in the CIS Cohort
Proportion of participants in the CIS cohort with CR at the specified time points using the protocol CR definition.
Measured: Baseline to months 6, 12, 18, and 24
Progression-Free Survival (PFS) Rates at Months 6, 12, 18, and 24 in the CIS Cohort
Proportion without progression at each time point. Progression is defined as lamina propria invasion from Ta/CIS to T1, or progression to ≥T2, or new nodal or distant metastasis.
Measured: Baseline to months 6, 12, 18, and 24
Overall Survival at Month 24, EOT+6 Months, and EOT+12 Months in the CIS Cohort
Survival status at prespecified landmarks.
Measured: Baseline to Month 24, EOT+6 months, and EOT+12 months
Incidence of Adverse Events in the CIS Cohort
Participants with treatment-emergent AEs and grade ≥3 AEs (MedDRA; CTCAE v5.0). Immune-related and instillation-related events summarised.
Measured: From first dose/instillation to 90 days post-last dose/instillation (≈ up to 27 months)
Recurrence-Free Survival Rates at Months 6, 12, 18, and 24 in the Non-CIS Cohort
Proportion without recurrence (high-grade Ta, any T1, or new CIS) or death at each time point; recurrence confirmation rules per protocol.
Measured: Baseline to months 6, 12, 18, and 24
High-Grade Recurrence-Free Survival (HG-RFS) Rates at Months 6, 12, 18, and 24 in the Non-CIS Cohort
Proportion without high-grade recurrence at each time point. High-grade recurrence is defined as Tis or Ta/T1 high-grade, or muscle-invasive disease identified at TURBT or cystectomy.
Measured: Baseline to months 6, 12, 18, and 24
Progression-Free Survival Rates at Months 6, 12, 18, and 24 in the Non-CIS Cohort
Proportion without progression (lamina propria invasion from Ta/CIS to T1, progression to ≥T2, or new nodal/distant metastasis). Death before documented progression counts as an event.
Measured: Baseline to Months 6, 12, 18, and 24
Overall Survival at Month 24, EOT+6 Months, and EOT+12 Months in the Non-CIS Cohort
Survival status at prespecified landmarks.
Measured: Baseline to Month 24, EOT+6 months, and EOT+12 months
Incidence of Adverse Events in the Non-CIS Cohort
Participants with treatment-emergent AEs and grade ≥3 AEs (MedDRA; CTCAE v5.0).
Measured: From first dose/instillation to 90 days post-last dose/instillation (≈ up to 27 months)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2026Oct 2028

First posted24 Sep 2025
Study startDec 2026 (estimated)
Primary completionOct 2027 (estimated)
Study completionOct 2028 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (6)Papers the sponsor has linked to this study.
Other trials for Urinary Bladder NeoplasmsStudies of the same condition, open and under way.
More trials run by First Affiliated Hospital of Wenzhou Medical UniversityThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Model description: Two-arm, 1:1 randomised, open-label, parallel-group phase 2 trial comparing toripalimab plus sequential intravesical gemcitabine→mitomycin C with toripalimab alone in adults with BCG-unresponsive or BCG-intolerant high-risk NMIBC. Randomisation stratified by CIS status, stage (Ta vs T1), BCG failure subtype, and study centre. Treatment up to 24 months.
Masking: None (open label)
Enrolment: 106 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT07189793
Sponsor's protocol ID: KY2025-352
Acronym: OHAI-NMIBC-01
Status last verified: Sep 2026
First submitted: 17 Sep 2025
Last updated: 8 Sep 2026
Results posted: No
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Undecided: The sponsor and investigators intend to share de-identified IPD but the final plan (scope, timing, and access mechanism) is pending institutional approvals. This record will be updated once the IPD sharing policy is finalised.

Publications

Holzbeierlein JM, Bixler BR, Buckley DI, Chang SS, Holmes R, James AC, Kirkby E, McKiernan JM, Schuckman AK. Diagnosis and Treatment of Non-Muscle Invasive Bladder Cancer: AUA/SUO Guideline: 2024 Amendment. J Urol. 2024 Apr;211(4):533-538. doi: 10.1097/JU.0000000000003846. Epub 2024 Jan 24.
PubMed 38265030
Lightfoot AJ, Breyer BN, Rosevear HM, Erickson BA, Konety BR, O'Donnell MA. Multi-institutional analysis of sequential intravesical gemcitabine and mitomycin C chemotherapy for non-muscle invasive bladder cancer. Urol Oncol. 2014 Jan;32(1):35.e15-9. doi: 10.1016/j.urolonc.2013.01.009. Epub 2013 Mar 17.
PubMed 23510863
Breyer BN, Whitson JM, Carroll PR, Konety BR. Sequential intravesical gemcitabine and mitomycin C chemotherapy regimen in patients with non-muscle invasive bladder cancer. Urol Oncol. 2010 Sep-Oct;28(5):510-4. doi: 10.1016/j.urolonc.2008.11.019. Epub 2009 Jan 26.
PubMed 19171491
Black PC, Tangen CM, Singh P, McConkey DJ, Lucia MS, Lowrance WT, Koshkin VS, Stratton KL, Bivalacqua TJ, Kassouf W, Porten SP, Bangs R, Plets M, Thompson IM Jr, Lerner SP. Phase 2 Trial of Atezolizumab in Bacillus Calmette-Guerin-unresponsive High-risk Non-muscle-invasive Bladder Cancer: SWOG S1605. Eur Urol. 2023 Dec;84(6):536-544. doi: 10.1016/j.eururo.2023.08.004. Epub 2023 Aug 16.
PubMed 37596191
Necchi A, Roumiguie M, Kamat AM, Shore ND, Boormans JL, Esen AA, Lebret T, Kandori S, Bajorin DF, Krieger LEM, Niglio SA, Uchio EM, Seo HK, de Wit R, Singer EA, Grivas P, Nishiyama H, Li H, Baranwal P, Van den Sigtenhorst-Fijlstra M, Kapadia E, Kulkarni GS. Pembrolizumab monotherapy for high-risk non-muscle-invasive bladder cancer without carcinoma in situ and unresponsive to BCG (KEYNOTE-057): a single-arm, multicentre, phase 2 trial. Lancet Oncol. 2024 Jun;25(6):720-730. doi: 10.1016/S1470-2045(24)00178-5. Epub 2024 May 10.
PubMed 38740030
Balar AV, Kamat AM, Kulkarni GS, Uchio EM, Boormans JL, Roumiguie M, Krieger LEM, Singer EA, Bajorin DF, Grivas P, Seo HK, Nishiyama H, Konety BR, Li H, Nam K, Kapadia E, Frenkl T, de Wit R. Pembrolizumab monotherapy for the treatment of high-risk non-muscle-invasive bladder cancer unresponsive to BCG (KEYNOTE-057): an open-label, single-arm, multicentre, phase 2 study. Lancet Oncol. 2021 Jul;22(7):919-930. doi: 10.1016/S1470-2045(21)00147-9. Epub 2021 May 26.
PubMed 34051177

Interested in taking part?

Qi Lin, MM

+8615205771010

devillynch@126.com

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See sites and contacts

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All Urinary Bladder Neoplasms trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Toripalimab With Sequential Intravesical Gemcitabine-Mitomycin C Versus Toripalimab Alone for the Treatment of BCG-Unresponsive/-Intolerant High-Risk Non-Muscle-Invasive Bladder Cancer: An Open-Label, Randomized, Multicenter, Phase 2 Study
ClinicalTrials.gov ID: NCT07189793
Acronym / sponsor ID: OHAI-NMIBC-01 / KY2025-352
Conditions: Urinary Bladder Neoplasms, Carcinoma, Transitional Cell, Carcinoma in Situ of Bladder, Non-Muscle-Invasive Bladder Cancer
Keywords: NMIBC, High-Risk NMIBC, BCG-Unresponsive, BCG-Intolerant, BCG Failure, Carcinoma in Situ (CIS), Intravesical Therapy, Ta
Phase: Phase 2
Study type: Interventional
Recruitment status: Not yet recruiting
Enrolment: 106 participants (estimated)
Lead sponsor: First Affiliated Hospital of Wenzhou Medical UniversityUniversity, hospital or other
Study start: Dec 2026 (estimated)
Primary completion: Oct 2027 (estimated)
Study completion: Oct 2028 (estimated)
First posted: 24 Sep 2025 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Interventions.

Arm B (Monotherapy): Toripalimab 240 mg IV Q3W up to 24 months.

Primary Endpoints.

Secondary Endpoints.

Exploratory Endpoints.

CIS cohort: 3-month CR rate in PD-L1-positive and PD-L1-negative subgroups (per protocol-specified assay and criteria; no prespecified CPS threshold).

Non-CIS cohort: Median RFS in PD-L1-positive and PD-L1-negative subgroups (recurrence definitions as above).

Sample Size and Oversight. Approximately 106 participants will be randomised 1:1. Safety will be monitored throughout; a Data Monitoring Committee oversees participant protection and study conduct.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2
Model description: Two-arm, 1:1 randomised, open-label, parallel-group phase 2 trial comparing toripalimab plus sequential intravesical gemcitabine→mitomycin C with toripalimab alone in adults with BCG-unresponsive or BCG-intolerant high-risk NMIBC. Randomisation stratified by CIS status, stage (Ta vs T1), BCG failure subtype, and study centre. Treatment up to 24 months.

Arm	Type	Receives
Combination: Toripalimab + Intravesical GEM-MMC	Experimental	Drug: Toripalimab, Drug: Gemcitabine (GEM), Drug: Mitomycin C (MMC)Toripalimab IV Q3W starting with the first intravesical cycle, plus sequential intravesical gemcitabine followed by mitomycin C in the same visit. Induction weekly ×6; if no recurrence at first tumour assessment (~month 3), monthly maintenance up to 24 months.
Monotherapy: Toripalimab Alone	Active comparator	Drug: ToripalimabToripalimab IV every 3 weeks (Q3W) up to 24 months or until disease progression, unacceptable toxicity, or withdrawal; no intravesical chemotherapy.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (12 listed)

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:
Adequate BCG, for the purposes of this protocol, is defined as receipt of at least 5 of 6 induction instillations (maintenance not required).
ECOG performance status 0-2.

Key exclusion criteria (13 listed)

Muscle-invasive bladder cancer (T2-T4).
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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ToripalimabAlso: JS001, TuoyiPD-1 inhibitor administered intravenously every 3 weeks (Q3W) for up to 24 months. Starts during the first intravesical treatment cycle.	Drug	Combination: Toripalimab + Intravesical GEM-MMC, Monotherapy: Toripalimab Alone
Gemcitabine (GEM)Also: GEMIntravesical instillation as part of a sequential regimen with mitomycin C: weekly for 6 weeks (induction); if no recurrence at first tumour assessment (~month 3), maintenance instillations continue monthly up to 24 months.	Drug	Combination: Toripalimab + Intravesical GEM-MMC
Mitomycin C (MMC)Intravesical instillation immediately after intravesical gemcitabine in the same visit (sequential regimen): weekly for 6 weeks (induction); if eligible, monthly maintenance up to 24 months.	Drug	Combination: Toripalimab + Intravesical GEM-MMC

Primary outcome

Complete Response (CR) Rate at Month 3 in the CIS Cohort
Time frame: Baseline to Month 3
Proportion of participants in the CIS cohort achieving CR at month 3. CR is met by any of the following protocol-specified scenarios indicating no high-grade bladder disease: (a) negative urine cytology and negative cystoscopy; (b) negative cytology with cystoscopic lesions that are benign or low-grade Ta on biopsy; or (c) negative cystoscopy with positive cytology attributed to tumour in the upper tract or prostatic urethra and random bladder biopsies negative.
Median Recurrence-Free Survival (RFS) in the Non-CIS Cohort
Time frame: From randomisation/start of treatment to first event, assessed up to 24 months
RFS is time to high-grade Ta recurrence, any T1, or new CIS, whichever occurs first. Recurrence requires cystoscopic suspicion confirmed by histopathology; if cytology becomes positive before histologic confirmation, the recurrence date is set at the first positive cytology once recurrence is subsequently confirmed. Death from any cause before documented recurrence is counted as an event.

9 secondary outcomes

CR Rates at Months 6, 12, 18, and 24 in the CIS Cohort
Time frame: Baseline to months 6, 12, 18, and 24
Progression-Free Survival (PFS) Rates at Months 6, 12, 18, and 24 in the CIS Cohort
Time frame: Baseline to months 6, 12, 18, and 24
Overall Survival at Month 24, EOT+6 Months, and EOT+12 Months in the CIS Cohort
Time frame: Baseline to Month 24, EOT+6 months, and EOT+12 months
Incidence of Adverse Events in the CIS Cohort
Time frame: From first dose/instillation to 90 days post-last dose/instillation (≈ up to 27 months)
Recurrence-Free Survival Rates at Months 6, 12, 18, and 24 in the Non-CIS Cohort
Time frame: Baseline to months 6, 12, 18, and 24
High-Grade Recurrence-Free Survival (HG-RFS) Rates at Months 6, 12, 18, and 24 in the Non-CIS Cohort
Time frame: Baseline to months 6, 12, 18, and 24
Progression-Free Survival Rates at Months 6, 12, 18, and 24 in the Non-CIS Cohort
Time frame: Baseline to Months 6, 12, 18, and 24
Overall Survival at Month 24, EOT+6 Months, and EOT+12 Months in the Non-CIS Cohort
Time frame: Baseline to Month 24, EOT+6 months, and EOT+12 months
Incidence of Adverse Events in the Non-CIS Cohort
Time frame: From first dose/instillation to 90 days post-last dose/instillation (≈ up to 27 months)

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2026.
Results publication: PubMed 38265030Holzbeierlein JM, Bixler BR, Buckley DI, Chang SS, Holmes R, James AC, Kirkby E, McKiernan JM, Schuckman AK. Diagnosis and Treatment of Non-Muscle Invasive Bladder Cancer: AUA/SUO Guideline: 2024 Amendment. J Urol. 2024 Apr;211(4):533-538. doi: 10.1097/JU.0000000000003846. Epub 2024 Jan 24.
Results publication: PubMed 23510863Lightfoot AJ, Breyer BN, Rosevear HM, Erickson BA, Konety BR, O'Donnell MA. Multi-institutional analysis of sequential intravesical gemcitabine and mitomycin C chemotherapy for non-muscle invasive bladder cancer. Urol Oncol. 2014 Jan;32(1):35.e15-9. doi: 10.1016/j.urolonc.2013.01.009. Epub 2013 Mar 17.
Results publication: PubMed 19171491Breyer BN, Whitson JM, Carroll PR, Konety BR. Sequential intravesical gemcitabine and mitomycin C chemotherapy regimen in patients with non-muscle invasive bladder cancer. Urol Oncol. 2010 Sep-Oct;28(5):510-4. doi: 10.1016/j.urolonc.2008.11.019. Epub 2009 Jan 26.
Results publication: PubMed 37596191Black PC, Tangen CM, Singh P, McConkey DJ, Lucia MS, Lowrance WT, Koshkin VS, Stratton KL, Bivalacqua TJ, Kassouf W, Porten SP, Bangs R, Plets M, Thompson IM Jr, Lerner SP. Phase 2 Trial of Atezolizumab in Bacillus Calmette-Guerin-unresponsive High-risk Non-muscle-invasive Bladder Cancer: SWOG S1605. Eur Urol. 2023 Dec;84(6):536-544. doi: 10.1016/j.eururo.2023.08.004. Epub 2023 Aug 16.
Results publication: PubMed 38740030Necchi A, Roumiguie M, Kamat AM, Shore ND, Boormans JL, Esen AA, Lebret T, Kandori S, Bajorin DF, Krieger LEM, Niglio SA, Uchio EM, Seo HK, de Wit R, Singer EA, Grivas P, Nishiyama H, Li H, Baranwal P, Van den Sigtenhorst-Fijlstra M, Kapadia E, Kulkarni GS. Pembrolizumab monotherapy for high-risk non-muscle-invasive bladder cancer without carcinoma in situ and unresponsive to BCG (KEYNOTE-057): a single-arm, multicentre, phase 2 trial. Lancet Oncol. 2024 Jun;25(6):720-730. doi: 10.1016/S1470-2045(24)00178-5. Epub 2024 May 10.
Results publication: PubMed 34051177Balar AV, Kamat AM, Kulkarni GS, Uchio EM, Boormans JL, Roumiguie M, Krieger LEM, Singer EA, Bajorin DF, Grivas P, Seo HK, Nishiyama H, Konety BR, Li H, Nam K, Kapadia E, Frenkl T, de Wit R. Pembrolizumab monotherapy for the treatment of high-risk non-muscle-invasive bladder cancer unresponsive to BCG (KEYNOTE-057): an open-label, single-arm, multicentre, phase 2 study. Lancet Oncol. 2021 Jul;22(7):919-930. doi: 10.1016/S1470-2045(21)00147-9. Epub 2021 May 26.
EAU Guidelines on Non-muscle-invasive Bladder Cancer (TaT1 and CIS)Link provided by the sponsor.
Individual participant dataSharing planned: Undecided. The sponsor and investigators intend to share de-identified IPD but the final plan (scope, timing, and access mechanism) is pending institutional approvals. This record will be updated once the IPD sharing policy is finalised.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.

Qi Lin, MM contact

Wei Chen, MD contact

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Qi Lin, MM (+8615205771010, devillynch@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Complete Response (CR) Rate at Month 3 in the CIS Cohort (time frame: Baseline to Month 3). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07189793, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Sep 2026.