Urothelial CarcinomaPhase 2NCT04553939
Neoadjuvant Toripalimab in Combination With Gemcitabine Therapy in Cisplatin Ineligible Local Advanved Bladder Cancer
A Phase 2 study testing Toripalimab and Gemcitabine in people with Urothelial Carcinoma and Bladder Cancer, run by Henan Cancer Hospital at 1 site in China.
Trial summary
This is a pre-surgical study involving subjects with muscle invasive bladder urothelial cancer, who are candidates for cisplatin ineligible neoadjuvant therapy. It is a one-arm phase II study in single center. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Urothelial Carcinoma, Bladder Cancer
- What is being tested?
- Toripalimab and Gemcitabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 4 Jan 2021to 12 Apr 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Be willing and able to provide written informed consent for the trial.
- 18-75 years of age on day of signing informed consent.
- Have histologically confirmed muscle invasive disease of the urinary bladder.
- Histology must be urothelial carcinoma (transitional cell carcinoma) or urothelial carcinoma with mixed histology/features.
- Clinical stage II-IIIB and Have a surgical evaluation that documents the plan for multimodality therapy with a consolidative radical cystectomy.
- ECOG 0-1 and good organ function.
- cisplatin ineligible.
Who cannot join
- A non-surgical approach recommended by the treating urologist due to any reason.
- Is currently participating in or has participated in a study of an investigational agent or using an investigational device within 28 days prior to study registration.
- Has a diagnosis of immunodeficiency or received systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to study registration.
- Has a known additional malignancy that is progressing or required treatment ≤ 48 months of study registration. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin, in situ cervical cancer.
- Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis.
- Has an active autoimmune disease requiring systemic treatment.
Show all 14Show fewer
- Has known evidence of interstitial lung disease or active, non-infectious pneumonitis.
- Has an active infection requiring systemic therapy.
- Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator
- Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
- Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment.
- Has received prior therapy with an anti-PD-1, anti-PD-L1, antibody.
- Has a known history of Human Immunodeficiency Virus.
- Has known active Hepatitis B or Hepatitis C.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase II Study of Neoadjuvant Toripalimab in Combination With Gemcitabine Therapy in Cisplatin Ineligible Stage II-IIIB Bladder Cancer
This is a pre-surgical study involving subjects with muscle invasive bladder urothelial cancer, who are candidates for cisplatin ineligible neoadjuvant therapy. It is a one-arm phase II study in single center.
- Conditions
- Urothelial Carcinoma, Bladder Cancer
- Being tested
- Toripalimab, Gemcitabine
- Keywords
- Toripalimab, Neoadjuvant therapy
- Medical subject headings
- Carcinoma, Transitional Cell, Urinary Bladder Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ToripalimabDrug
Toripalimab 3mg/m2 ivgtt, D1 every 14 days for 2-4 cycles.
GemcitabineDrug
Gemcitabine 800mg/m2 ivgtt, D1 every 14 days for 2-4 cycles.
| Study group | Role | What they receive |
|---|---|---|
| Toripalimab in Combination With Gemcitabine Therapy | Experimental | Not described Receives: Drug: Toripalimab; Drug: Gemcitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR，objective response rate
ORR was defined as the percentage of participants in the analysis population who have a CR (disappearance of all target lesions) or a PR (≥30% decrease in the sum of diameters of target lesions) based upon RECIST 1.1.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2020
- Study start4 Jan 2021
- Primary completion12 Apr 2023
- Study completion12 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Urothelial CarcinomaStudies of the same condition, open and under way.
- More trials run by Henan Cancer HospitalThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (actual)
Sponsor and investigators
- Lead sponsor
- Henan Cancer Hospital
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- zhao Shiming, Principal investigator, Henan Cancer Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04553939
- Sponsor's protocol ID
- 2020051314
- Status last verified
- Apr 2025
- First submitted
- 13 Sep 2020
- Last updated
- 2 Jan 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
von der Maase H, Sengelov L, Roberts JT, Ricci S, Dogliotti L, Oliver T, Moore MJ, Zimmermann A, Arning M. Long-term survival results of a randomized trial comparing gemcitabine plus cisplatin, with methotrexate, vinblastine, doxorubicin, plus cisplatin in patients with bladder cancer. J Clin Oncol. 2005 Jul 20;23(21):4602-8. doi: 10.1200/JCO.2005.07.757.
PubMed 16034041Forde PM, Chaft JE, Smith KN, Anagnostou V, Cottrell TR, Hellmann MD, Zahurak M, Yang SC, Jones DR, Broderick S, Battafarano RJ, Velez MJ, Rekhtman N, Olah Z, Naidoo J, Marrone KA, Verde F, Guo H, Zhang J, Caushi JX, Chan HY, Sidhom JW, Scharpf RB, White J, Gabrielson E, Wang H, Rosner GL, Rusch V, Wolchok JD, Merghoub T, Taube JM, Velculescu VE, Topalian SL, Brahmer JR, Pardoll DM. Neoadjuvant PD-1 Blockade in Resectable Lung Cancer. N Engl J Med. 2018 May 24;378(21):1976-1986. doi: 10.1056/NEJMoa1716078. Epub 2018 Apr 16.
PubMed 29658848Balar AV, Castellano D, O'Donnell PH, Grivas P, Vuky J, Powles T, Plimack ER, Hahn NM, de Wit R, Pang L, Savage MJ, Perini RF, Keefe SM, Bajorin D, Bellmunt J. First-line pembrolizumab in cisplatin-ineligible patients with locally advanced and unresectable or metastatic urothelial cancer (KEYNOTE-052): a multicentre, single-arm, phase 2 study. Lancet Oncol. 2017 Nov;18(11):1483-1492. doi: 10.1016/S1470-2045(17)30616-2. Epub 2017 Sep 26.
PubMed 28967485Wang Z, Till B, Gao Q. Chemotherapeutic agent-mediated elimination of myeloid-derived suppressor cells. Oncoimmunology. 2017 Jun 16;6(7):e1331807. doi: 10.1080/2162402X.2017.1331807. eCollection 2017.
PubMed 28811975Galluzzi L, Zitvogel L, Kroemer G. Immunological Mechanisms Underneath the Efficacy of Cancer Therapy. Cancer Immunol Res. 2016 Nov;4(11):895-902. doi: 10.1158/2326-6066.CIR-16-0197.
PubMed 27803050
Study at a glance
From the registry record, last updated by the sponsor on 2 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Neoadjuvant Toripalimab in Combination With Gemcitabine Therapy in Cisplatin Ineligible Stage II-IIIB Bladder Cancer
- ClinicalTrials.gov ID
- NCT04553939
- Acronym / sponsor ID
- 2020051314
- Conditions
- Urothelial Carcinoma, Bladder Cancer
- Keywords
- Toripalimab, Neoadjuvant therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Henan Cancer HospitalOther government
- Study start
- 4 Jan 2021 (actual)
- Primary completion
- 12 Apr 2023 (actual)
- Study completion
- 12 Apr 2025 (actual)
- First posted
- 18 Sep 2020 (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.
This is a pre-surgical study involving subjects with muscle invasive bladder urothelial cancer, who are candidates for cisplatin ineligible neoadjuvant therapy. It is a one-arm phase II study in single center.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Toripalimab in Combination With Gemcitabine Therapy | Experimental | Drug: Toripalimab, Drug: Gemcitabine |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Be willing and able to provide written informed consent for the trial.
- 18-75 years of age on day of signing informed consent.
- Have histologically confirmed muscle invasive disease of the urinary bladder.
- Histology must be urothelial carcinoma (transitional cell carcinoma) or urothelial carcinoma with mixed histology/features.
- Clinical stage II-IIIB and Have a surgical evaluation that documents the plan for multimodality therapy with a consolidative radical cystectomy.
Key exclusion criteria (14 listed)
- A non-surgical approach recommended by the treating urologist due to any reason.
- Is currently participating in or has participated in a study of an investigational agent or using an investigational device within 28 days prior to study registration.
- Has a diagnosis of immunodeficiency or received systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to study registration.
- Has a known additional malignancy that is progressing or required treatment ≤ 48 months of study registration. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin, in situ cervical cancer.
- Has known active central nervous system (CNS) metastases and/or carcinomatous meningitis.
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 |
|---|---|---|
| ToripalimabToripalimab 3mg/m2 ivgtt, D1 every 14 days for 2-4 cycles. | Drug | Toripalimab in Combination With Gemcitabine Therapy |
| GemcitabineGemcitabine 800mg/m2 ivgtt, D1 every 14 days for 2-4 cycles. | Drug | Toripalimab in Combination With Gemcitabine Therapy |
Primary outcome
ORR，objective response rate
Time frame: 3 years
ORR was defined as the percentage of participants in the analysis population who have a CR (disappearance of all target lesions) or a PR (≥30% decrease in the sum of diameters of target lesions) based upon RECIST 1.1.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jan 2026.
- Background: PubMed 16034041von der Maase H, Sengelov L, Roberts JT, Ricci S, Dogliotti L, Oliver T, Moore MJ, Zimmermann A, Arning M. Long-term survival results of a randomized trial comparing gemcitabine plus cisplatin, with methotrexate, vinblastine, doxorubicin, plus cisplatin in patients with bladder cancer. J Clin Oncol. 2005 Jul 20;23(21):4602-8. doi: 10.1200/JCO.2005.07.757.
- Background: PubMed 29658848Forde PM, Chaft JE, Smith KN, Anagnostou V, Cottrell TR, Hellmann MD, Zahurak M, Yang SC, Jones DR, Broderick S, Battafarano RJ, Velez MJ, Rekhtman N, Olah Z, Naidoo J, Marrone KA, Verde F, Guo H, Zhang J, Caushi JX, Chan HY, Sidhom JW, Scharpf RB, White J, Gabrielson E, Wang H, Rosner GL, Rusch V, Wolchok JD, Merghoub T, Taube JM, Velculescu VE, Topalian SL, Brahmer JR, Pardoll DM. Neoadjuvant PD-1 Blockade in Resectable Lung Cancer. N Engl J Med. 2018 May 24;378(21):1976-1986. doi: 10.1056/NEJMoa1716078. Epub 2018 Apr 16.
- Background: PubMed 28967485Balar AV, Castellano D, O'Donnell PH, Grivas P, Vuky J, Powles T, Plimack ER, Hahn NM, de Wit R, Pang L, Savage MJ, Perini RF, Keefe SM, Bajorin D, Bellmunt J. First-line pembrolizumab in cisplatin-ineligible patients with locally advanced and unresectable or metastatic urothelial cancer (KEYNOTE-052): a multicentre, single-arm, phase 2 study. Lancet Oncol. 2017 Nov;18(11):1483-1492. doi: 10.1016/S1470-2045(17)30616-2. Epub 2017 Sep 26.
- Background: PubMed 28811975Wang Z, Till B, Gao Q. Chemotherapeutic agent-mediated elimination of myeloid-derived suppressor cells. Oncoimmunology. 2017 Jun 16;6(7):e1331807. doi: 10.1080/2162402X.2017.1331807. eCollection 2017.
- Background: PubMed 27803050Galluzzi L, Zitvogel L, Kroemer G. Immunological Mechanisms Underneath the Efficacy of Cancer Therapy. Cancer Immunol Res. 2016 Nov;4(11):895-902. doi: 10.1158/2326-6066.CIR-16-0197.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
zhao Shiming principal investigator
Henan Cancer Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Toripalimab and Gemcitabine in people with Urothelial Carcinoma and Bladder Cancer, run by Henan Cancer Hospital at 1 site in China.
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 7 inclusion and 14 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: finished, as of the sponsor's last update on 2 Jan 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ORR，objective response rate (time frame: 3 years). 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 NCT04553939, which also lists 5 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 2 Jan 2026.