UTUCPhase 2NCT06927128
Neoadjuvant Chemotherapy in High - Risk Upper Tract Urothelial Carcinoma
A Phase 2 study testing Gemcitabine, Cisplatin in people with UTUC and NAC, run by Changhai Hospital at 1 site in China.
Trial summary
Upper urinary tract urothelial carcinoma (UTUC) represents a rare yet aggressive malignancy associated with a dismal prognosis. At the point of diagnosis, nearly half of the patients already have invasive disease, and over 70% present with high-grade UTUC. Read more about this study
- Who is it for?
- Ages 18 years and older
- What is being tested?
- Gemcitabine, Cisplatin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 1 siteChina
- Study dates
- 15 May 2025to 15 Jun 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
- Histologically confirmed high - risk upper tract urothelial carcinoma (UTUC). For mixed - type tumors, the histological type should be mainly urothelial carcinoma (≥50%), or urine cytology is positive and imaging diagnosis supports UC.
- Clinically non - metastatic urothelial carcinoma (N≤1 M0), determined by imaging examinations (CT or MRI) of the chest/abdomen/pelvis.
- Patients must plan to undergo radical nephroureterectomy (RNU).
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1.
- Patients are potential beneficiaries of cisplatin - based neoadjuvant chemotherapy, with relatively good renal function (GFR≥45ml/min) and able to tolerate drug treatment and surgery.
- Sufficient organ and bone marrow function determined by screening tests.
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- Recovered from any reversible toxicity of previous surgery.
- At least 18 years old on the date of registration.
- Informed of the study nature and signed the informed consent form.
Who cannot join
- Imaging - identified ≥N2 disease or metastatic disease (M1).
- History of invasive or lymph node - positive or metastatic urothelial carcinoma or invasive contralateral upper tract carcinoma within 2 years before registration.
- Patients with only one kidney, or cisplatin intolerant patients.
- Participating in other interventional clinical trials at the time of registration.
- History of non - urothelial malignancies, except those who have been disease - free for at least 1 year as judged by the treating oncologist. History of adequately treated (at the discretion of the treating oncologist) basal cell or squamous cell skin cancer or in - situ cervical cancer is allowed.
- Pregnant or lactating women. Reproductive - potential women/men must agree to use effective contraception methods.
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- Any other medical conditions that make the treating doctor consider their participation in the study inappropriate.
- Withdrawal/Termination Criteria
- Investigator - determined Withdrawal:
- Tumor recurrence or progression during the clinical trial, and the investigator determines that the subject needs new treatment (such as immediate tumor resection or systemic anti - tumor treatment) and is not suitable to continue the trial. If the investigator judges that the subject can still receive treatment or follow - up for efficacy after tumor recurrence, the subject may not be withdrawn for the time being.
- The subject develops comorbidities, complications, or special physiological changes that are not suitable for continuing the trial.
- Poor compliance of the subject affecting safety and efficacy evaluation:
- The subject does not take medicine or undergo examinations as required.
- The subject uses other drugs or foods that affect safety evaluation.
- The subject has other behaviors that affect the test results.
- Unbearable toxicity, other adverse events, or serious adverse events occur, and the subject is not suitable to continue the trial.
- A grade 3 adverse event related to the study drug occurs and persists for more than 12 days after taking sufficient treatment measures.
- Systemic metastasis or progression is detected during the drug - administration observation period or the surgical period.
- Subject - initiated Withdrawal: Subjects have the right to withdraw from the trial midway according to the informed consent form. If the subject does not withdraw the informed consent but no longer accepts trial drug administration and examinations or is lost to follow - up, it is also regarded as withdrawal. All withdrawn subjects should try to complete the corresponding observation content of the last visit as specified in the protocol. The reason for termination should be noted in the original document. If the subject withdraws due to an adverse event, appropriate treatment should be provided and recorded in the original record.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Changhai hospital
Shanghai, Shanghai Municipality, 200433
Shuxiong Zeng, +86 18930568759, zengshuxiong@126.com
About this study
Official title: Single - Center Phase II Clinical Study: Efficacy Evaluation of Neoadjuvant Chemotherapy With Gemcitabine and Cisplatin in High - Risk Upper Tract Urothelial Carcinoma
Upper urinary tract urothelial carcinoma (UTUC) represents a rare yet aggressive malignancy associated with a dismal prognosis. At the point of diagnosis, nearly half of the patients already have invasive disease, and over 70% present with high-grade UTUC. Currently, radical nephroureterectomy (RNU) remains the gold standard of care for high-risk UTUC.
Previous investigations have demonstrated that, in contrast to RNU alone, chemotherapy can effectively reduce the disease recurrence rate and mortality. Moreover, it may confer benefits to patients' overall survival (OS) without impeding the implementation of subsequent definitive surgical treatment. However, the majority of these studies are predominantly retrospective analyses. Although they can, to some degree, reflect the clinical value of neoadjuvant chemotherapy, due to inherent limitations in study design and other confounding factors, there is still a paucity of prospective research evidence for further validation.
Considering that RNU can cause a decline in renal function in patients, and in light of prospective trial outcomes, preoperative neoadjuvant chemotherapy (NAC) has emerged as a preferred treatment option for chemotherapy-eligible UTUC patients.
- Being tested
- Gemcitabine, Cisplatin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gemcitabine, CisplatinDrug
Gemcitabine (1000mg/m²) and cisplatin (70mg/m²). Gemcitabine is used on the 1st and 8th days of a 21 - day cycle, and cisplatin is used on the 2nd day of the cycle. A total of 3 - 4 cycles are carried out.A dose reduction to 60% of the original dose (adjusted to 0.6 times the initial dose) will be implemented if deemed clinically necessary, contingent upon meeting either of the following criteria: 1) occurrence of grade 3 or higher treatment-related adverse events as per CTCAE v5.0 guidelines, or 2) evidence of renal impairment manifested by a ≥40% decline in glomerular filtration rate (GFR) from baseline measurements.
| Study group | Role | What they receive |
|---|---|---|
| Neoadjuvant Gemcitabine Plus Cisplatin Chemotherapy Group | Experimental | Participants receive intravenous gemcitabine (1000 mg/m²) on days 1 and 8, followed by intravenous cisplatin (70 mg/m²) on day 1, every 3 weeks for 4 cycles. This neoadjuvant chemotherapy is administered prior to planned radical nephroureterectomy. Receives: Drug: Gemcitabine, Cisplatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pathological response rate（pRR）
defined as postoperative pathology <ypT2N0
Measured: At the time of pathological report issuance，approximately within 7-10 business days after surgery
Secondary outcomes (6)
Pathological complete response rate (pCR)
defined as postoperative pathology <ypT1N0
Measured: At the time of pathological report issuance，approximately within 7-10 business days after surgery
Imaging response rate
defined as tumor size reduction observed on imaging comparisons between pre- and post-chemotherapy assessments, with any decrease classified as a positive response.Tumor size reduction, measured as the change in the longest diameter of the primary tumor via computed tomography (CT) scan according to the Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 guidelines.
Measured: From the date of the first chemotherapy administration until 2 weeks after the last chemotherapy session, assessed up to 11-14 weeks.
Surgery completion rate
Proportion of patients undergoing surgery
Measured: From chemotherapy initiation to surgical resection.Patients should undergo definitive surgery within 6 weeks ( ± 2 weeks) following completion of neoadjuvant chemotherapy
Overall survival (OS)
defined as the duration from the date of surgical resection to the occurrence of death from any cause
Measured: From the date of surgery until the date of death or loss to follow-up, with a maximum follow-up period of 2 years.
Cancer - specific survival (CSS)
defined as the time from diagnosis to death directly attributable to the primary cancer or its metastases, excluding mortality from unrelated causes
Measured: From the date of diagnosis until the date of death from urothelial carcinoma or last documented follow-up, with a maximum follow-up period of 2 years.
Recurrence - free survival (RFS)
defined as the time from post-surgery to the first documented evidence of local/regional recurrence, distant metastasis, or death from any cause, whichever occurs first
Measured: From the date of surgical resection until the date of disease recurrence (radiographically or pathologically confirmed) or death from any cause, whichever occurs first, with a maximum follow-up of 2 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2025
- Study start15 May 2025 (estimated)
- Primary completion15 May 2026 (estimated)
- Study completion15 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for UTUCStudies of the same condition, open and under way.
- More trials run by Changhai 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
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Changhai Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06927128
- Sponsor's protocol ID
- CH-NAC-UTUC
- Status last verified
- Apr 2025
- First submitted
- 16 Mar 2025
- Last updated
- 15 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single - Center Phase II Clinical Study: Efficacy Evaluation of Neoadjuvant Chemotherapy With Gemcitabine and Cisplatin in High - Risk Upper Tract Urothelial Carcinoma
- ClinicalTrials.gov ID
- NCT06927128
- Acronym / sponsor ID
- CH-NAC-UTUC
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Changhai HospitalUniversity, hospital or other
- Study start
- 15 May 2025 (estimated)
- Primary completion
- 15 May 2026 (estimated)
- Study completion
- 15 Jun 2028 (estimated)
- First posted
- 15 Apr 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.
Upper urinary tract urothelial carcinoma (UTUC) represents a rare yet aggressive malignancy associated with a dismal prognosis. At the point of diagnosis, nearly half of the patients already have invasive disease, and over 70% present with high-grade UTUC. Currently, radical nephroureterectomy (RNU) remains the gold standard of care for high-risk UTUC.
Previous investigations have demonstrated that, in contrast to RNU alone, chemotherapy can effectively reduce the disease recurrence rate and mortality. Moreover, it may confer benefits to patients' overall survival (OS) without impeding the implementation of subsequent definitive surgical treatment. However, the majority of these studies are predominantly retrospective analyses. Although they can, to some degree, reflect the clinical value of neoadjuvant chemotherapy, due to inherent limitations in study design and other confounding factors, there is still a paucity of prospective research evidence for further validation.
Considering that RNU can cause a decline in renal function in patients, and in light of prospective trial outcomes, preoperative neoadjuvant chemotherapy (NAC) has emerged as a preferred treatment option for chemotherapy-eligible UTUC patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Neoadjuvant Gemcitabine Plus Cisplatin Chemotherapy Group | Experimental | Drug: Gemcitabine, CisplatinParticipants receive intravenous gemcitabine (1000 mg/m²) on days 1 and 8, followed by intravenous cisplatin (70 mg/m²) on day 1, every 3 weeks for 4 cycles. This neoadjuvant chemotherapy is administered prior to planned radical nephroureterectomy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Histologically confirmed high - risk upper tract urothelial carcinoma (UTUC). For mixed - type tumors, the histological type should be mainly urothelial carcinoma (≥50%), or urine cytology is positive and imaging diagnosis supports UC.
- Clinically non - metastatic urothelial carcinoma (N≤1 M0), determined by imaging examinations (CT or MRI) of the chest/abdomen/pelvis.
- Patients must plan to undergo radical nephroureterectomy (RNU).
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1.
- Patients are potential beneficiaries of cisplatin - based neoadjuvant chemotherapy, with relatively good renal function (GFR≥45ml/min) and able to tolerate drug treatment and surgery.
Key exclusion criteria (19 listed)
- Imaging - identified ≥N2 disease or metastatic disease (M1).
- History of invasive or lymph node - positive or metastatic urothelial carcinoma or invasive contralateral upper tract carcinoma within 2 years before registration.
- Patients with only one kidney, or cisplatin intolerant patients.
- Participating in other interventional clinical trials at the time of registration.
- History of non - urothelial malignancies, except those who have been disease - free for at least 1 year as judged by the treating oncologist. History of adequately treated (at the discretion of the treating oncologist) basal cell or squamous cell skin cancer or in - situ cervical cancer is allowed.
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 |
|---|---|---|
| Gemcitabine, CisplatinGemcitabine (1000mg/m²) and cisplatin (70mg/m²). Gemcitabine is used on the 1st and 8th days of a 21 - day cycle, and cisplatin is used on the 2nd day of the cycle. A total of 3 - 4 cycles are carried out.A dose reduction to 60% of the original dose (adjusted to 0.6 times the initial dose) will be implemented if deemed clinically necessary, contingent upon meeting either of the following criteria: 1) occurrence of grade 3 or higher treatment-related adverse events as per CTCAE v5.0 guidelines, or 2) evidence of renal impairment manifested by a ≥40% decline in glomerular filtration rate (GFR) from baseline measurements. | Drug | Neoadjuvant Gemcitabine Plus Cisplatin Chemotherapy Group |
Primary outcome
Pathological response rate（pRR）
Time frame: At the time of pathological report issuance，approximately within 7-10 business days after surgery
defined as postoperative pathology <ypT2N0
6 secondary outcomes
Pathological complete response rate (pCR)
Time frame: At the time of pathological report issuance，approximately within 7-10 business days after surgery
Imaging response rate
Time frame: From the date of the first chemotherapy administration until 2 weeks after the last chemotherapy session, assessed up to 11-14 weeks.
Surgery completion rate
Time frame: From chemotherapy initiation to surgical resection.Patients should undergo definitive surgery within 6 weeks ( ± 2 weeks) following completion of neoadjuvant chemotherapy
Overall survival (OS)
Time frame: From the date of surgery until the date of death or loss to follow-up, with a maximum follow-up period of 2 years.
Cancer - specific survival (CSS)
Time frame: From the date of diagnosis until the date of death from urothelial carcinoma or last documented follow-up, with a maximum follow-up period of 2 years.
Recurrence - free survival (RFS)
Time frame: From the date of surgical resection until the date of disease recurrence (radiographically or pathologically confirmed) or death from any cause, whichever occurs first, with a maximum follow-up of 2 years.
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 15 Apr 2025.
- Individual participant dataSharing planned: Undecided
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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.
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 Gemcitabine, Cisplatin in people with UTUC and NAC, run by Changhai 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 9 inclusion and 19 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 15 Apr 2025. 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 Shuxiong Zeng (+86 18930568759, zengshuxiong@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pathological response rate（pRR） (time frame: At the time of pathological report issuance，approximately within 7-10 business days after surgery). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Apr 2025.