UTUCNCT07155044
A Comparative Study of Neoadjuvant Chemotherapy Versus Upfront Radical Surgery for Upper Tract Urothelial Carcinoma
An observational study of UTUC and UC (Urothelial Cancer), run by Changhai Hospital at 1 site in China.
Trial summary
This is a single-center, open-label, non-randomized controlled trial comparing the efficacy and safety of neoadjuvant chemotherapy (NAC) followed by radical surgery versus upfront radical surgery alone in patients with high-risk, non-metastatic upper tract urothelial carcinoma (UTUC). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- UTUC, UC (Urothelial Cancer)
- What is being tested?
- NAC
- How many people
- 120 participants
- Where
- 1 siteChina
- Study dates
- 15 May 2024to 15 May 2029
- Phase
- Observational
- 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
Study population: The study population will consist of adult patients (≥18 years) with histologically confirmed, non-metastatic upper tract urothelial carcinoma (UTUC) who are planned for radical nephroureterectomy (RNU). Participants will be recruited from the urology oncology clinics at a single tertiary care center (e.g., The First Affiliated Hospital of Naval Military Medical University). Eligibility will be determined based on predefined inclusion and exclusion criteria, focusing on patients with adequate renal function (GFR ≥45 mL/min) and performance status (ECOG 0-1) to be potential candidates for cisplatin-based chemotherapy. The population will include both patients who are deemed eligible and choose to receive neoadjuvant chemotherapy prior to surgery and those who proceed directly to surgery.
Who may be able to join
- Histologically confirmed upper tract urothelial carcinoma (UTUC). For mixed histology, urothelial carcinoma must be the predominant component (≥50%).
- Clinically non-metastatic disease (cN≤1, M0) as determined by cross-sectional imaging (CT or MRI of chest/abdomen/pelvis).
- Planned treatment with radical nephroureterectomy (RNU).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
- Adequate renal function, defined as glomerular filtration rate (GFR) ≥ 45 mL/min, making the patient a potential candidate for cisplatin-based neoadjuvant chemotherapy.
- Adequate organ and bone marrow function as determined by standard screening tests.
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- Recovery from all reversible toxicities of any prior surgery.
- Age ≥ 18 years at the time of enrollment.
- Ability to understand the study and provide signed informed consent.
Who cannot join
- Radiographic evidence of ≥cN2 lymph node disease or distant metastases (M1).
- History of invasive, lymph node-positive, or metastatic urothelial carcinoma within 2 years prior to enrollment, or history of invasive contralateral UTUC.
- Presence of only a solitary kidney or cisplatin ineligibility.
- Concurrent participation in another interventional clinical trial at the time of enrollment.
- History of a non-urothelial malignant tumor unless the patient has been disease-free for at least 1 year (exceptions include adequately treated basal cell or squamous cell skin cancer, or carcinoma in situ of the cervix).
- Pregnancy or lactation. Women and men of reproductive potential must agree to use effective contraception.
- Any other medical condition, comorbidity, or psychiatric illness that, in the investigator's judgment, would make the patient an unsuitable candidate for the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluating the Application Value of Neoadjuvant Chemotherapy Compared to Upfront Surgery in Patients With Upper Tract Urothelial Carcinoma
This is a single-center, open-label, non-randomized controlled trial comparing the efficacy and safety of neoadjuvant chemotherapy (NAC) followed by radical surgery versus upfront radical surgery alone in patients with high-risk, non-metastatic upper tract urothelial carcinoma (UTUC).
The study aims to answer the following key questions:
Read more
Does NAC improve pathologic response rates (defined as downstaging to <ypT2N0) compared to immediate surgery? What is the rate of pathologic complete response (pCR; ypT0N0) in the NAC group? How do the two treatment strategies compare in terms of overall survival, cancer-specific survival, and recurrence-free survival? What is the safety and tolerability profile of NAC using gemcitabine and cisplatin in this patient population? Eligible participants will be assigned to either the NAC-plus-surgery group or the surgery-only group based on clinical evaluation and patient preference. The study will also explore potential biomarkers (e.g., chromosomal instability in liquid biopsies) for predicting treatment response.
Key Eligibility Criteria:
Adults with histologically confirmed high-risk UTUC Clinical stage ≤N1 M0 Adequate renal function (GFR ≥45 mL/min) and ECOG performance status 0-1
Primary Outcome:
Pathologic response rate (proportion of patients with <ypT2N0)
- Conditions
- UTUC, UC (Urothelial Cancer)
- Being tested
- NAC
- Keywords
- NAC
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NACDrug
Participants in NAC+RNU cohort will receive 3-4 cycles of neoadjuvant chemotherapy (NAC) with the gemcitabine and cisplatin (GC) regimen prior to undergoing definitive surgery. Gemcitabine (1000 mg/m²) will be administered on Day 1 and Day 8, and cisplatin (70 mg/m²) will be administered on Day 2 of each 21-day cycle.
| Study group | Role | What they receive |
|---|---|---|
| NAC+RNU | Intervention: Participants in this cohort will receive 3-4 cycles of neoadjuvant chemotherapy (NAC) with the gemcitabine and cisplatin (GC) regimen prior to undergoing definitive surgery. Gemcitabine (1000 mg/m²) will be administered on Day 1 and Day 8, and cisplatin (70 mg/m²) will be administered on Day 2 of each 21-day cycle.
Surgical Intervention: Following the completion of NAC, patients will undergo a radical nephroureterectomy (RNU) with bladder cuff excision. The procedure will be performed via robot-assisted (Da Vinci), laparoscopic, or open approach, based on surgical discretion. Surgery must be performed within 6 weeks (±2 weeks) after the last dose of chemotherapy.
Objective: The primary objective for this cohort is to evaluate the efficacy of NAC in inducing pathological downstaging (defined as achieving pathological stage <ypT2N0 at surgery) and to assess the pathological complete response (pCR) rate (ypT0N0). Receives: Drug: NAC | |
| RNU | Intervention: Participants in this cohort will proceed directly to radical nephroureterectomy (RNU) with bladder cuff excision without receiving any prior systemic chemotherapy. The surgery will be scheduled as soon as feasibly possible after enrollment and confirmation of eligibility. Surgical Intervention: The surgical approach (robot-assisted, laparoscopic, or open) will be identical to that offered in Cohort A, chosen at the surgeon's discretion based on standard clinical practice. Objective: This cohort serves as the control group to provide a contemporary benchmark for standard-of-care outcomes. The objective is to establish the baseline rates of pathological stage distribution, survival, and recurrence without the influence of NAC. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pathological Downstaging Rate (≤ ypT1N0)
The proportion of patients achieving pathological downstaging to stage ≤ ypT1N0 in the surgical specimen following neoadjuvant chemotherapy and radical nephroureterectomy (RNU), as assessed by central pathology review. Patients in the NAC cohort who do not undergo surgery will be classified as non-responders.
Measured: Assessed at the time of surgery, approximately 10-14 weeks after initiation of treatment for the NAC+Surgery cohort and approximately 2-4 weeks after enrollment for the Surgery Only cohort.
Secondary outcomes (5)
Pathological Complete Response (pCR) Rate
The proportion of patients achieving a pathological complete response (ypT0N0) in the surgical specimen in the NAC+Surgery cohort.
Measured: Assessed at the time of surgery, approximately 10-14 weeks after initiation of treatment.
Incidence of Treatment-Related Adverse Events
Safety and tolerability of neoadjuvant chemotherapy as measured by the incidence, type, and severity of treatment-related adverse events graded according to CTCAE v5.0.
Measured: From start of NAC through surgery (approximately 10-14 weeks).
Overall Survival (OS)
The time from the date of enrollment to the date of death due to any cause.
Measured: From enrollment until death from any cause, assessed up to 60 months.
Recurrence-Free Survival (RFS)
The time from surgery to the first documented occurrence of local/regional recurrence, distant metastasis, or death from any cause, whichever occurs first.
Measured: From surgery until first documented recurrence or death, assessed up to 60 months.
Cancer-Specific Survival (CSS)
The time from enrollment to death directly attributable to upper tract urothelial carcinoma.
Measured: From enrollment until death due to cancer, assessed up to 60 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Sep 2025
- Study start15 May 2024
- Primary completion15 May 2027 (estimated)
- Study completion15 May 2029 (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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 120 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
- NCT07155044
- Sponsor's protocol ID
- CH-NAC+UTUC
- Status last verified
- May 2025
- First submitted
- 27 Aug 2025
- Last updated
- 4 Sep 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data (IPD) will not be shared to ensure the confidentiality and privacy of our study participants. The informed consent documents approved by our institutional ethics committee do not include provisions for public sharing of individual-level data. Furthermore, the data contains potentially identifiable sensitive health information, and sharing it would be inconsistent with our commitments to participants and the requirements of local data protection regulations.
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating the Application Value of Neoadjuvant Chemotherapy Compared to Upfront Surgery in Patients With Upper Tract Urothelial Carcinoma
- ClinicalTrials.gov ID
- NCT07155044
- Acronym / sponsor ID
- CH-NAC+UTUC
- Conditions
- UTUC, UC (Urothelial Cancer)
- Keywords
- NAC
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Changhai HospitalUniversity, hospital or other
- Study start
- 15 May 2024 (actual)
- Primary completion
- 15 May 2027 (estimated)
- Study completion
- 15 May 2029 (estimated)
- First posted
- 4 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.
This is a single-center, open-label, non-randomized controlled trial comparing the efficacy and safety of neoadjuvant chemotherapy (NAC) followed by radical surgery versus upfront radical surgery alone in patients with high-risk, non-metastatic upper tract urothelial carcinoma (UTUC).
The study aims to answer the following key questions:
Does NAC improve pathologic response rates (defined as downstaging to <ypT2N0) compared to immediate surgery? What is the rate of pathologic complete response (pCR; ypT0N0) in the NAC group? How do the two treatment strategies compare in terms of overall survival, cancer-specific survival, and recurrence-free survival? What is the safety and tolerability profile of NAC using gemcitabine and cisplatin in this patient population? Eligible participants will be assigned to either the NAC-plus-surgery group or the surgery-only group based on clinical evaluation and patient preference. The study will also explore potential biomarkers (e.g., chromosomal instability in liquid biopsies) for predicting treatment response.
Key Eligibility Criteria:
Adults with histologically confirmed high-risk UTUC Clinical stage ≤N1 M0 Adequate renal function (GFR ≥45 mL/min) and ECOG performance status 0-1
Primary Outcome:
Pathologic response rate (proportion of patients with <ypT2N0)
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NAC+RNU | – | Drug: NACIntervention: Participants in this cohort will receive 3-4 cycles of neoadjuvant chemotherapy (NAC) with the gemcitabine and cisplatin (GC) regimen prior to undergoing definitive surgery. Gemcitabine (1000 mg/m²) will be administered on Day 1 and Day 8, and cisplatin (70 mg/m²) will be administered on Day 2 of each 21-day cycle. Surgical Intervention: Following the completion of NAC, patients will undergo a radical nephroureterectomy (RNU) with bladder cuff excision. The procedure will be performed via robot-assisted (Da Vinci), laparoscopic, or open approach, based on surgical discretion. Surgery must be performed within 6 weeks (±2 weeks) after the last dose of chemotherapy. Objective: The primary objective for this cohort is to evaluate the efficacy of NAC in inducing pathological downstaging (defined as achieving pathological stage <ypT2N0 at surgery) and to assess the pathological complete response (pCR) rate (ypT0N0). |
| RNU | – | –Intervention: Participants in this cohort will proceed directly to radical nephroureterectomy (RNU) with bladder cuff excision without receiving any prior systemic chemotherapy. The surgery will be scheduled as soon as feasibly possible after enrollment and confirmation of eligibility. Surgical Intervention: The surgical approach (robot-assisted, laparoscopic, or open) will be identical to that offered in Cohort A, chosen at the surgeon's discretion based on standard clinical practice. Objective: This cohort serves as the control group to provide a contemporary benchmark for standard-of-care outcomes. The objective is to establish the baseline rates of pathological stage distribution, survival, and recurrence without the influence of NAC. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population will consist of adult patients (≥18 years) with histologically confirmed, non-metastatic upper tract urothelial carcinoma (UTUC) who are planned for radical nephroureterectomy (RNU). Participants will be recruited from the urology oncology clinics at a single tertiary care center (e.g., The First Affiliated Hospital of Naval Military Medical University). Eligibility will be determined based on predefined inclusion and exclusion criteria, focusing on patients with adequate renal function (GFR ≥45 mL/min) and performance status (ECOG 0-1) to be potential candidates for cisplatin-based chemotherapy. The population will include both patients who are deemed eligible and choose to receive neoadjuvant chemotherapy prior to surgery and those who proceed directly to surgery.
Key inclusion criteria (9 listed)
- Histologically confirmed upper tract urothelial carcinoma (UTUC). For mixed histology, urothelial carcinoma must be the predominant component (≥50%).
- Clinically non-metastatic disease (cN≤1, M0) as determined by cross-sectional imaging (CT or MRI of chest/abdomen/pelvis).
- Planned treatment with radical nephroureterectomy (RNU).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
- Adequate renal function, defined as glomerular filtration rate (GFR) ≥ 45 mL/min, making the patient a potential candidate for cisplatin-based neoadjuvant chemotherapy.
Key exclusion criteria (7 listed)
- Radiographic evidence of ≥cN2 lymph node disease or distant metastases (M1).
- History of invasive, lymph node-positive, or metastatic urothelial carcinoma within 2 years prior to enrollment, or history of invasive contralateral UTUC.
- Presence of only a solitary kidney or cisplatin ineligibility.
- Concurrent participation in another interventional clinical trial at the time of enrollment.
- History of a non-urothelial malignant tumor unless the patient has been disease-free for at least 1 year (exceptions include adequately treated basal cell or squamous cell skin cancer, or carcinoma in situ of the cervix).
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 |
|---|---|---|
| NACParticipants in NAC+RNU cohort will receive 3-4 cycles of neoadjuvant chemotherapy (NAC) with the gemcitabine and cisplatin (GC) regimen prior to undergoing definitive surgery. Gemcitabine (1000 mg/m²) will be administered on Day 1 and Day 8, and cisplatin (70 mg/m²) will be administered on Day 2 of each 21-day cycle. | Drug | NAC+RNU |
Primary outcome
Pathological Downstaging Rate (≤ ypT1N0)
Time frame: Assessed at the time of surgery, approximately 10-14 weeks after initiation of treatment for the NAC+Surgery cohort and approximately 2-4 weeks after enrollment for the Surgery Only cohort.
The proportion of patients achieving pathological downstaging to stage ≤ ypT1N0 in the surgical specimen following neoadjuvant chemotherapy and radical nephroureterectomy (RNU), as assessed by central pathology review. Patients in the NAC cohort who do not undergo surgery will be classified as non-responders.
5 secondary outcomes
Pathological Complete Response (pCR) Rate
Time frame: Assessed at the time of surgery, approximately 10-14 weeks after initiation of treatment.
Incidence of Treatment-Related Adverse Events
Time frame: From start of NAC through surgery (approximately 10-14 weeks).
Overall Survival (OS)
Time frame: From enrollment until death from any cause, assessed up to 60 months.
Recurrence-Free Survival (RFS)
Time frame: From surgery until first documented recurrence or death, assessed up to 60 months.
Cancer-Specific Survival (CSS)
Time frame: From enrollment until death due to cancer, assessed up to 60 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 4 Sep 2025.
- Individual participant dataSharing planned: No. Individual participant data (IPD) will not be shared to ensure the confidentiality and privacy of our study participants. The informed consent documents approved by our institutional ethics committee do not include provisions for public sharing of individual-level data. Furthermore, the data contains potentially identifiable sensitive health information, and sharing it would be inconsistent with our commitments to participants and the requirements of local data protection regulations.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of UTUC and UC (Urothelial Cancer), 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 7 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: under way, not looking for participants, as of the sponsor's last update on 4 Sep 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.
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?
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?
Pathological Downstaging Rate (≤ ypT1N0) (time frame: Assessed at the time of surgery, approximately 10-14 weeks after initiation of treatment for the NAC+Surgery cohort and approximately 2-4 weeks after enrollment for the Surgery Only cohort.). 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 4 Sep 2025.