Urothelial Carcinoma of the Renal Pelvis and UreterPhase 2LUXUS0701NCT07720921
Neoadjuvant Disitamab Vedotin, Toripalimab, and Radiotherapy for High-risk UTUC ( LUXUS07.1 )
A Phase 2 study testing Neoadjuvant radiotherapy and ADC + PD1 monoclonal antibody in people with Urothelial Carcinoma of the Renal Pelvis and Ureter and Neoadjuvant Therapy, run by Peking University First Hospital at 1 site in China.
Trial summary
This is an open-label, single-arm, single-cohort, prospective clinical study evaluating neoadjuvant antibody-drug conjugate therapy combined with immunotherapy and sequential radiotherapy followed by radical surgery in patients with high-risk upper tract urothelial carcinoma (UTUC). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Urothelial Carcinoma of the Renal Pelvis and Ureter, Neoadjuvant Therapy
- What is being tested?
- Neoadjuvant radiotherapy and ADC + PD1 monoclonal antibody
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Nov 2026to 1 Aug 2029
- 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, male or female.
- Imaging evaluation before treatment excludes distant organ metastasis or other concurrent malignancy and suggests resectable high-risk UTUC.
- Completed pathological biopsy with a clear pathological diagnosis : including puncture biopsy, ureteroscopy and urine cytology, at least one of which is clearly diagnosed as uroepithelial carcinoma, which may include no more than 50% or more proportion of squamous, adenoid, sarcomatoid differentiation and other special differentiation types;
- High-risk features, including muscle-invasive disease, high-grade tumor, multifocal disease, tumor diameter ≥2 cm, hydronephrosis, or regional lymph node involvement.
- HER2 expression by immunohistochemistry 1+ to 3+; PD-L1 expression not restricted.
- Have the willingness to undergo radical surgical treatment and perioperative adjuvant treatment, have good compliance, and be able to co-operate with the treatment and follow-up plan specified by the clinician;
- Postoperative life expectancy of at least 6 months; ECOG score ≤ 2.
Who cannot join
- Distant metastases or other malignant neoplastic diseases combined with other malignant neoplasms in the same period had been detected at the time of surgery, or the present was a progression after palliative resection of previous epithelial carcinoma of the urothelium;
- History of pelvic and abdominal radiotherapy; history of inflammatory bowel disease; history of systemic chemotherapy;
- Pregnant women or breastfeeding women; or women of childbearing potential who are not using reliable contraception;
- The presence of active infections in those with pre-existing or coexisting haemorrhagic disorders
- clinically significant cardiac disease (e.g., hypertension controlled with medications, unstable angina, New York Heart Association (NYHA) class ≥II congestive heart failure, unstable symptomatic arrhythmias, or class ≥II peripheral vascular disease);
- Psychological, familial, and social factors leading to lack of informed consent.
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
Departmeng of Urology, Peking University First Hospita
Beijing, Beijing Municipality
Xuesong Li, Dr, 86-15801399116, pineneedle@sina.com
WEI ZUO, vivian19980814@126.com
About this study
Official title: Efficacy and Safety of Neoadjuvant Disitamab Vedotin, Toripalimab, and Radiotherapy for High-risk Upper Tract Urothelial Carcinoma: A Single-arm, Open Label, Prospective Cohort Study
This is an open-label, single-arm, single-cohort, prospective clinical study evaluating neoadjuvant antibody-drug conjugate therapy combined with immunotherapy and sequential radiotherapy followed by radical surgery in patients with high-risk upper tract urothelial carcinoma (UTUC). Eligible patients will receive neoadjuvant disitamab vedotin (RC48) plus toripalimab, followed by response-guided neoadjuvant radiotherapy and radical nephroureterectomy with bladder cuff excision. The study will assess the safety, feasibility, and preliminary antitumor activity of this multimodal neoadjuvant strategy, with pathological complete response rate as the primary endpoint.
Read the detailed description
High-risk UTUC is associated with a substantial risk of recurrence and metastasis after radical surgery. Many patients are unable to tolerate cisplatin-based perioperative chemotherapy because of impaired renal function or treatment-related toxicity. Radiotherapy may improve local control, and low-dose radiation may also modulate the tumor immune microenvironment. Combining an antibody-drug conjugate, immune checkpoint blockade, and sequential radiotherapy before surgery may provide systemic tumor control, enhance local tumor response, and improve pathological downstaging.
This study will enroll patients with resectable, non-metastatic, high-risk UTUC at Peking University First Hospital. Patients will undergo baseline imaging, pathological confirmation, biomarker evaluation, and collection of blood, urine, and tumor specimens. A safety run-in phase of 6 patients will be used to evaluate early safety and dose-limiting toxicities during the first treatment cycle, with special attention to days 7-14. If the safety threshold is met, an additional 14 patients will be enrolled. Interim efficacy assessment will be performed before the penultimate systemic treatment cycle to guide subsequent radiotherapy and surgical planning. Patients will then undergo radical surgery when clinically appropriate and will be followed for recurrence, metastasis, survival, safety, and exploratory molecular endpoints.
- Being tested
- Neoadjuvant radiotherapy, ADC + PD1 monoclonal antibody
- Keywords
- UTUC, neoadjuvant radiotherapy, neoadjuvant immunotherapy, neoadjuvant Antibody-Drug Conjugate, prospective cohort, disitamab vedotin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Neoadjuvant radiotherapyRadiation
Patients with a clear pathological diagnosis of high-risk UTUC were treated with a short course of 5 days of naSBRT: radiotherapy irradiation was directed to the primary lesion on the affected side and to the lymphatic drainage area, with a dose of 25 Gy (5 Gy*5 days).The safety of the neoadjuvant radiotherapy dose and regimen can be evaluated by metrological ramping in the initial 5 patients, with subsequent patients following the optimal dose from ramping.
ADC + PD1 monoclonal antibodyDrug
Participants will receive disitamab vedotin (RC48) 2.0 mg/kg intravenously every 2 weeks, on day 1 of each 14-day cycle, for up to 6 cycles, with a maximum safe dose of 120 mg. Participants will also receive toripalimab 3.0 mg/kg intravenously every 2 weeks, on day 1 of each 14-day cycle, for up to 6 cycles. A safety run-in phase will evaluate dose-limiting toxicities. If ≥2 of the first 6 patients experience DLT, the ADC schedule will be adjusted from every 2 weeks for 6 cycles to every 3 weeks for 4 cycles, with continued safety and efficacy monitoring.
| Study group | Role | What they receive |
|---|---|---|
| Neoadjuvant ADC, immunotherapy and radiotherapy + radical surgery | Experimental | Participants in this single cohort will receive neoadjuvant disitamab vedotin (RC48) plus toripalimab, followed by response-guided targeted-volume radiotherapy and radical nephroureterectomy with bladder cuff excision. Surgery is planned approximately 2-4 weeks after completion of the last neoadjuvant treatment, if the patient's clinical condition permits. Receives: Radiation: Neoadjuvant radiotherapy; Drug: ADC + PD1 monoclonal antibody |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pathological complete response rate
Proportion of participants with no residual viable tumor in the surgical specimen, as assessed by pathological evaluation.
Measured: At radical surgery
Secondary outcomes (6)
Objective response rate
Rate of participants achieving complete response or partial response based on imaging assessment using RECIST or protocol-defined criteria.
Measured: At 2 months after initiation of treatment
Treatment-related adverse events
Incidence and severity of treatment-related adverse events and serious adverse events, graded according to CTCAE.
Measured: through study completion, an average of 1 year"
Disease-free survival
Time from radical surgery to disease recurrence, progression, or death from any cause
Measured: Up to 1 year and 3 years after surgery
Local recurrence-free survival
Time from radical surgery to local recurrence, including recurrence in the operative bed or regional retroperitoneal lymph nodes.
Measured: Up to 3 years after surgery.
Metastasis-free survival
Time from radical surgery to distant metastasis.
Measured: Up to 3 years after surgery.
Overall survival
Description
Measured: Up to 3 years after surgery.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2026
- Study start1 Nov 2026 (estimated)
- Primary completion1 Aug 2027 (estimated)
- Study completion1 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urothelial Carcinoma of the Renal Pelvis and UreterStudies of the same condition, open and under way.
- More trials run by Peking University First 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
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University First Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xuesong Li, Peking University First Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07720921
- Sponsor's protocol ID
- LUXUS 7.1
- Other IDs
- LUXUS
- Acronym
- LUXUS0701
- Status last verified
- Jul 2026
- First submitted
- 17 Jun 2026
- Last updated
- 28 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Neoadjuvant Disitamab Vedotin, Toripalimab, and Radiotherapy for High-risk Upper Tract Urothelial Carcinoma: A Single-arm, Open Label, Prospective Cohort Study
- ClinicalTrials.gov ID
- NCT07720921
- Acronym / sponsor ID
- LUXUS0701 / LUXUS 7.1
- Keywords
- UTUC, neoadjuvant radiotherapy, neoadjuvant immunotherapy, neoadjuvant Antibody-Drug Conjugate, prospective cohort, disitamab vedotin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Peking University First HospitalUniversity, hospital or other
- Study start
- 1 Nov 2026 (estimated)
- Primary completion
- 1 Aug 2027 (estimated)
- Study completion
- 1 Aug 2029 (estimated)
- First posted
- 22 Jul 2026 (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 an open-label, single-arm, single-cohort, prospective clinical study evaluating neoadjuvant antibody-drug conjugate therapy combined with immunotherapy and sequential radiotherapy followed by radical surgery in patients with high-risk upper tract urothelial carcinoma (UTUC). Eligible patients will receive neoadjuvant disitamab vedotin (RC48) plus toripalimab, followed by response-guided neoadjuvant radiotherapy and radical nephroureterectomy with bladder cuff excision. The study will assess the safety, feasibility, and preliminary antitumor activity of this multimodal neoadjuvant strategy, with pathological complete response rate as the primary endpoint.
Detailed description
High-risk UTUC is associated with a substantial risk of recurrence and metastasis after radical surgery. Many patients are unable to tolerate cisplatin-based perioperative chemotherapy because of impaired renal function or treatment-related toxicity. Radiotherapy may improve local control, and low-dose radiation may also modulate the tumor immune microenvironment. Combining an antibody-drug conjugate, immune checkpoint blockade, and sequential radiotherapy before surgery may provide systemic tumor control, enhance local tumor response, and improve pathological downstaging.
This study will enroll patients with resectable, non-metastatic, high-risk UTUC at Peking University First Hospital. Patients will undergo baseline imaging, pathological confirmation, biomarker evaluation, and collection of blood, urine, and tumor specimens. A safety run-in phase of 6 patients will be used to evaluate early safety and dose-limiting toxicities during the first treatment cycle, with special attention to days 7-14. If the safety threshold is met, an additional 14 patients will be enrolled. Interim efficacy assessment will be performed before the penultimate systemic treatment cycle to guide subsequent radiotherapy and surgical planning. Patients will then undergo radical surgery when clinically appropriate and will be followed for recurrence, metastasis, survival, safety, and exploratory molecular endpoints.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Neoadjuvant ADC, immunotherapy and radiotherapy + radical surgery | Experimental | Radiation: Neoadjuvant radiotherapy, Drug: ADC + PD1 monoclonal antibodyParticipants in this single cohort will receive neoadjuvant disitamab vedotin (RC48) plus toripalimab, followed by response-guided targeted-volume radiotherapy and radical nephroureterectomy with bladder cuff excision. Surgery is planned approximately 2-4 weeks after completion of the last neoadjuvant treatment, if the patient's clinical condition permits. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥18 years, male or female.
- Imaging evaluation before treatment excludes distant organ metastasis or other concurrent malignancy and suggests resectable high-risk UTUC.
- Completed pathological biopsy with a clear pathological diagnosis : including puncture biopsy, ureteroscopy and urine cytology, at least one of which is clearly diagnosed as uroepithelial carcinoma, which may include no more than 50% or more proportion of squamous, adenoid, sarcomatoid differentiation and other special differentiation types;
- High-risk features, including muscle-invasive disease, high-grade tumor, multifocal disease, tumor diameter ≥2 cm, hydronephrosis, or regional lymph node involvement.
- HER2 expression by immunohistochemistry 1+ to 3+; PD-L1 expression not restricted.
Key exclusion criteria (6 listed)
- Distant metastases or other malignant neoplastic diseases combined with other malignant neoplasms in the same period had been detected at the time of surgery, or the present was a progression after palliative resection of previous epithelial carcinoma of the urothelium;
- History of pelvic and abdominal radiotherapy; history of inflammatory bowel disease; history of systemic chemotherapy;
- Pregnant women or breastfeeding women; or women of childbearing potential who are not using reliable contraception;
- The presence of active infections in those with pre-existing or coexisting haemorrhagic disorders
- clinically significant cardiac disease (e.g., hypertension controlled with medications, unstable angina, New York Heart Association (NYHA) class ≥II congestive heart failure, unstable symptomatic arrhythmias, or class ≥II peripheral vascular disease);
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 |
|---|---|---|
| Neoadjuvant radiotherapyPatients with a clear pathological diagnosis of high-risk UTUC were treated with a short course of 5 days of naSBRT: radiotherapy irradiation was directed to the primary lesion on the affected side and to the lymphatic drainage area, with a dose of 25 Gy (5 Gy*5 days).The safety of the neoadjuvant radiotherapy dose and regimen can be evaluated by metrological ramping in the initial 5 patients, with subsequent patients following the optimal dose from ramping. | Radiation | Neoadjuvant ADC, immunotherapy and radiotherapy + radical surgery |
| ADC + PD1 monoclonal antibodyParticipants will receive disitamab vedotin (RC48) 2.0 mg/kg intravenously every 2 weeks, on day 1 of each 14-day cycle, for up to 6 cycles, with a maximum safe dose of 120 mg. Participants will also receive toripalimab 3.0 mg/kg intravenously every 2 weeks, on day 1 of each 14-day cycle, for up to 6 cycles. A safety run-in phase will evaluate dose-limiting toxicities. If ≥2 of the first 6 patients experience DLT, the ADC schedule will be adjusted from every 2 weeks for 6 cycles to every 3 weeks for 4 cycles, with continued safety and efficacy monitoring. | Drug | Neoadjuvant ADC, immunotherapy and radiotherapy + radical surgery |
Primary outcome
Pathological complete response rate
Time frame: At radical surgery
Proportion of participants with no residual viable tumor in the surgical specimen, as assessed by pathological evaluation.
6 secondary outcomes
Objective response rate
Time frame: At 2 months after initiation of treatment
Treatment-related adverse events
Time frame: through study completion, an average of 1 year"
Disease-free survival
Time frame: Up to 1 year and 3 years after surgery
Local recurrence-free survival
Time frame: Up to 3 years after surgery.
Metastasis-free survival
Time frame: Up to 3 years after surgery.
Overall survival
Time frame: Up to 3 years after surgery.
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 28 Oct 2026.
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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 Neoadjuvant radiotherapy and ADC + PD1 monoclonal antibody in people with Urothelial Carcinoma of the Renal Pelvis and Ureter and Neoadjuvant Therapy, run by Peking University First 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 6 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 28 Oct 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 ZUO WEI, Dr (+8618811650832, vivian19980814@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pathological complete response rate (time frame: At radical 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 28 Oct 2026.