Urothelial CarcinomaPhase 2NCT01261728
Gemcitabine and Cisplatin as Neoadjuvant Chemotherapy in Patients With High-Grade Upper Tract Urothelial Carcinoma
A Phase 2 study testing Gemcitabine and Cisplatin in people with Urothelial Carcinoma, run by Memorial Sloan Kettering Cancer Center at 9 sites in United States.
Trial summary
The purpose of this study is to see if getting chemotherapy with Gemcitabine and Cisplatin for four 21 day cycles for a total of 12 weeks can help shrink the tumor before undergoing surgery for kidney cancer. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Urothelial Carcinoma
- What is being tested?
- Gemcitabine and Cisplatin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 57 participants
- Where
- 9 sitesUnited States
- Study dates
- 14 Dec 2010to 6 Aug 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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-grade upper tract transitional cell carcinoma at MSKCC or a participating site and/or radiographically visible tumor stage T2-T4a N0/X M0 disease with positive selective urinary cytology or high-grade concomitant bladder tumor. Hydronephrosis associated with tumor on biopsy will be considered invasive by definition.
- Medically appropriate candidate for radical nephroureterectomy or ureterectomy as per MSKCC or a participating site attending urologic oncologist
- Karnofsky Performance Status ≥ 70%
- Age ≥ 18 years of age
- Required Initial Laboratory Values:
- Absolute neutrophil count ≥ 1500 cells/mm3
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- Platelets ≥ 100,000 cells/mm3
- Hemoglobin ≥ 9.0g/dL
- Bilirubin ≤ 1.2
- Aspartate transaminase (AST) and alanine transaminase (ALT) ≤ 2.5 x ULN for the institution
- Alkaline phosphatase ≤ 2.5 x ULN for the institution
- Serum creatinine ≤ 1.3 mg/dL if male or ≤ 1.1 mg/dL if female OR calculated creatinine clearance ≥ 55 ml/min/1.73m^2 If female of childbearing potential, serum pregnancy test is negative.
- Patients with reproductive potential must use an effective method to avoid pregnancy for the duration of the trial.
- ml/min/1.73m2 using the formula: CKD epi : GFR = 141 X min(Scr/κ,1)α X max(Scr/κ,1)-1.209 X 0.993Age X 1.018 [if female] X 1.159 [if black]
- Scr is serum creatinine, k is 0.7 for females and 0.9 for males, a is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/k or 1, and max indicates the maximum of Scr/k or 1.
- If female of childbearing potential, serum pregnancy test is negative.
- Patients with reproductive potential must use an effective method to avoid pregnancy for the duration of the trial
Who cannot join
- Concomitant bladder urothelial carcinoma is acceptable if it is organ confined and surgically resectable.
- Presence of carcinoma in situ (CIS)
- Prior systemic chemotherapy (prior intravesical therapy is allowed)
- Prior radiation therapy to the bladder
- Evidence of NYHA functional class III or IV heart disease.
- Serious intercurrent medical or psychiatric illness, including serious active infection.
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- Preexisting sensory grade 3 neuropathy
- Major surgery or radiation therapy < 4 weeks of starting study treatment.
- Concomitant use of any other investigational drugs
- Any of the following within the 6 months prior to study drug administration:
- myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident or transient ischemic attack, or pulmonary embolism.
- Ongoing cardiac dysrhythmias of NCI CTCAE Version 4.0 grade ≥ 2.
- Uncontrolled hypertension (>150/100 mmHg despite optimal medical therapy).
- Known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)-related illness or other active infection. Patients with HIV but no evidence of AIDS will be considered candidates.
- Concurrent treatment on another clinical trial involving an intervention which may affect the primary endpoint. Supportive care trials or non-treatment trials, e.g. QOL, are allowed.
- Ongoing treatment with therapeutic doses of warfarin or low molecular weight heparin (low dose warfarin up to 2 mg po daily or use of subcutaneous low molecular weight heparin for thromboembolic prophylaxis is allowed).
- Pregnancy or breast-feeding. Patients must be surgically sterile or be postmenopausal, or must agree to use effective contraception during the period of therapy. The definition of effective contraception will be based on the judgment of the MSKCC and participating site PI. Male patients must be surgically sterile or agree to use effective contraception
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Lehigh Valley Health Network (Data Collection Only)
Allentown, Pennsylvania, 18103
Memorial Sloan Kettering at Basking Ridge
Basking Ridge, New Jersey, 07920
Memorial Sloan Kettering Cancer Center @ Suffolk
Commack, New York, 11725
Memorial Sloan Kettering Westchester
Harrison, New York, 10604
Hartford Hospital (Data Collection Only)
Hartford, Connecticut
Memorial Sloan Kettering Bergen
Montvale, New Jersey, 07645
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Mayo Clinic - Arizona
Scottsdale, Arizona, 85259
Memorial Sloan Kettering Nassau (Consent and Follow-Up only)
Uniondale, New York, 11553
About this study
Official title: Phase II Study of Gemcitabine and Cisplatin as Neoadjuvant Chemotherapy in Patients With High-Grade Upper Tract Urothelial Carcinoma
The purpose of this study is to see if getting chemotherapy with Gemcitabine and Cisplatin for four 21 day cycles for a total of 12 weeks can help shrink the tumor before undergoing surgery for kidney cancer.
- Conditions
- Urothelial Carcinoma
- Being tested
- Gemcitabine and Cisplatin
- Keywords
- Gemcitabine, Cisplatin, Bladder, radical nephroureterectomy, ureterectomy, 10-208
- Medical subject headings
- Carcinoma, Transitional Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gemcitabine and CisplatinDrug
Patients will receive four cycles of GC administered every 21 days. Gemcitabine 1,000 mg/m2 and Cisplatin 35 mg/m2 will be administered intravenously on days 1 and 8. A total of four cycles of therapy will be administered at 21 day intervals followed by radical nephroureterectomy or distal ureterectomy.
| Study group | Role | What they receive |
|---|---|---|
| Gemcitabine and Cisplatin | Experimental | This is a Phase II Study of Gemcitabine and Cisplatin (GC) as neoadjuvant chemotherapy in patients with upper tract high-grade urothelial carcinoma who are candidates for radical nephroureterectomy or distal ureterectomy. Receives: Drug: Gemcitabine and Cisplatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pathologic Response Rate (<pT2)
of neoadjuvant Gemcitabine and Cisplatin/GC regimen in patients with upper tract high-grade urothelial carcinoma. Pathologic response to neoadjuvant GC is defined as the absence of high-grade carcinoma (<pT2 disease) and the absence of microscopic lymph node metastases (N0) on the final nephroureterectomy specimen.
Measured: up to 2 years
Secondary outcomes (3)
Percentage of Participants With Progression Free Survival Rate
will be estimated using the methods of Kaplan and Meier in patients with upper tract high-grade urothelial carcinoma with neoadjuvant GC followed by radical nephroureterectomy.
Measured: 2 years
Overall Survival
will be estimated using the methods of Kaplan and Meier in patients with upper tract high-grade urothelial carcinoma treated with neoadjuvant GC followed by radical nephroureterectomy.
Measured: 2 years
Number of Participants Evaluated for Toxicity
of neoadjuvant Gemcitabine and Cisplatin in this setting. Toxicity will be graded according to the National Cancer Institute Common Toxicity Criteria version 4.0.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2010 (estimated)
- Study start14 Dec 2010
- Primary completion6 Aug 2025
- Study completion6 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)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 Memorial Sloan Kettering Cancer CenterThe 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
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Mayo Clinic, Hartford Hospital
- Responsible party
- Sponsor
- Investigators
- Jonathan Coleman, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01261728
- Sponsor's protocol ID
- 10-208
- Status last verified
- Aug 2025
- First submitted
- 15 Dec 2010
- Last updated
- 2 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Coleman JA, Yip W, Wong NC, Sjoberg DD, Bochner BH, Dalbagni G, Donat SM, Herr HW, Cha EK, Donahue TF, Pietzak EJ, Hakimi AA, Kim K, Al-Ahmadie HA, Vargas HA, Alvim RG, Ghafoor S, Benfante NE, Meraney AM, Shichman SJ, Kamradt JM, Nair SG, Baccala AA Jr, Palyca P, Lash BW, Rizvi MA, Swanson SK, Muina AF, Apolo AB, Iyer G, Rosenberg JE, Teo MY, Bajorin DF. Multicenter Phase II Clinical Trial of Gemcitabine and Cisplatin as Neoadjuvant Chemotherapy for Patients With High-Grade Upper Tract Urothelial Carcinoma. J Clin Oncol. 2023 Mar 10;41(8):1618-1625. doi: 10.1200/JCO.22.00763. Epub 2023 Jan 5.
PubMed 36603175Hird AE, Magee DE, Cheung DC, Sander B, Sridhar S, Nam RK, Kulkarni GS. Neoadjuvant Versus Adjuvant Chemotherapy for Upper Tract Urothelial Carcinoma: A Microsimulation Model. Clin Genitourin Cancer. 2021 Apr;19(2):e135-e147. doi: 10.1016/j.clgc.2020.10.001. Epub 2020 Oct 13.
PubMed 33168398
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Gemcitabine and Cisplatin as Neoadjuvant Chemotherapy in Patients With High-Grade Upper Tract Urothelial Carcinoma
- ClinicalTrials.gov ID
- NCT01261728
- Acronym / sponsor ID
- 10-208
- Conditions
- Urothelial Carcinoma
- Keywords
- Gemcitabine, Cisplatin, Bladder, radical nephroureterectomy, ureterectomy, 10-208
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Mayo Clinic, Hartford Hospital
- Study start
- 14 Dec 2010 (actual)
- Primary completion
- 6 Aug 2025 (actual)
- Study completion
- 6 Aug 2025 (actual)
- First posted
- 16 Dec 2010 (estimated)
- Results
- Posted 2 Sep 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to see if getting chemotherapy with Gemcitabine and Cisplatin for four 21 day cycles for a total of 12 weeks can help shrink the tumor before undergoing surgery for kidney cancer.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Gemcitabine and Cisplatin | Experimental | Drug: Gemcitabine and CisplatinThis is a Phase II Study of Gemcitabine and Cisplatin (GC) as neoadjuvant chemotherapy in patients with upper tract high-grade urothelial carcinoma who are candidates for radical nephroureterectomy or distal ureterectomy. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Histologically confirmed high-grade upper tract transitional cell carcinoma at MSKCC or a participating site and/or radiographically visible tumor stage T2-T4a N0/X M0 disease with positive selective urinary cytology or high-grade concomitant bladder tumor. Hydronephrosis associated with tumor on biopsy will be considered invasive by definition.
- Medically appropriate candidate for radical nephroureterectomy or ureterectomy as per MSKCC or a participating site attending urologic oncologist
- Karnofsky Performance Status ≥ 70%
- Age ≥ 18 years of age
- Required Initial Laboratory Values:
Key exclusion criteria (17 listed)
- Concomitant bladder urothelial carcinoma is acceptable if it is organ confined and surgically resectable.
- Presence of carcinoma in situ (CIS)
- Prior systemic chemotherapy (prior intravesical therapy is allowed)
- Prior radiation therapy to the bladder
- Evidence of NYHA functional class III or IV heart 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 |
|---|---|---|
| Gemcitabine and CisplatinPatients will receive four cycles of GC administered every 21 days. Gemcitabine 1,000 mg/m2 and Cisplatin 35 mg/m2 will be administered intravenously on days 1 and 8. A total of four cycles of therapy will be administered at 21 day intervals followed by radical nephroureterectomy or distal ureterectomy. | Drug | Gemcitabine and Cisplatin |
Primary outcome
Pathologic Response Rate (<pT2)
Time frame: up to 2 years
of neoadjuvant Gemcitabine and Cisplatin/GC regimen in patients with upper tract high-grade urothelial carcinoma. Pathologic response to neoadjuvant GC is defined as the absence of high-grade carcinoma (<pT2 disease) and the absence of microscopic lymph node metastases (N0) on the final nephroureterectomy specimen.
3 secondary outcomes
Percentage of Participants With Progression Free Survival Rate
Time frame: 2 years
Overall Survival
Time frame: 2 years
Number of Participants Evaluated for Toxicity
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36603175Coleman JA, Yip W, Wong NC, Sjoberg DD, Bochner BH, Dalbagni G, Donat SM, Herr HW, Cha EK, Donahue TF, Pietzak EJ, Hakimi AA, Kim K, Al-Ahmadie HA, Vargas HA, Alvim RG, Ghafoor S, Benfante NE, Meraney AM, Shichman SJ, Kamradt JM, Nair SG, Baccala AA Jr, Palyca P, Lash BW, Rizvi MA, Swanson SK, Muina AF, Apolo AB, Iyer G, Rosenberg JE, Teo MY, Bajorin DF. Multicenter Phase II Clinical Trial of Gemcitabine and Cisplatin as Neoadjuvant Chemotherapy for Patients With High-Grade Upper Tract Urothelial Carcinoma. J Clin Oncol. 2023 Mar 10;41(8):1618-1625. doi: 10.1200/JCO.22.00763. Epub 2023 Jan 5.
- Derived from this study: PubMed 33168398Hird AE, Magee DE, Cheung DC, Sander B, Sridhar S, Nam RK, Kulkarni GS. Neoadjuvant Versus Adjuvant Chemotherapy for Upper Tract Urothelial Carcinoma: A Microsimulation Model. Clin Genitourin Cancer. 2021 Apr;19(2):e135-e147. doi: 10.1016/j.clgc.2020.10.001. Epub 2020 Oct 13.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
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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.
Jonathan Coleman, MD principal investigator
Memorial Sloan Kettering Cancer Center
9 sites 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 and Cisplatin in people with Urothelial Carcinoma, run by Memorial Sloan Kettering Cancer Center at 9 sites in United States.
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 17 inclusion and 17 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 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 9 sites in United States. 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?
Yes. Results are posted in the registry (first posted 2 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Pathologic Response Rate (<pT2) (time frame: up to 2 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 NCT01261728, which also lists 2 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 Sep 2026.