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A transitional cell carcinoma that arises from the renal pelvis and ureter.
Biomarker and diagnostic research for renal pelvis/ureter urothelial carcinoma has been reported in the published literature.
No approved treatments are currently available for renal pelvis/ureter urothelial carcinoma. An additional 1 compound holds orphan drug designation.
While no drugs are FDA-approved specifically for renal pelvis/ureter urothelial carcinoma, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for renal pelvis/ureter urothelial carcinoma. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
Estimated prevalence: Unknown (Unknown prevalence).
9 clinical trials registered, 4 recruiting. Interventions under study include drug therapy, other interventions, procedural interventions, and biologic therapy. Pipeline includes 1 PHASE4, 2 PHASE3, 4 PHASE2. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT06774131](https://clinicaltrials.gov/study/NCT06774131) |
Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 8:14 PM UTC
European rare disease database
Designated
Exclusivity End |
|---|
Designation Status |
|---|
gemcitabine | gemcitabine | TARIS Biomedical LLC | 2015 | — | Designated |
9 trials found
A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer |
PHASE3 |
UroGen Pharma Ltd. |
RECRUITING |
[NCT03244384](https://clinicaltrials.gov/study/NCT03244384) | Testing MK-3475 (Pembrolizumab) After Surgery for Localized Muscle-Invasive Bladder Cancer and Locally Advanced Urothelial Cancer | PHASE3 | National Cancer Institute (NCI) | UNKNOWN |
[NCT06124976](https://clinicaltrials.gov/study/NCT06124976) | Multicenter Clinical Trial of ST-02 for Ablation of Upper Tract Urothelial Carcinoma | PHASE2 | Peter Black | UNKNOWN |
[NCT06040762](https://clinicaltrials.gov/study/NCT06040762) | A Home-Based Prehabilitation Exercise Intervention for Improving Physical Function in Patients With Bladder Cancer and Upper Tract Urothelial Cancer, Get Moving Trial | NA | University of Washington | UNKNOWN |
[NCT05775471](https://clinicaltrials.gov/study/NCT05775471) | Pembrolizumab and Enfortumab Vedotin With Pembrolizumab Prior to and After Radical Nephroureterectomy for High-Risk Upper Tract Urothelial Cancer | PHASE2 | Jonsson Comprehensive Cancer Center | UNKNOWN |
52 publications have been identified in PubMed for renal pelvis/ureter urothelial carcinoma. Research spans Epidemiology / Natural History (31%), Case Report / Case Series (29%), and Review / Meta-Analysis (23%).
Research Type | Count | % of Total |
|---|---|---|
Disease patterns and progression | 16 | 31% |
Patient case studies | 15 | 29% |
Research summaries | 12 | 23% |
Testing and diagnosis research | 6 | 12% |
Clinical study results | 2 | 4% |
Laboratory research | 1 | 2% |
Deng Z (2026). [PMID: 42172026](https://pubmed.ncbi.nlm.nih.gov/42172026/). *JAMA Netw Open*. [Review / Meta-Analysis]
Moryousef J (2026). [PMID: 41671923](https://pubmed.ncbi.nlm.nih.gov/41671923/). *Urol Oncol*. [Review / Meta-Analysis]
Koterazawa S (2026). [PMID: 41941023](https://pubmed.ncbi.nlm.nih.gov/41941023/). *Ann Surg Oncol*. [Diagnostic / Biomarker]
Taniguchi K (2026). [PMID: 41726849](https://pubmed.ncbi.nlm.nih.gov/41726849/). *IJU Case Rep*. [Case Report / Case Series]
Wang S (2026). [PMID: 41894298](https://pubmed.ncbi.nlm.nih.gov/41894298/). *Medicine (Baltimore)*. [Review / Meta-Analysis]
Cheng KW (2026). [PMID: 41948153](https://pubmed.ncbi.nlm.nih.gov/41948153/). *Case Rep Urol*. [Case Report / Case Series]
Zhang GN (2026). [PMID: 42061470](https://pubmed.ncbi.nlm.nih.gov/42061470/). *Hum Pathol*. [Case Report / Case Series]
Duh A (2026). [PMID: 41742587](https://pubmed.ncbi.nlm.nih.gov/41742587/). *J Vet Intern Med*. [Case Report / Case Series]
Guan B (2026). [PMID: 41677900](https://pubmed.ncbi.nlm.nih.gov/41677900/). *World J Urol*. [Epidemiology / Natural History]
Eichenauer R (2026). [PMID: 41916385](https://pubmed.ncbi.nlm.nih.gov/41916385/). *Aktuelle Urol*. [Epidemiology / Natural History]
AI-curated news mentioning renal pelvis/ureter urothelial carcinoma
Updated Feb 6, 2026
A recent study investigates the diagnostic features and oncologic outcomes associated with venous invasion in upper tract urothelial carcinoma. The findings may enhance understanding of disease progression and inform treatment strategies.