ORPHA:598216
Upper tract urothelial carcinoma
Also known as: Transitional cell carcinoma of the pelvis and ureter · Transitional cell carcinoma of the upper urinary tract · UTUC
Publications
4,295
Trials
39
Interventional, condition-specific
Researchers
1,359
Distinct authors in sample
Gene link
—
Readiness
2/6
Stages with a signal
Clinical definition (Orphanet)
A rare urinary tract tumor characterized by a malignant neoplasm arising from urothelial cells of the pyelocaliceal cavities and ureter. The tumor may be multifocal and bilateral in some cases and is more common in males than in females. Cigarette smoking and occupational exposure are major risk factors. Patients most commonly present with visible or non-visible hematuria and/or flank pain due to obstruction of the flow of urine by tumor tissue, or (less often) to local tumor growth. Systemic symptoms such as anorexia, weight loss, malaise, fatigue, fever, night sweats, or cough can be associated with advanced stages.
How rare: How common this is has not been clearly measured.
Cross-references
Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.
Research stages
Trial readiness signals
Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”
2/6 stages with a signal
An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
4,295 matched papers (3,366 in last 10 years) Source
- Phenotype characterisedNot found
No HPO disease–phenotype associations via Monarch for these Mondo IDs
- Animal modelNot found
No Alliance genotype “model of” associations via Monarch for these Mondo IDs
- Orphan designationNot found
No FDA or EMA orphan-drug designation matched this disease via UMLS or preferred name Source
- Interventional trialPresent
39 matched on ClinicalTrials.gov (21 recruiting in sample)
Biology
Genes and phenotypes
Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.
Do we know what causes it?
Not yet — the cause hasn't been pinned down in GenCC.
No strong gene–disease assertion joined for this Orphanet entity.
Phenotypes (Monarch / HPO)
None returned for this Mondo ID. That often means “not linked under this ID,” not “no clinical features.”
Animal models (Monarch / Alliance)
None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.
Monarch fetch 2026-07-27
Therapies
Designations, candidates, and chemicals
FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.
Orphan designation (FDA · EMA)
No designation matched this disease via UMLS or preferred name on the FDA OOPD mirror or EMA orphan register. Absence here is not proof that none exists under another wording.
Open Targets candidates
No drugs or clinical candidates returned for this Mondo ID on Open Targets.
CTD chemicals (MyDisease.info)
No CTD chemical associations returned for this Mondo ID.
Literature
Is anyone studying this?
4,295
4,295 papers have been published on this condition. That is a real research literature — still far smaller than common diseases (breast cancer: over 700,000 papers). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.
4,295 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).
3,366 in the last 10 years · low confidence
Phrase hits: 4,295 · MeSH hits: 0
Who's working on it?
1,359
Distinct author names in 200 sampled papers — named people below.
Who's working on it?
People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.
- 01Singla N10 papers · 2026
Brady Urological Institute, Johns Hopkins University School of Medicine, Baltimore, USA.
Papers in Europe PMC - 02Zhang S10 papers · 2026
Department of Urology, Peking University Third Hospital, Peking University Health Science Center, Beijing, China. Electronic address: zhangshudong@bjmu.edu.cn.
Papers in Europe PMC - 03Li X9 papers · 2026
Department of Urology and Institute of Urology, West China Hospital, Sichuan University, Chengdu, China.
Papers in Europe PMC - 04Zhang Y9 papers · 2026
State Key Laboratory of Oncology in South China, Guangdong Provincial Clinical Research Center for Cancer, Sun Yat-sen University Cancer Center, Guangzhou 510060, P. R. China.
Papers in Europe PMC - 05Zhang H8 papers · 2026
Department of Urology, Peking University Third Hospital, Peking University Health Science Center, Beijing, China.
Papers in Europe PMC - 06Margulis V7 papers · 2026
Department of Urology, UT Southwestern School of Medicine, Dallas, TX, USA.
Papers in Europe PMC - 07Raman JD7 papers · 2026
Department of Urology, Penn State Health Milton S Hershey Medical Center, Hershey, PA. Electronic address: jraman@pennstatehealth.psu.edu.
Papers in Europe PMC - 08Liedberg F6 papers · 2026
Department of Urology Skåne University Hospital Malmö Sweden.
Papers in Europe PMC - 09Matin SF6 papers · 2026
Department of Urology, University of Texas MD Anderson Cancer Center, Houston, TX, USA. Electronic address: surmatin@mdanderson.org.
Papers in Europe PMC - 10Antonelli A5 papers · 2026
Department of Urology, University of Verona, Azienda Ospedaliera Universitaria Integrata, Verona, Italy.
Papers in Europe PMC
Clinical research
Is a treatment being tested?
39
interventional trials for this specific condition
39 interventional trials matched this specific condition name; 21 currently recruiting in our sample.
Data as of 9 September 2026
39 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 96.7th percentile).
low confidence · 96.7th percentile (trials denominator)
Recruiting interventional trials
From the matched ClinicalTrials.gov set
39 interventional trials matched after quoted-phrase search and title/condition post-filter.
- NCT07321210·RECRUITING·Radical Nephroureterectomy With vs Without Template Lymph Node Dissection in High-Risk Upper Tract Urothelial Carcinoma (T-LND UTUC)
Uncertain·Conditions: Upper Tract Urothelial Carcinoma · Lymph Node Dissection·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07265947·RECRUITING·Phase 2A/B Efficacy and Safety of Dabogratinib in Participants With Low Grade Upper Tract Urothelial Carcinoma
Uncertain·Conditions: Low Grade Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT06040762·RECRUITING·A Home-Based Prehabilitation Exercise Intervention for Improving Physical Function in Patients With Bladder Cancer and Upper Tract Urothelial Cancer, Get Moving Trial
Uncertain·Conditions: Bladder Cancer · Urothelial Carcinoma · Upper Tract Urothelial Carcinoma · Renal Pelvis and Ureter Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07225374·RECRUITING·Cisplatin-Based Chemotherapy Followed by Maintenance Avelumab in UTUC
Uncertain·Conditions: Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT04865939·RECRUITING·Gemcitabine Versus Water Irrigation in Upper Tract Urothelial Carcinoma
Uncertain·Conditions: Urothelial Cancer of Renal Pelvis · Urothelial Carcinoma Ureter·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT05837806·RECRUITING·Efficacy and Safety of Tislelizumab in Combination With Disitamab-vedotin as Neoadjuvant Therapy for HER2-positive High-risk Upper Tract Urothelial Carcinoma (UTUC)
Uncertain·Conditions: Neoadjuvant Immunotherapy·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07126119·RECRUITING·Neoadjuvant Tislelizumab in Combination With Nab-Paclitaxel for UTUC
Uncertain·Conditions: Upper Tract Urothelial Carcinoma · Neoadjuvant Therapy · PD-1 Inhibitor · Nab-paclitaxel·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07492628·RECRUITING·Dual-Target Nectin-4/HER2 CAR-NK Cells in Advanced Urothelial Carcinoma
Uncertain·Conditions: Bladder Cancer · Urothelial Carcinoma · Metastatic Urothelial Carcinoma · Locally Advanced Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT05979909·NOT YET RECRUITING·Intravesical Mitomycin C After Diagnostic Ureteroscopy for Upper Tract Urothelial Carcinoma
Uncertain·Conditions: Urothelial Carcinoma Ureter · Urothelial Cancer of Renal Pelvis·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT06848868·RECRUITING·Ambulatory Flexible URS for UTUC Surveillance
Uncertain·Conditions: Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT06927128·NOT YET RECRUITING·Neoadjuvant Chemotherapy in High - Risk Upper Tract Urothelial Carcinoma
Uncertain·Conditions: UTUC · NAC·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07498907·NOT YET RECRUITING·Disitamab Vedotin Plus Radiotherapy for Adjuvant Treatment of HER2-Expressing Cisplatin-Ineligible Upper Tract Urothelial Carcinoma
Uncertain·Conditions: Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT05917158·RECRUITING·A Study of RC48-ADC Combined with JS001 for Postoperative Adjuvant Treatment of Upper Tract Urothelial Carcinoma
Uncertain·Conditions: Carcinoma · Upper Tract Urothelial Carcinoma · UTUC·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT06948552·RECRUITING·Photodynamic Diagnosis of Upper Tract Urothelial Carcinoma Using Fluorescence Endoscopy and Oral 5-ALA
Uncertain·Conditions: Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07557563·NOT YET RECRUITING·Flexible Ureterorenoscopy for Surveillance of Upper Tract Urothelial Carcinoma
Uncertain·Conditions: Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
Observational and natural-history studies
15 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.
Recruiting or not-yet-recruiting
- NCT06798246·NOT YET RECRUITING·HEFTME System for Adjuvant Therapeutic Selection of Locally Advanced UTUC Postoperatively：A Perspective Cohort Study
Uncertain·Conditions: Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT06204406·RECRUITING·Prognosis of Patients With Upper Tract Urothelial Carcinoma Receiving Kidney-sparing Therapy
Uncertain·Conditions: Upper Tract Urothelial Carcinoma Receiving Kidney-sparing Therapy·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07452432·RECRUITING·Korean Prospective Upper Tract Urothelial Carcinoma Cohort
Uncertain·Conditions: Urothelial Carcinoma (UC)·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07633353·ENROLLING BY INVITATION·Real-World Outcomes of First-Line Treatment of Metastatic or Locally Advanced Upper Tract Urothelial Carcinoma With Enfortumab Vedotin Plus Pembrolizumab : A French Multicenter Retrospective Observational Study
Uncertain·Conditions: Advanced Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07504939·NOT YET RECRUITING·Multimodal Kidney-Sparing Strategy for High-Risk Upper Tract Urothelial Carcinoma
Uncertain·Conditions: Upper Tract Urothelial Carcinoma Receiving Kidney-sparing Therapy · Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT07614594·RECRUITING·Non-invasive Urinary Genomics-Based Risk Assessment for Early Urothelial Carcinoma Diagnosis
Uncertain·Conditions: Urothelial Carcinoma (UC) · Upper Tract Urothelial Carcinoma · UTUC · Bladder Cancer (BC)·Matched via name phraseAt least one provider returned uncertain or parent-category.
- NCT05595408·RECRUITING·Circulating and Urine Tumor DNA Dynamics Predict Minimal Residual Disease and Recurrence Risk in Locally Advanced Upper Tract Urothelial Carcinoma
Uncertain·Conditions: Muscle Invasive Upper Tract Urothelial Carcinoma·Matched via name phraseAt least one provider returned uncertain or parent-category.
Other registries (secondary)
Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.
raw 27 · after dedupe 27 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 27 · dropped 0 · fetched 2026-07-27
Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri
No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).
Uncertain / not reviewed (27)
- ctis·2025-523539-20-00·Authorised, recruiting·A Phase 2A/B, Multi-center, Open-Label Study Evaluating the Efficacy and Safety of Dabogratinib (TYRA-300) in Participants with Low Grade Upper Tract Urothelial Carcinoma (SURF303)
Uncertain — At least one provider returned uncertain or parent-category.
- ctis·2024-514360-70-00·Authorised·A Phase 1/2, Single-arm, Open-Label Trial to Evaluate the Safety and Efficacy of Nadofaragene Firadenovec Instilled to the Renal Pelvis in Adult Subjects with Low-Grade Upper Tract Urothelial Carcinoma (LG-UTUC)
Uncertain — At least one provider returned uncertain or parent-category.
- ctis·2024-514991-41-01·Under evaluation·Neo-adjuvant versus Adjuvant chemotherapy in Upper Tract Urothelial Carcinoma: A feasibility phase II randomized clinical trial (“URANUS”)
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN87678318·Recruiting·Preoperative assessment of tumours of the upper urinary tract by photon-counting computed tomography and magnetic resonance tomography
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN16925539·Recruiting·A phase 3, randomized, open-label, multicenter study evaluating the efficacy and safety of TAR-210 erdafitinib intravesical delivery system versus investigator’s choice of intravesical chemotherapy in participants with high-risk non-muscle-invasive bladder cancer with susceptible FGFR alterations who had received intravesical Bacillus Calmette-Guérin (BCG)
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN49174478·Recruiting·HIVEC® HEAT - the drug mitomycin medac will be warmed to 43°C and administered through a catheter into the bladder via HIVEC® device for patients with nonmuscle-invasive bladder cancer that have previously had no response to BCG treatment
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN15306308·Recruiting·Bladder EpiCheck - testing a urine test to help detect bladder cancer in people with blood in their urine
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN72352492·Recruiting·A research study of an investigational drug delivery system for adults with high-risk non-muscle invasive bladder cancer
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN15842444·Recruiting·Assessing the safety of atezolizumab being directly administered to the bladder in bladder cancer patients undergoing radical cystectomy
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN22771691·No longer recruiting·A study to investigate if atezolizumab can reduce the size of urothelial cancer before surgery and to determine how the drug works
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN18038733·Recruiting·SAGA-trial - investigating if intraoperative sterile water irrigation during an operation to remove the kidney due to cancer (nephroureterectomy for upper tract urothelial carcinoma) decreases the risk of tumor recurrence.
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN17038127·No longer recruiting·A study investigating if a new medication, bintrafusp alfa, can reduce the size of urothelial carcinoma, a type of bladder cancer, before surgery to remove the bladder
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN17940603·No longer recruiting·Non-invasive diagnosis of urinary bladder cancer
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN43698103·No longer recruiting·A comparison of standard chemoradiotherapy treatment to standard chemoradiotherapy treatment given in combination with durvalumab to see if the addition of durvalumab leads to improved survival
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN83155790·Recruiting·Evaluation of lymph node surgery in patients with upper urinary tract carcinoma
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN65084068·No longer recruiting·A study of intravesical TMX-101 in subjects with Non-Muscle-Invasive Bladder Cancer
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN73030316·No longer recruiting·Pazopanib versus pacLitaxel in relapsed Urothelial TumOurs
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN98387754·No longer recruiting·A Phase III randomised trial of perioperative chemotherapy versus surveillance in upper tract urothelial cancer
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN44149443·No longer recruiting·Fibroblast growth factor receptors (FGFR) Inhibition for Epithelial Solid Tumours
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN68146831·No longer recruiting·Vandetanib in non-cisplatin fit patients with urothelial cancers
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN85785327·Stopped·HYMN: a trial comparing hyperthermia and mitomycin chemotherapy with a second BCG treatment, or other standard treatment, for bladder cancer that has come back
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN38528926·No longer recruiting·Bladder cancer: Open versus Laparoscopic or RObotic cystectomy
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN54607216·No longer recruiting·Sunitinib in advanced urothelial cancer in combination with standard cisplatin/gemcitabine chemotherapy treatment
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN31546330·No longer recruiting·Trial of temsirolimus for advanced urothelial cancer
Uncertain — At least one provider returned uncertain or parent-category.
- isrctn·ISRCTN11908197·Recruiting·Disitamab vedotin with pembrolizumab vs chemotherapy in previously untreated urothelial cancer expressing HER2
Uncertain — At least one provider returned uncertain or parent-category.
Where to find support
Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.
Orphanet entry for Upper tract urothelial carcinoma — check Associations / patient organisations on that page.
India — NPRD
Last verified 2026-07-26This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.
Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.
How we counted this
Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.
"Upper tract urothelial carcinoma" OR "Transitional cell carcinoma of the pelvis and ureter" OR "Transitional cell carcinoma of the upper urinary tract"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Upper tract urothelial carcinoma" OR "Transitional cell carcinoma of the pelvis and ureter" OR "Transitional cell carcinoma of pelvis and ureter" OR "Transitional cell carcinoma of the upper urinary tract" OR "Transitional cell carcinoma of upper urinary tract"
Interventional trials matched via: phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).
Study-type breakdown: 39 interventional · 15 observational · 1 expanded access. Only interventional studies enter the trial headline.
Query health: ok — strategies attempted: phrase; with hits: phrase
Run this search on ClinicalTrials.gov
Synonyms dropped by stoplist: UTUC
Confidence reasoning
- Preferred label is multi-word and distinctive
- 1 synonym(s) dropped by stoplist (may under-count)
- No label/synonym collisions with other diseases in this corpus
- Publication count (4295) is extremely high with unknown/missing prevalence — treat as possible over-matching, not proven research intensity
Ingested 2026-07-26T02:11:51.820Z