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A Multi-Center Natural History of Urothelial Cancer and Rare Genitourinary Tract Malignancies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04923178
Recruitment Status : Recruiting
First Posted : June 11, 2021
Last Update Posted : May 23, 2023
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

Tracking Information
First Submitted Date June 10, 2021
First Posted Date June 11, 2021
Last Update Posted Date May 23, 2023
Actual Study Start Date October 24, 2022
Estimated Primary Completion Date December 1, 2041   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: June 10, 2021)
natural history of urothelial and rare genitourinary cancer [ Time Frame: ongoing ]
clinical presentation, patterns of disease progression, therapeutic response, disease recurrence and participant overall survival
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title A Multi-Center Natural History of Urothelial Cancer and Rare Genitourinary Tract Malignancies
Official Title A Multi-Center Natural History of Urothelial Cancer and Rare Genitourinary Tract Malignancies
Brief Summary


Tumors in the genitourinary tracts can occur in the kidney, bladder, prostate, and testicles and can have common and rare histologies. Some cancers that occur along the genitourinary (GU) tract are rare. Some GU tumors are so rare that they are not included in treatment studies or tissue banks. This makes it hard for researchers to determine standards of care. Researchers want to learn more about common and rare GU tumors.


To learn more about urinary tract cancers.


People ages 18 and older with urinary tract or GU cancer such as bladder, kidney, testicular, prostate, penis, or neuroendocrine cancer.


Participants will be screened with questions about their medical history. Their medical records will be reviewed.

Participants will have a physical exam. They will give blood and urine samples. They will complete a survey about their family cancer history. Clinical photographs will be taken to document skin lesions.

Participants may have imaging scans of their chest, abdomen, and pelvis. They may have a contrast agent injected into their arm.

Participants will get recommendations about how to best manage and treat their cancer. They can ask as many questions as they would like.

Participants will provide existing tumor samples if available. They may have optional tumor biopsies up to twice a year. For needle biopsies, the biopsy area will be numbed and they will get a sedative. A needle will be inserted through their skin to collect a tumor sample. For skin biopsies, their skin will be numbed. A small circle of skin will be removed.

Some blood and tumor samples may be used for genetic tests.

Participants will have frequent follow-up visits. If they cannot visit NIH, their home doctor will be contacted. They will be followed on this study for life....

Detailed Description


Rare histological variants of the GU tract include bladder/urachal adenocarcinoma, squamous cell carcinoma, and small cell carcinoma; variants of urothelial carcinoma including plasmacytoid, sarcomatoid; renal tumors including sarcomatoid renal cell carcinoma and renal medullary carcinoma; penile cancers; micropapillary, giant cell, lipid rich, clear cell and nested variants, large cell neuroendocrine carcinoma, lymphoepithelioma-like carcinoma and mixed patterns; small cell neuroendocrine carcinoma of the prostate, testicular Sertoli or Leydig cell tumors, and papillary and chromophobe RCC.

Some GU tumors occur so infrequently that they are not systematically captured by currently available registries, treatment protocols or tissue banks. The rarity of these tumors limits the sufficient numbers of patients needed in larger randomized clinical studies to characterize standard treatments or disease course.

Systematic and longitudinal collection and annotation of clinical history, tissue samples, imaging studies, participant reported outcomes, and other pertinent information in participants with these rare tumors will yield future knowledge and help with the development of subsequent prospective studies to optimize diagnosis and treatment paradigms for less common GU tumors.


Characterization of the natural history of urothelial and rare GU tract malignancies


Participants >= 18 years of age diagnosed with urothelial and rare GU tract malignancies.


This will be a multi-center long-term study to comprehensively study participants with rare GU tumors

Medical history will be collected and participants followed throughout the course of their illnesses, with particular attention to patterns of disease presentation, recurrence and progression, response to therapies, duration of responses and participant reported outcomes.

Tissue samples and blood will be obtained from participants during this study.

Broad spectrum of scientific experiments, including genomics and immune monitoring will be performed.

We anticipate accruing 1000 participants on this protocol

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Probability Sample
Study Population primary clinical
  • Urothelial Cancer
  • Bladder Cancer
  • Genitourinary Cancer
  • Urogenital Neoplasms
  • Urogenital Cancer
Intervention Not Provided
Study Groups/Cohorts
  • 1 / Urothelial cancer
    Participants with urothelial cancer.
  • 2 / Rare Bladder or Urinary Tract Histology
    Participants with small cell carcinoma, adenocarcinoma, urachal squamous cell carcinoma, or pure sarcomatoid carcinoma. These pure histologies can occur in the bladder and/or urinary tract.
  • 3 / Urothelial Carcinoma Variants
    Participants diagnosed with a urothelial variant histologies at any stage.
  • 4 / Rare GU Tumors
    Participants with renal medullary carcinoma, testicular Sertoli or Leydig cell tumors, penile cancer, refractory germ cell tumors.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: June 10, 2021)
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 1, 2042
Estimated Primary Completion Date December 1, 2041   (Final data collection date for primary outcome measure)
Eligibility Criteria
  • Participants must have histologically or cytologically confirmed urothelial or rare genitourinary cancer including but not limited to the following: small cell carcinoma of the bladder; adenocarcinoma of the bladder; squamous cell carcinoma of the bladder; plasmacytoid urothelial carcinoma; any penile cancer; any testicular cancer, sarcomatoid renal cell carcinoma; sarcomatoid urothelial carcinoma; renal medullary carcinoma or other miscellaneous histologic variants of the urothelial carcinoma, such as, but not limited to micropapillary, giant cell, lipid-rich, clear cell and nested variants, large cell neuroendocrine carcinoma, lymphoepithelioma-like carcinoma and mixed patterns will be considered, as well as small cell neuroendocrine prostate cancer, testicular Sertoli or Leydig cell tumors. Any genitourinary cancer can be included at the principal investigator s discretion.
  • Age >= 18 years.
  • Ability of subject to understand and the willingness to sign a written informed consent




Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contact: Tzu-Fang Wang, R.N. (240) 858-3236 tzu-fang.wang@nih.gov
Contact: Andrea B Apolo, M.D. (301) 480-0536 apoloab@mail.nih.gov
Listed Location Countries United States
Removed Location Countries  
Administrative Information
NCT Number NCT04923178
Other Study ID Numbers 10000302
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Yes
Plan Description: .All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
Supporting Materials: Study Protocol
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: Informed Consent Form (ICF)
Time Frame: Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active.
Access Criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI. @@@@@@Genomic data are made available via dbGaP through requests to the data custodians
Current Responsible Party National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )
Original Responsible Party Same as current
Current Study Sponsor National Cancer Institute (NCI)
Original Study Sponsor Same as current
Collaborators Not Provided
Principal Investigator: Andrea B Apolo, M.D. National Cancer Institute (NCI)
PRS Account National Institutes of Health Clinical Center (CC)
Verification Date May 15, 2023