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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. Identifier: NCT04923178
Recruitment Status : Not yet recruiting
First Posted : June 11, 2021
Last Update Posted : September 16, 2021
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) )

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.

Condition or disease
Urothelial Cancer Bladder Cancer Genitourinary Cancer Urogenital Neoplasms Urogenital Cancer

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 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

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Study Type : Observational
Estimated Enrollment : 1000 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Natural History of Urothelial Cancer and Rare Genitourinary Tract Malignancies
Estimated Study Start Date : September 21, 2021
Estimated Primary Completion Date : December 1, 2041
Estimated Study Completion Date : December 1, 2042

1/Non-invasive urothelial cancer
Participants with high-grade and, low-grade non-invasive bladder cancer
2/Muscle invasive bladder and high-grade upper tract urothelial cancer
Participants with muscle invasive bladder and invasive upper tract urothelial cancer
3/Variant urothelial cancer
Participants diagnosed with a urothelial variant histologies at any stage
4/Metastatic Urothelial cancer
Participants diagnosed with urothelial cancer who cannot be placed in cohorts 1-3
5/Localized rare genitourinary tumors
Participants with localized rare genitourinary tumors treated with curative intent
6/Locally advanced and metastatic rare genitourinary tumors
Participants with locally advanced and metastatic rare genitourinary tumors

Primary Outcome Measures :
  1. 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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
primary clinical
  • 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




Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04923178

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Contact: Olena Sierra Ortiz (240) 858-3185

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United States, Maryland
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office    888-624-1937      
Sponsors and Collaborators
National Cancer Institute (NCI)
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Principal Investigator: Andrea B Apolo, M.D. National Cancer Institute (NCI)
Additional Information:
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Responsible Party: National Cancer Institute (NCI) Identifier: NCT04923178    
Other Study ID Numbers: 10000302
First Posted: June 11, 2021    Key Record Dates
Last Update Posted: September 16, 2021
Last Verified: September 9, 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Cancer Institute (NCI) ):
bladder/urachal adenocarcinoma
renal tumors
penile cancers
small cell neuroendocrine carcinoma of the prostate
Renal Cell Carcinoma
Additional relevant MeSH terms:
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Urogenital Neoplasms
Neoplasms by Site