Neoplasms

All Conditions

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Rare Diseases

Carcinoma

Carcinoma, Transitional Cell

Urinary Bladder Neoplasms

Neoplasms, Glandular and Epithelial

Neoplasms by Histologic Type

Transitional Cell Carcinoma

Jussi Nikkola, MD PhD

Peter Black

Riikka Järvinen

Tuomas Jalanko

Jussi Nikkola

Thea Veitonmäki

Matti Annala

Peter Boström

Otto Ettala

Urothelial cancers of the bladder and upper urinary tract account for over 200,000 deaths and 600,000 diagnoses annually worldwide. The most common presenting symptom is hematuria (blood in urine), triggering a cascade of tests, including an invasive examination of the bladder using a flexible scope (cystoscopy). Millions of cystoscopies are performed every year worldwide for patients presenting with hematuria, but only 10% result in a cancer diagnosis. The UROSCOUT-1 trial is a prospective multicenter observational study that explores the potential of urine tumor DNA (utDNA) testing to replace a significant portion of cystoscopies in the diagnostic setting for hematuria or other reasons to rule out urothelial cancer. The goal is to enhance patient quality of life, reduce healthcare costs, and address increased workloads in urology centers. Sample collection will be conducted by mail, and the samples will be analyzed in a blinded manner, without knowledge of which patients are diagnosed with cancer. Random subsampling will be applied to cancer-negative patients to achieve an approximate 1:1 ratio between cancer-positive and -negative patients.

Prospective Validation Study of High Volume Urine DNA Testing in Individuals With Suspicion of Urothelial Cancer

Observational Prospective Multicenter Validation Study Investigating the Possibility of Replacing Cystoscopies With High Volume Urine DNA Testing in Individuals With Suspicion of Urothelial Cancer (UROSCOUT-1)

Characteristics of urothelial cancers that were not detected by the utDNA test (TNM pathological stage, World Health Organization (WHO) 2004/2016 grade, and tumor size)

Concordance between utDNA and urine cytology results, with respect to positivity of the test

Sensitivity and specificity of utDNA test for urothelial cancer detection, using histologically confirmed cancer diagnoses within 1 year of the diagnostic workup as ground truth

Sensitivity and specificity of cystoscopy for urothelial cancer detection, using histologically confirmed cancer diagnoses within 1 year of the diagnostic workup as ground truth

Sensitivity and specificity or urine cytology for urothelial cancer detection, using histologically confirmed cancer diagnoses within 1 year of the diagnostic workup as ground truth

Time to diagnosis of urothelial cancer in patients with a positive utDNA test but negative cystoscopy

Adverse event rate for cystoscopy, based on CTCAE 5.0

Fraction of all analyzed urine samples for which the analysis met the quality control criteria for a technically successful analysis

Estimated fraction of diagnostic cystoscopies that could have been avoided by pre-screening patients with a utDNA test (calculated as the fraction of all diagnostic utDNA samples that were negative and for which subsequent cystoscopy did not lead to clinical interventions)

Specificity of a high-stringency utDNA threshold (at least 2 known urothelial cancer driver mutations with allele fraction \> 5%) for clinically detectable urothelial cancer

Tampere University

Tampere University Hospital

Urethral Cancer

All Drugs and Chemicals

Vaccines

Immunomodulating Agents

Subjects received infusion of DN24-02, at 2-week intervals, for a total of 3 infusions.

Subjects randomized to the control arm were treated per standard of care, which in this patient population is generally observation, as there is currently no evidence that treatment with non-cisplatin containing chemotherapy is beneficial in the adjuvant setting for this patient population.

DN24-02 is an autologous cellular immunotherapy product designed to stimulate an immune response against HER2/neu. It consists of autologous peripheral blood mononuclear cells (PBMCs), including antigen presenting cells (APCs), which are activated ex vivo with a recombinant fusion protein, BA7072.

DN24-02

Observation only until documentation of disease recurrence.

Standard of Care

Robert Israel, MD

This study was conducted to examine survival, disease-free survival, safety, and the magnitude of the immune response induced following administration of DN24-02 in subjects with HER2+ urothelial carcinoma.

Multicenter, open-label, Phase 2 study. Subjects were randomized to either the investigational product, DN24-02, or to standard of care. Subjects randomized to the experimental arm received DN24-02 at 2-week intervals, for a total of 3 infusions. The study evaluated survival, disease-free survival, safety and the magnitude of the immune response between these 2 subject groups.

DN24-02 as Adjuvant Therapy in Subjects With High Risk HER2+ Urothelial Carcinoma

A Randomized, Phase 2, Open-label Study Evaluating DN24-02 as Adjuvant Therapy in Subjects With High Risk HER2+ Urothelial Carcinoma

Overall survival is defined as the time from randomization to death due to any cause.

\*This study was terminated early due to administrative reasons.

Dendreon

Adverse events are reported in the safety population. The safety population was defined as all subjects in the treatment arm who underwent at least 1 leukapheresis and all randomized control subjects with at least 1 post-baseline safety assessment or visit.

Subjects randomized to the control arm were treated per standard of care, which in this patient population is generally observation, as there is currently no evidence that treatment with non-cisplatin-containing chemotherapy is beneficial in the adjuvant setting for this patient population.

Total

Age, Categorical

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

Baseline weight for one subject in standard of care group is missing.

Body weight

ECOG 0=Fully Active; No restrictions

ECOG 1= Restricted Strenuous Activity

ECOG 2=Ambulatory, capable of self-care; no work

ECOG Performance Status is a method used to assess the functional status of a patient. The scale ranges from 0-5. 0=Fully active, able to carry on all pre-disease performance without restriction; 1=Restricted in physically strenuous activity but ambulatory and able to carry out light or sedentary work; 2=Ambulatory, capable of all self-care but unable to carry out work activities. Up and about \>50% of waking hour; 3=Capable of limited self-care, confined to bed or chair \>50% of waking hours; 4=Completely disabled. Cannot carry on any self-care. Totally confined to bed or chair; 5=Dead

Baseline ECOG data is missing for one subject in each group.

Eastern Cooperative Oncology Group (ECOG) Performance Status

Shabnam Vaziri

Subjects randomized to the control arm were treated per standard of care, which in this patient population is generally observation, as there is currently no evidence that treatment with non-cisplatin containing chemotherapy is beneficial in the adjuvant setting for this patient population.

Standard of Care: Standard of care

Intent to Treat (ITT) population.

\*This study was terminated early due to administrative reasons.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Prospective Validation Study of High Volume Urine DNA Testing in Individuals With Suspicion of Urothelial Cancer	NCT06310759	Urothelial Carc...		18 Years - 100 Years	Tampere University Hospital
DN24-02 as Adjuvant Therapy in Subjects With High Risk HER2+ Urothelial Carcinoma	NCT01353222	Urothelial Carc...	DN24-02 Standard of Car...	18 Years -	Dendreon
DN24-02 as Adjuvant Therapy in Subjects With High Risk HER2+ Urothelial Carcinoma	NCT01353222	Urothelial Carc...	DN24-02 Standard of Car...	18 Years -	Dendreon

Spots Global Cancer Trial Database for urothelial neoplasms