Safety and efficacy study of nadofaragene firadenovec administered to the renal pelvis in adults with low-grade upper tract urothelial carcinoma

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What is this study about?

This study focuses on treating patients with Low-grade Upper Tract Urothelial Carcinoma (LG-UTUC), which is a type of cancer that develops in the lining of the kidney’s collecting system. The treatment being tested is nadofaragene firadenovec (also known as ADSTILADRIN), which is given as a liquid that is delivered directly into the kidney through a procedure called intravesical administration.

The purpose of this research is to determine if this treatment is both safe and effective for patients with this specific type of kidney cancer. During the study, participants will receive the medication directly into their renal pelvis (the part of the kidney that collects urine). The treatment will be monitored over time to see how well it works in treating the cancer.

This is a single-arm, open-label study, which means all participants will receive the same treatment, and there is no comparison group. The study will track how patients respond to the treatment over an extended period, with particular attention paid to any changes in their cancer status and any side effects that may occur. Participants will be followed for at least 18 months after receiving their first dose of the medication.

1 Initial assessment

Your eligibility for the trial will be confirmed through a review of your medical history and laboratory test results

A biopsy confirmation of low-grade upper tract urothelial cancer within the last 2 months is required

You must have at least one measurable tumor (5-15 mm in size) above the ureteropelvic junction

2 Laboratory testing

Blood tests will be performed to check your:

– Blood count (hemoglobin, white blood cells, platelets)

– Liver function (AST, ALT, bilirubin)

– Kidney function (estimated glomerular filtration rate)

– Blood clotting parameters

– Sodium and potassium levels

3 Treatment administration

You will receive nadofaragene firadenovec (ADSTILADRIN) as a suspension directly into the renal pelvis

The medication will be administered through a procedure called intravesical instillation

4 Follow-up evaluations

Your response to treatment will be assessed at 3 and 6 months

Assessment includes:

– Urine tests to check for cancer cells

– Visual examination of the urinary tract (ureteroscopy)

– Additional tissue samples (biopsies) if necessary

5 Safety monitoring

You will be monitored for any side effects throughout the trial

Regular check-ups will continue for at least 18 months after your first treatment

Who Can Join the Study?

Must be 18 years or older
Must have adequate blood test results including:
- Hemoglobin level of at least 10 g/dL (a protein that carries oxygen in blood)
- White blood cell count of at least 4000/µL (cells that fight infection)
- Neutrophil count of at least 2000/µL (a type of white blood cell)
- Platelet count of at least 100,000/µL (blood cells that help with clotting)
- Normal blood clotting tests (INR and aPTT)
- Liver function tests (AST and ALT) no more than 1.5 times above normal
- Normal levels of sodium and potassium in blood
Must have good kidney function with filtration rate of at least 45 mL/min
Must be able to provide written consent
Must have confirmed low-grade upper tract urothelial cancer by biopsy within 2 months before joining
Must have at least one measurable tumor between 5-15 millimeters in size
Must be willing to participate for at least 18 months
Must have life expectancy of more than 2 years
Must be able to perform daily activities with minimal assistance (ECOG status 2 or less)
For women who can become pregnant:
- Must have negative pregnancy test
- Must use effective birth control during treatment and for 6 months after
For men:
- Must use effective birth control and condoms during treatment and for 3 months after

Who Cannot Join the Study?

Age below 18 years old
Presence of high-grade urothelial carcinoma (more aggressive form of cancer in the urinary system)
Previous treatment with nadofaragene firadenovec (the study medication)
Currently pregnant or breastfeeding women
Active infections in the urinary system
Major surgery within the last 4 weeks
Participation in other clinical trials within the last 30 days
Known allergies to components of the study medication
Severe kidney function problems
History of organ transplantation
Active or chronic autoimmune diseases (conditions where the immune system attacks healthy cells)
Unstable medical conditions that could interfere with the study procedures
Mental conditions that could affect the ability to provide informed consent
History of other cancers in the last 2 years (except for successfully treated non-melanoma skin cancer)

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Hopitaux Universitaires Pitie Salpetriere	Paris	France
Hopital Beaujon	Clichy	France
Fundacio Puigvert	Barcelona	Spain
Ajoitfgzh Ucl	Amsterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
France	Not yet recruiting	01.02.2026
Spain	Not yet recruiting	01.02.2026
The Netherlands	Not yet recruiting	01.02.2026

Trial locations

Investigated drugs:

NADOFARAGENE FIRADENOVEC

Nadofaragene firadenovec is a gene therapy medication designed to treat upper tract urothelial carcinoma (cancer in the lining of the upper urinary tract). It is administered by direct instillation into the renal pelvis (part of the kidney). This therapy works by delivering genetic material to help fight cancer cells in the urinary system. It is being studied as a potential treatment option for patients with low-grade tumors in this area.

Investigated diseases:

Transitional cell carcinoma

Low-grade Upper Tract Urothelial Carcinoma – A type of cancer that develops in the lining of the upper urinary tract, specifically in the renal pelvis or ureter. This cancer grows slowly and tends to stay within the inner layer of the urinary tract tissue. The cancer cells in this condition are well-differentiated, meaning they still look similar to normal cells. It typically develops as small growths or tumors on the inner surface of the urinary tract. The condition can occur in one or both kidneys and their connecting tubes.

Trial ID:

2024-514360-70-00

Protocol code:

000425

Trial Phase:

Human Pharmacology (Phase I) – Other

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Safety and efficacy study of nadofaragene firadenovec administered to the renal pelvis in adults with low-grade upper tract urothelial carcinoma

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?