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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

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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
Step 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
Step 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
Step 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
Step 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 trial?

18 criteria

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 trial?

14 criteria

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)

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Responsive
Amsterdam UMC
Amsterdam, The Netherlands
Verified
Fundacio Puigvert
Barcelona, Spain
Verified
Hopital Beaujon
Clichy, France

Trial locations

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Investigated drugs

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.

NADOFARAGENE FIRADENOVEC

What is already known about the treatment

Nadofaragene firadenovec - A novel gene therapy medication administered directly into the renal pelvis through instillation for treating low-grade upper tract urothelial carcinoma (LG-UTUC). This investigational drug works by delivering genetic material that helps produce interferon alpha-2b protein, which stimulates the immune system to fight cancer cells in the urinary tract. The medication belongs to the class of gene therapy agents and is currently being evaluated in clinical trials to assess its safety and effectiveness as a potential alternative to conventional treatments for patients with LG-UTUC, showing promising results in early studies.

Investigated diseases

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.