AuthorisedPhase I and Phase II (Integrated)- OtherInterventional

A Phase 1/2, Single-arm, Open-Label Trial to Evaluate the Safety and Efficacy of Nadofaragene Firadenovec Instilled to the Renal Pelvis in Adult Subjects with Low-Grade Upper Tract Urothelial Carcinoma (LG-UTUC)

This research study is looking at a new medicine called ADSTILADRIN for adults who have a specific type of cancer in the upper urinary tract, known as low-grade upper tract urothelial carcinoma (LG-UTUC). This cancer affects the renal pelvis, which is part of the kidney where urine collects, and the tubes (ureters) that carry urine to the bladder. The main goals of the study are to find out if ADSTILADRIN is safe and if it can effectively treat this cancer. Researchers will be checking for any side effects and seeing if the treatment can make the cancer disappear, based on tests like urine samples and internal examinations.

At a glance

Status

Authorised

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Ferring Pharmaceuticals A/S

Enrolment target

Start

16 Sep 2025

Low-grade upper track urothelial carcinoma (LG-UTUC)

What is this study about?

This study is designed to explore a new way of treating a type of cancer called low-grade upper tract urothelial carcinoma, often shortened to LG-UTUC. This cancer affects the 'plumbing' of your body that handles urine – specifically the renal pelvis (the funnel-shaped part of your kidney that collects urine) and the tubes that carry urine from your kidneys to your bladder. For people with LG-UTUC, current treatment options might involve surgery, but this study is looking at a new medicine that could offer another approach.

The new treatment being tested is called ADSTILADRIN. Instead of being taken as a pill or injection, ADSTILADRIN is given directly into the renal pelvis, where the cancer is located. The idea is to target the cancer cells more directly. Because this is a newer treatment for this specific condition, researchers need to carefully assess how well it works and if it causes any unwanted side effects. It’s important to find new and better ways to treat these cancers, and studies like this help medical experts learn more about potential new medicines.

This study is in what's called a 'Phase 1/2' stage. This means it's still relatively early research. In Phase 1, the main focus is on safety – making sure the treatment doesn't cause serious harm. In Phase 2, if it seems safe, the study then starts to look more closely at how effective the treatment is, meaning how well it makes the cancer go away or shrink. By doing these integrated phases, researchers can gather information on both safety and effectiveness at the same time, helping them decide if this treatment should be studied further.

Key takeaways

This study is testing a new treatment (ADSTILADRIN) for a specific type of kidney/urinary tract cancer.
The treatment is given directly into the affected area.
Researchers are looking for both safety and effectiveness.
It's for adults 18 and over with low-grade upper tract urothelial carcinoma.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you would need to be an adult, as the study is for people aged 18 and older. There isn't an upper age limit mentioned, so older adults could also potentially take part.

This study is specifically for individuals diagnosed with low-grade upper tract urothelial carcinoma (LG-UTUC). This means the cancer must be in your renal pelvis (part of the kidney) or ureter (tube connecting kidney to bladder) and it needs to be of the 'low-grade' type.

Both men and women are welcome to participate, as the study is open to all sexes.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Have you been diagnosed with low-grade upper tract urothelial carcinoma (LG-UTUC)?
Is your cancer located in the renal pelvis (part of your kidney)?
Are you able to attend regular appointments for treatment and check-ups?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, the main part of the treatment involves receiving the study medicine, ADSTILADRIN, directly into your renal pelvis. This would be done by a medical professional. The study team will explain exactly how often and for how long you will receive this treatment. Throughout the study, you would have regular appointments to monitor your health and check how the treatment is working. This will include keeping track of any new health problems or symptoms you experience, which the study calls 'treatment-emergent adverse events'. The medical team will also perform specific tests, such as urine tests to check for cancer cells and possibly an examination called a ureteroscopy, where a thin tube with a camera is used to look inside your urinary tract, or biopsies (taking small tissue samples) if needed. The visits and tests are designed to assess both the safety of the treatment and whether it is effectively clearing up the cancer. The total duration of your involvement in the study, including all treatments and follow-up checks, would be fully explained by the study team.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. For this study, a potential benefit is that ADSTILADRIN could effectively treat your low-grade upper tract urothelial carcinoma, potentially shrinking or clearing the cancer. However, as with any new medicine, there are potential risks, such as side effects from the treatment, which the study team will carefully monitor. You would be fully informed about these potential risks and benefits before deciding to join. It's very important to remember that participation is entirely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Spain
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Unverified
France
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Unverified
Netherlands

Common questions

What is low-grade upper tract urothelial carcinoma (LG-UTUC)?

It's a type of slow-growing cancer found in the renal pelvis (part of your kidney) or the ureters (tubes connecting kidneys to the bladder).

What is ADSTILADRIN?

ADSTILADRIN is the new medicine being tested in this study. It's given directly into the area where the cancer is located.

What does 'Phase 1/2' mean?

It means the study is checking both the safety of the treatment (Phase 1) and how well it works to treat the cancer (Phase 2).

Will I know if the treatment is working?

The study team will monitor your progress with tests like urine samples and internal examinations to see if the cancer disappears.

Can I leave the study if I change my mind?

Yes, you have the right to leave the study at any time without it affecting your regular medical care.