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Study on the Safety and Effectiveness of Padeliporfin Therapy for Patients with Low-Grade Upper Tract Urothelial Cancer

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What is this trial about?

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

This clinical trial is focused on studying a type of cancer called Low-Grade Upper Tract Urothelial Cancer (UTUC). This cancer affects the lining of the urinary system, specifically in areas like the kidney and ureter. The study is testing a treatment called TOOKAD (padeliporfin), which is a special therapy known as Vascular Targeted Photodynamic Therapy (VTP). This treatment involves using a drug that is activated by light to target and treat the cancer cells.

The purpose of the study is to evaluate how safe and effective TOOKAD is in treating this type of cancer. Participants in the study will receive the treatment through an intravenous infusion, which means the drug is given directly into a vein. The drug is then activated by a laser light delivery system, which helps to target the cancer cells more precisely. The study will monitor the participants over a period of time to see how well the treatment works and to check for any side effects.

Throughout the study, researchers will look for the absence of cancer in the treated areas, such as the kidney and ureter, using methods like endoscopy, which is a procedure that allows doctors to look inside the body. The study will also assess the overall health of the kidney and whether the treatment helps in preserving kidney function. Safety follow-ups will be conducted to record any adverse events experienced by the participants. The study aims to provide valuable information on the effectiveness and safety of TOOKAD in treating Low-Grade Upper Tract Urothelial Cancer.

The research process

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

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria, including age, understanding of the study, and medical condition.

    A biopsy confirms the presence of low-grade upper tract urothelial cancer (UTUC) in specific areas of the kidney.

  2. Step 2

    Treatment preparation

    Preparation involves understanding the treatment process and potential outcomes.

    The treatment uses TOOKAD (padeliporfin), a medication administered through an intravenous infusion.

  3. Step 3

    Treatment administration

    The medication TOOKAD (padeliporfin) is given as a solution for injection.

    The treatment is known as vascular targeted photodynamic therapy (VTP), which targets the cancerous areas.

  4. Step 4

    Post-treatment evaluation

    After treatment, an endoscopic evaluation checks for the absence of UTUC tumors.

    Success is defined by no visible tumors, negative biopsy results, and negative urinary cytology.

  5. Step 5

    Follow-up and monitoring

    Regular follow-ups assess the duration of response and overall kidney function.

    Safety monitoring includes recording any adverse events experienced during the study.

Who can join the trial?

13 criteria

  • Male and female patients who are 18 years or older.
  • Must be able to understand and provide written consent, and agree to follow all tests and procedures in the study.
  • Have a new or returning low-grade, non-invasive **UTUC** disease. **UTUC** stands for **Upper Tract Urothelial Cancer**, which affects the urinary system.
  • The disease must be confirmed by a biopsy, which is a small sample of tissue taken for testing. A central pathology expert must agree with the diagnosis for eligibility.
  • Can have up to 2 confirmed sites of low-grade cancer, with the largest tumor being between 5 mm and 15 mm in size, located in the calyces, renal pelvis, or ureter of the same kidney. There should be no high-grade cells found in urine tests. If the ureter is involved, it should be in one area and not more than 20 mm in length.
  • Must have a **Karnofsky Performance Status** of 50% or higher. This is a scale that measures the ability to perform daily activities, with higher percentages indicating better function.
  • Must have adequate organ function at the start of the study, defined as:
    • **ANC** (Absolute Neutrophil Count) of 1,000 or more per microliter. Neutrophils are a type of white blood cell important for fighting infections.
    • **Platelets** of 75,000 or more per microliter. Platelets help with blood clotting.
    • **Hemoglobin (Hb)** of 9 grams per deciliter or more. Hemoglobin is a protein in red blood cells that carries oxygen.
    • **INR** (International Normalized Ratio) of 2 or less. This is a measure of blood clotting.
    • **Estimated glomerular filtration rate (eGFR)** of 30 ml/min or more, using the **CKD-EPI Method**. This measures kidney function.
    • **Total serum bilirubin** less than 3 mg/dL. Bilirubin is a substance made during the breakdown of red blood cells.
    • **AST/ALT** levels that are 5 times or less the upper limit of normal. These are liver enzymes, and their levels indicate liver health.
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Investigated drugs

TOOKAD (padeliporfin) is a medication used in a therapy called Vascular Targeted Photodynamic Therapy (VTP). This treatment is designed to target and destroy cancer cells in specific areas of the body. In this clinical trial, TOOKAD is being used to treat low-grade upper tract urothelial cancer, which affects parts of the urinary system such as the calyces, renal pelvis, and ureter. The therapy works by activating the medication with light, which then helps to destroy the cancer cells while minimizing damage to surrounding healthy tissue.

What is already known about the treatment

TOOKAD (padeliporfin) – This medication is administered through a procedure called vascular targeted photodynamic therapy (VTP), where it is activated by light to target cancer cells. It is currently being studied in clinical trials for its effectiveness in treating low-grade upper tract urothelial cancer, specifically in areas like the calyces, renal pelvis, and ureter. The main therapeutic indication is for low-grade urothelial cancer in the upper urinary tract. At the molecular level, TOOKAD works by generating reactive oxygen species when exposed to light, which damages the blood vessels supplying the tumor, leading to cancer cell death. It is classified as a photodynamic therapy agent.

Investigated diseases

Low Grade Upper Tract Urothelial Cancer – This is a type of cancer that occurs in the lining of the upper urinary tract, which includes the renal pelvis and ureter. It is characterized by the presence of abnormal cells that grow slowly and are less likely to spread compared to high-grade cancers. The disease progresses as these abnormal cells form tumors, which can obstruct the flow of urine and cause symptoms such as blood in the urine or pain. Over time, the tumors may grow larger and affect kidney function. The progression of the disease can vary, with some tumors remaining stable for long periods while others may grow or recur after treatment.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-512322-28-00Protocol codeCLIN2001UCM301Estimated enrolment120 patientsSponsorSteba Biotech S.A.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).