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Study on Pembrolizumab and Cyclophosphamide for Patients with Metastatic Urothelial Cancer

Verified siteRegistered drugNo placebo
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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 metastatic urothelial cancer, a type of cancer that affects the urinary system and has spread to other parts of the body. The study involves the use of two treatments: Pembrolizumab, a medication that helps the immune system fight cancer, and cyclophosphamide monohydrate, a chemotherapy drug known by the brand name Endoxan®. The purpose of the study is to evaluate the effectiveness of these treatments when used together in patients whose cancer has progressed despite previous treatments.

Participants in the study will receive Pembrolizumab and oral doses of cyclophosphamide over a period of six months. The study aims to observe how well the cancer is controlled during this time. Some participants will receive a placebo instead of cyclophosphamide to compare the effects. The study will also look at how long the cancer responds to treatment, the overall control of the disease, and the time it takes for the cancer to progress further. Additionally, researchers will collect blood and tissue samples to explore how the body responds to the treatment and to identify any markers that might predict treatment success.

The trial is designed to provide valuable information about the potential benefits of combining Pembrolizumab with cyclophosphamide for treating metastatic urothelial cancer. The study will continue until July 2025, and the results will help determine if this combination of treatments can offer a new option for patients with this type of cancer.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, diagnosis of urothelial carcinoma, and ongoing treatment with pembrolizumab.

    The assessment also checks for adequate blood, kidney, and liver function, as well as the presence of measurable disease according to specific criteria.

  2. Step 2

    Treatment initiation

    The treatment phase begins with the administration of Endoxan® (cyclophosphamide monohydrate) in the form of a coated tablet.

    The medication is taken orally. The specific dosage and frequency are determined by the study protocol and the patient's condition.

  3. Step 3

    Ongoing treatment and monitoring

    Throughout the study, regular monitoring is conducted to assess the patient's response to treatment and overall health.

    This includes evaluating the non-progressive rate at 6 months, as well as secondary measures such as duration of response, disease control rate, and time to progression.

  4. Step 4

    Completion of study

    The study is estimated to conclude by July 10, 2025. At this point, a final assessment is conducted to evaluate the overall outcomes and any long-term effects of the treatment.

Who can join the trial?

12 criteria

  • Must be at least 18 years old.
  • Must have a confirmed diagnosis of **urothelial carcinoma**, which is a type of bladder cancer, that is advanced or has spread to other parts of the body.
  • Must have **PD-L1 expression**, which is a protein found on some cancer cells, determined by a specific test in a local laboratory.
  • Must have an **ECOG performance status** of 2 or less, which is a scale used to assess how a disease affects a patient's daily living abilities.
  • Must have adequate blood, kidney, and liver function.
  • Must have measurable disease according to **RECIST 1.1**, which is a set of guidelines to evaluate how well a cancer treatment is working.
  • Must be currently receiving **Pembrolizumab**, a type of cancer treatment, after progression following platinum-based chemotherapy.
  • Must have disease progression according to **RECIST criteria**, which means the cancer has grown or spread.
  • Must meet criteria for receiving **ICIs** (immune checkpoint inhibitors) beyond RECIST progression, which includes clinical benefit, stable condition, tolerance of treatment, and no immediate need for intervention.
  • Must have **iUPD** (immune unconfirmed progressive disease) according to **iRECIST criteria**, which requires confirmation through imaging tests.
  • Must have **oligometastatic disease**, meaning a minimal state of cancer spread with only a few sites affected.
  • Must provide written informed consent to participate in the study.

Who cannot join the trial?

3 criteria

  • Patients who have a different type of cancer than metastatic urothelial cancer cannot participate. Metastatic means the cancer has spread to other parts of the body.
  • Patients who are not within the specified age range cannot participate. The age range includes adults and older adults.
  • Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups who may need special protection, like children or people with certain disabilities.
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Investigated drugs

  • Pembrolizumab

    is a type of immunotherapy used in this trial. It works by helping the immune system recognize and attack cancer cells. It is often used in various types of cancer treatments to slow down or stop the growth of cancer cells.

  • Cyclophosphamide

    is a chemotherapy drug used in this trial. It works by slowing or stopping the growth of cancer cells. In this study, it is used in a low-dose, continuous manner, known as metronomic therapy, to help manage metastatic urothelial cancer.

What is already known about the treatment

  • Pembrolizumab

    This medication is administered intravenously and is used in the treatment of metastatic urothelial cancer. It is currently being studied in clinical trials to assess its effectiveness beyond traditional progression criteria. Pembrolizumab works by blocking a specific protein on immune cells, allowing the immune system to better attack cancer cells. It is classified as an immune checkpoint inhibitor and is widely recognized in medical literature for its role in cancer immunotherapy.

  • Cyclophosphamide

    This medication is taken orally and is used in combination with other treatments for metastatic urothelial cancer. It is part of ongoing clinical trials to evaluate its effectiveness in a metronomic dosing schedule, which involves low, regular doses. Cyclophosphamide works by interfering with the DNA of cancer cells, preventing them from growing and dividing. It is classified as an alkylating agent and has been used in various cancer treatments for many years.

Investigated diseases

Metastatic Urothelial Cancer – This is a type of cancer that begins in the urothelial cells lining the bladder and can spread to other parts of the body. It often starts in the bladder but can also occur in the ureters and other parts of the urinary tract. As the disease progresses, cancer cells can travel through the lymphatic system or bloodstream to distant organs, forming new tumors. Symptoms may include blood in the urine, frequent urination, and pain during urination. Over time, the disease can lead to more severe symptoms as it affects other organs. The progression of the disease can vary, with some cases advancing more rapidly than others.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-514979-17-04Estimated enrolment43 patientsSponsorI.F.O. Istituti Fisioterapici Ospitalieri

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