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Study of BT-001 and Pembrolizumab for Patients with Advanced Solid Tumors, Including Sarcoma, Merkel Cell Carcinoma, Melanoma, Breast, and Lung 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 several types of advanced cancers, including metastatic or advanced soft tissue sarcoma, Merkel cell carcinoma, melanoma, triple-negative breast cancer, and non-small cell lung cancer. The study is testing a new treatment approach using a medication called BT-001, which is administered directly into the tumor. This treatment is being tested alone and in combination with another medication called pembrolizumab, which is given through an intravenous infusion. The purpose of the study is to evaluate the safety and effectiveness of these treatments in patients with these types of cancers.

Participants in the study will receive the treatment over a period of time, with regular monitoring to assess how their body responds to the medication. The study will look at both the local effects at the site of the tumor and the overall effects on the body. The goal is to determine the best dose of BT-001 and to see how well it works when used with pembrolizumab. Some participants may receive a placebo as part of the study to help compare the effects of the actual treatment.

Throughout the study, participants will undergo various assessments to monitor their health and the progression of their cancer. This includes regular check-ups and tests to ensure the treatment is safe and to measure its impact on the cancer. The study aims to provide valuable information on the potential benefits of combining BT-001 with pembrolizumab for treating these challenging types of 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

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and current health status.

    A fresh tumor sample from the lesion to be injected first is required. This helps in understanding the specific characteristics of the tumor.

  2. Step 2

    Treatment phase i part a

    In this phase, **BT-001** is administered alone through injections directly into the tumor. The dosage ranges from 10 million to 100 million plaque-forming units per milliliter (PFU/mL).

    The main goal is to assess the safety and tolerability of **BT-001** when given repeatedly.

  3. Step 3

    Treatment phase i part b

    In this phase, **BT-001** is combined with **pembrolizumab**, which is given through an intravenous infusion.

    The focus is on evaluating the safety and tolerability of this combination treatment.

  4. Step 4

    Treatment phase ii

    This phase involves repeated administration of **BT-001** in combination with **pembrolizumab**.

    The objective is to evaluate the antitumor activity of the combination treatment.

  5. Step 5

    Monitoring and follow-up

    Throughout the trial, regular monitoring is conducted to track the response to treatment and any side effects.

    Follow-up visits are scheduled to assess overall health and the effectiveness of the treatment.

Who can join the trial?

13 criteria

  • Provide signed written informed consent, which means you agree to participate in the study after understanding all the details.
  • Use a highly effective contraception method combined with a barrier method during and after the study treatment to prevent pregnancy.
  • Have received complete COVID-19 primary vaccination at least 30 days before the first study treatment.
  • Have an interval of at least 3 weeks between the first study treatment and your last chemotherapy session.
  • Have adequate hematological (blood), hepatic (liver), and renal (kidney) functions, meaning these organs are working well enough.
  • Be a male or female patient aged 18 years or older.
  • Have a confirmed diagnosis of advanced or metastatic sarcoma, Merkel cell carcinoma, melanoma, triple negative breast cancer, or non-small cell lung cancer, with lesions that can be easily injected.
  • Have tried and not responded to standard treatment options or cannot tolerate them.
  • Have at least one lesion that can be injected, which could be on the skin, under the skin, or in a lymph node.
  • Have an expected survival of at least 3 months.
  • Know your anti-variola vaccine status, which refers to whether you have been vaccinated against smallpox.
  • Agree to provide a fresh tumor sample from the lesion that will be injected first.
  • Have an Eastern Cooperative Oncology Group performance status of 0 or 1, which means you are fully active or have some symptoms but can carry out light work.

Who cannot join the trial?

10 criteria

  • Patients with other types of cancer not listed in the study.
  • Patients who have not reached the age required for the study.
  • Patients who are not able to give their consent to participate.
  • Patients who are pregnant or breastfeeding.
  • Patients with severe or uncontrolled medical conditions that could interfere with the study.
  • Patients who have had another cancer treatment recently that might interfere with the study.
  • Patients with known allergies to the study medication or its components.
  • Patients who are participating in another clinical trial at the same time.
  • Patients with a history of certain heart conditions.
  • Patients with active infections that require treatment.
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Investigated drugs

  • BT-001

    is an experimental treatment being tested in this clinical trial. It is designed to be injected directly into tumors or nearby lymph nodes. The goal of this treatment is to see if it can help the body's immune system fight cancer more effectively. Researchers are looking at how safe it is for patients and how well it works when used alone or with another medication.

  • Pembrolizumab

    is a medication that helps the immune system attack cancer cells. It is given through an intravenous (IV) infusion, which means it is delivered directly into the bloodstream. In this trial, pembrolizumab is used in combination with BT-001 to see if the two treatments together can improve the body's ability to fight cancer. Researchers are studying how well this combination works and how safe it is for patients. Pembrolizumab is already used in treating some types of cancer, and this trial is exploring its effectiveness with the new treatment, BT-001.

What is already known about the treatment

  • BT-001

    BT-001 is administered directly into tumors through injections, specifically targeting skin or easily accessible lymph node tumors. It is currently being studied in clinical trials to assess its safety and effectiveness, both alone and in combination with another drug called pembrolizumab. The main therapeutic indications for BT-001 include various advanced cancers such as soft tissue sarcoma, Merkel cell carcinoma, melanoma, triple-negative breast cancer, and non-small cell lung cancer. At the molecular level, BT-001 works by stimulating the immune system to attack cancer cells. It is classified as an oncolytic virus therapy, which means it uses a virus to target and destroy cancer cells.

  • Pembrolizumab

    Pembrolizumab is given through intravenous infusions, meaning it is administered directly into the bloodstream. It is a well-established medication in the medical field, widely used for treating several types of cancer. In this trial, it is being tested in combination with BT-001 to enhance its effectiveness against tumors. Pembrolizumab is primarily used for treating cancers like melanoma, non-small cell lung cancer, and others. It works by blocking a specific protein on immune cells, allowing them to better recognize and attack cancer cells. Pembrolizumab is classified as an immune checkpoint inhibitor, a type of drug that helps the immune system fight cancer.

Investigated diseases

  • Soft Tissue Sarcoma

    Soft tissue sarcoma is a type of cancer that begins in the tissues that connect, support, and surround other body structures. These tissues include muscle, fat, blood vessels, nerves, tendons, and the lining of the joints. The disease can develop anywhere in the body but is most common in the arms, legs, and trunk. It often starts as a painless lump, which may grow and press against nerves or muscles, causing pain or discomfort. As it progresses, it can spread to other parts of the body, including the lungs. The progression can vary significantly depending on the specific type of sarcoma and its location.

  • Merkel Cell Carcinoma

    Merkel cell carcinoma is a rare and aggressive form of skin cancer that originates in the Merkel cells, which are found at the base of the outermost layer of the skin. It typically appears as a fast-growing, painless nodule on sun-exposed areas of the skin, such as the face, neck, or arms. The disease can spread quickly to nearby lymph nodes and other parts of the body. As it progresses, it may cause swelling in the lymph nodes and other symptoms depending on where it spreads. The exact cause of this cancer is not fully understood, but it is often associated with a virus called Merkel cell polyomavirus. The progression can be rapid, necessitating prompt medical attention.

  • Melanoma

    Melanoma is a type of skin cancer that develops from the pigment-producing cells known as melanocytes. It is most commonly found on the skin but can also occur in the eyes and, rarely, in internal organs. The disease often begins as a change in an existing mole or as a new, unusual-looking growth on the skin. As melanoma progresses, it can invade deeper layers of the skin and spread to other parts of the body, including the lymph nodes, liver, lungs, and brain. The progression of melanoma can vary, with some forms growing and spreading more quickly than others. Early detection is crucial for managing the disease effectively.

  • Triple Negative Breast Cancer

    Triple negative breast cancer is a type of breast cancer that lacks the three most common types of receptors known to fuel most breast cancer growth: estrogen, progesterone, and the HER-2/neu gene. This form of cancer is more aggressive and has fewer treatment options compared to other types of breast cancer. It often grows and spreads more quickly than other types of breast cancer. The disease typically presents as a lump or mass in the breast, which may be accompanied by changes in breast shape or skin texture. As it progresses, it can spread to nearby lymph nodes and other parts of the body. The progression can be rapid, requiring prompt medical intervention.

  • Non-Small Cell Lung Cancer

    Non-small cell lung cancer is the most common type of lung cancer, accounting for about 85% of cases. It begins in the tissues of the lungs and can spread to other parts of the body. The disease often starts with symptoms such as a persistent cough, chest pain, and shortness of breath. As it progresses, it may cause more severe symptoms, including weight loss, fatigue, and coughing up blood. The cancer can spread to nearby lymph nodes and other organs, such as the brain, liver, and bones. The progression of non-small cell lung cancer can vary, with some forms growing and spreading more quickly than others.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-518529-14-00Protocol codeBT-001.01Estimated enrolment30 patientsSponsorTransgene

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