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Study on the Safety and Effectiveness of Patritumab Deruxtecan for Children with Relapsed or Refractory Hepatoblastoma and Rhabdomyosarcoma

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 the effects of a new treatment called patritumab deruxtecan (also known by its code name MK-1022) in children with certain types of cancer. The specific cancers being studied are hepatoblastoma and rhabdomyosarcoma, which are types of solid tumors that have either returned after treatment or have not responded to previous treatments. The purpose of the study is to evaluate the safety and effectiveness of this new treatment in these young patients.

The study will involve administering patritumab deruxtecan to participants through an intravenous infusion, which means the medication is given directly into a vein. The trial will be conducted in two parts. In the first part, researchers will focus on understanding how safe the treatment is and determining the best dose to use. In the second part, they will look at how well the treatment works in controlling the cancer. Throughout the study, participants will be closely monitored for any side effects and to see how their cancer responds to the treatment.

In addition to patritumab deruxtecan, other medications may be used to help manage symptoms or side effects. These include dexamethasone, which is a type of steroid, and other medications that help prevent nausea and vomiting. The study aims to provide valuable information on the potential benefits and risks of using patritumab deruxtecan in treating these challenging pediatric cancers.

The research process

The trial runs in 6 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, participants will be introduced to the clinical trial process. This includes understanding the purpose of the study, which is to evaluate the safety and effectiveness of patritumab deruxtecan in children with certain types of cancer, such as hepatoblastoma and rhabdomyosarcoma.

  2. Step 2

    Initial assessment

    Participants will undergo an initial assessment to confirm eligibility. This involves reviewing medical history and ensuring recovery from any previous treatments. Participants must have specific types of cancer and have tried other treatments without success.

  3. Step 3

    Treatment administration

    The treatment involves receiving patritumab deruxtecan through an intravenous infusion. The dosage and frequency will be determined by the study team based on individual needs and responses. The treatment aims to assess safety and preliminary effectiveness.

  4. Step 4

    Monitoring and follow-up

    Participants will be closely monitored for any side effects or adverse reactions. Regular follow-up visits will be scheduled to assess the treatment's impact on the cancer and overall health. Blood tests and other assessments may be conducted to measure the drug's presence in the body.

  5. Step 5

    Evaluation of results

    The study will evaluate the treatment's effectiveness by measuring the response rate of the cancer to the drug. This includes assessing any reduction in tumor size and overall disease control.

  6. Step 6

    Completion of study

    Upon completion of the study, participants will have a final assessment to determine the overall impact of the treatment. Long-term follow-up may be recommended to monitor health outcomes.

Who can join the trial?

5 criteria

  • The patient must have one of the following confirmed advanced or metastatic solid tumors: Rhabdomyosarcoma or Hepatoblastoma.
  • The patient must have experienced disease progression after at least one prior treatment for Rhabdomyosarcoma or Hepatoblastoma and have no satisfactory alternative treatment options available.
  • If the patient has side effects from previous cancer treatments, they must have recovered to a mild level (Grade 1) or to their original state. Patients with hormone-related side effects that are well-managed with hormone replacement or those with moderate nerve damage (Grade 2) are eligible.
  • Patients who test positive for Hepatitis B can participate if they have received antiviral therapy and have an undetectable viral load.
  • Patients with a history of Hepatitis C infection can participate if their viral load is undetectable.

Who cannot join the trial?

9 criteria

  • Patients with other types of cancer not specified in the study.
  • Patients who have had a severe allergic reaction to similar medications in the past.
  • Patients with uncontrolled or serious medical conditions that could interfere with the study.
  • Patients who are pregnant or breastfeeding.
  • Patients who are participating in another clinical trial at the same time.
  • Patients who have not recovered from major surgery or a significant injury.
  • Patients with active infections that require treatment.
  • Patients with a history of drug or alcohol abuse that could affect their ability to participate in the study.
  • Patients who are unable to comply with the study procedures or follow-up visits.
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Investigated drugs

Patritumab Deruxtecan is a medication being studied for its potential to treat certain types of cancer. It is designed to target and attach to a specific protein found on the surface of some cancer cells. Once attached, it delivers a powerful anti-cancer agent directly into the cancer cell, which may help to stop the cancer from growing and spreading. This medication is being tested to see if it is safe and effective for children with solid tumors that have come back after treatment or have not responded to other treatments.

What is already known about the treatment

Patritumab Deruxtecan – This medication is administered through an intravenous infusion, meaning it is given directly into the bloodstream through a vein. It is currently being studied in clinical trials for its potential use in treating certain types of cancer, specifically hepatoblastoma and rhabdomyosarcoma, which are types of solid tumors. The main purpose of this medication is to help fight cancer by targeting and binding to a specific protein on cancer cells, known as HER3, and delivering a toxic substance to kill these cells. It belongs to a class of drugs called antibody-drug conjugates, which are designed to specifically target cancer cells while minimizing damage to normal cells.

Investigated diseases

  • Hepatoblastoma

    Hepatoblastoma is a rare malignant liver tumor that primarily affects children, usually under the age of three. It originates from immature liver precursor cells and can grow rapidly. The disease often presents as an abdominal mass, and symptoms may include abdominal pain, swelling, and weight loss. As the tumor progresses, it can invade nearby structures and spread to other parts of the body, such as the lungs. The growth of the tumor can lead to liver dysfunction and other systemic effects. Early detection is crucial for managing the disease effectively.

  • Rhabdomyosarcoma

    Rhabdomyosarcoma is a rare type of cancer that forms in soft tissue, such as muscles, and is most common in children. It arises from cells that normally develop into skeletal muscles. The disease can occur in various parts of the body, including the head, neck, bladder, and extremities. Symptoms depend on the tumor's location and may include a noticeable lump, pain, or functional impairment of the affected area. As the tumor grows, it can invade surrounding tissues and metastasize to distant sites, such as the lungs or bone marrow. The progression of rhabdomyosarcoma can lead to significant physical and systemic challenges.

Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase ITrial ID2024-518771-66-00Protocol codeMK-9999-01CEstimated enrolment77 patientsSponsorMerck Sharp & Dohme LLC

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