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

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What is this study about?

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.

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 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 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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Institut Gustave Roussy Villejuif France
Centre Hospitalier Universitaire De Bordeaux Bordeaux France
Hospital Universitario Y Politecnico La Fe Valencia Spain
Institut Curie – Site Paris Paris France

Other Sites

Site Name City Country Status
Hospital Infantil Universitario Nino Jesus Madrid Spain
Universitair Ziekenhuis Gent Gent Belgium
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Nosokomeio Paidon I Agia Sofia Athens Greece
Narodny Ustav Detskych Chorob Bratislava Slovakia
Universitaetsklinikum Tuebingen AöR Tuebingen Germany
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Brno Brno Czechia
Semmelweis University Budapest Hungary
Prinses Maxima Centrum voor Kinderoncologie B.V. Utrecht The Netherlands
Justus-Liebig-Universitaet Giessen Giessen Germany
Centre Hospitalier Universitaire De Nantes Nantes France
Rigshospitalet Copenhagen Denmark
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Cbxfee Lqns Btkrqn Lyon France
Uczlrioyla Hhtpfwxh Cazahlt Cologne Germany
Fkfebwgb nokcivxkl Mbwkp a Hydymky Prague Czechia
Aecfqvcwue Plagbfwp Hofacihn Di Meinqkrbx Marseille France
Axjugdh Onignufrngb Unlbdwkxwwkbs Cwzbmaudtgmy Duhov Sbiadj E Dygsq Sxnpldj Dx Tldfjr Turin Italy
Uthmptzybfmutztyocwkg Mlsyhbzt Ady Munster Germany
Hjvuyaas Vlwc dqqzzamh Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Recruiting
15.05.2025
Czechia Czechia
Recruiting
15.05.2025
Denmark Denmark
Recruiting
15.05.2025
France France
Recruiting
15.05.2025
Germany Germany
Recruiting
15.05.2025
Greece Greece
Recruiting
15.05.2025
Hungary Hungary
Recruiting
15.05.2025
Italy Italy
Recruiting
15.05.2025
Slovakia Slovakia
Recruiting
15.05.2025
Spain Spain
Recruiting
15.05.2025
Sweden Sweden
Recruiting
15.05.2025
The Netherlands The Netherlands
Recruiting
15.05.2025

Trial locations

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.

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 ID:
2024-518771-66-00
Protocol code:
MK-9999-01C
Trial Phase:
Human Pharmacology (Phase I) – Other

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