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Rhabdomyosarcoma – Trials in Disease

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Ongoing Clinical Trials for Rhabdomyosarcoma

This article provides an overview of 10 ongoing clinical trials investigating new treatment approaches for rhabdomyosarcoma, a type of cancer that develops in muscle tissue. These trials are being conducted across multiple countries in Europe and involve various investigational drugs and treatment combinations aimed at improving outcomes for patients with newly diagnosed, relapsed, or treatment-resistant disease.

Clinical trial locations

Study of Crizotinib and Temsirolimus for Children with ALK, ROS1, or MET Positive Cancers, Including Neuroblastoma and Rhabdomyosarcoma

This trial is investigating treatment options for children with specific types of cancer that test positive for ALK, ROS1, or MET genes. The study focuses on patients between 1 and 21 years old who have tumors that have returned after treatment or not responded to standard therapies.

Main inclusion criteria: Participants must have a confirmed diagnosis of specific cancers including neuroblastoma, rhabdomyosarcoma, or other ALK, ROS1, or MET positive malignancies. Patients must be between 1 and 21 years old with a performance status score above 60 percent. They must have measurable disease and life expectancy of at least 12 weeks. Patients must not have received previous treatments targeting ALK, ROS1, or MET genes.

Main exclusion criteria: Patients with cancer types not listed in the study or those outside the specified age range cannot participate. Those unable to follow study procedures, with interfering medical conditions, who are pregnant or breastfeeding, or currently participating in another clinical trial are excluded. Patients who have had recent surgery, have allergies to study medications, certain heart conditions, uncontrolled infections, or history of drug or alcohol abuse are also excluded.

Focus of the trial: The study tests two medications: crizotinib, which can be used alone or combined with temsirolimus. Crizotinib is a targeted therapy available in capsules and oral solutions. The main goal is to determine the best dose of crizotinib when combined with temsirolimus and assess the safety and initial effectiveness of crizotinib on its own. The treatment is given in cycles with regular monitoring through check-ups and tests to track tumor response and side effects.

Investigational drugs: Crizotinib targets specific proteins in cancer cells to stop their growth and spread. Temsirolimus blocks a protein that helps cancer cells grow, potentially slowing cancer progression. The study aims to determine the optimal dosage for this combination and evaluate safety and effectiveness.

Study of drug combinations including irinotecan, ifosfamide, vincristine, dactinomycin, regorafenib and other agents for children and adults with rhabdomyosarcoma

This large international trial is studying various chemotherapy combinations specifically for patients with rhabdomyosarcoma. The study includes both newly diagnosed patients and those whose disease has returned or not responded to treatment.

Main inclusion criteria: Patients must have confirmed rhabdomyosarcoma diagnosis (excluding pleomorphic type). Age requirements vary by study group, with most requiring participants to be at least 6 months old, though some groups accept patients up to 25 years. Participants must have good liver function with normal or slightly elevated bilirubin and adequate blood counts. Good kidney function and agreement to use birth control during treatment are also required.

Main exclusion criteria: Patients without confirmed rhabdomyosarcoma diagnosis or those outside the age range are excluded. Those with severe heart, kidney, or liver problems that would make treatment unsafe cannot participate. Pregnant or breastfeeding women, those who received other cancer treatment within 30 days, patients with serious medical or psychological conditions, those with allergies to study medications, active uncontrolled infections, or inability to undergo required imaging are excluded.

Focus of the trial: The study tests multiple chemotherapy medications including irinotecan, ifosfamide, vincristine, actinomycin D, vinorelbine, cyclophosphamide, regorafenib, and temozolomide in different combinations. Treatment plans vary based on whether the disease is newly diagnosed or relapsed. For newly diagnosed patients, treatment may include ifosfamide, vincristine, and actinomycin D or a combination adding irinotecan. Some patients may receive radiation therapy depending on age and disease status. Maintenance therapy with vinorelbine and cyclophosphamide follows initial treatment.

Investigational drugs: The trial uses eight different chemotherapy medications. Irinotecan interferes with cancer cell DNA replication, while ifosfamide and cyclophosphamide damage cancer cell DNA. Vincristine and vinorelbine prevent cancer cell division by disrupting microtubules. Actinomycin D intercalates with DNA to prevent RNA synthesis. Regorafenib blocks proteins that promote cancer growth, and temozolomide damages cancer cell DNA.

Study of Trabectedin alone versus Trabectedin with tTF-NGR combination therapy in adults with metastatic or refractory soft tissue sarcoma who failed first-line treatment

This trial compares two treatment approaches for adults with soft tissue sarcoma that has spread or not responded to first-line treatment. One group receives trabectedin alone, while another receives trabectedin combined with an experimental drug called tTF-NGR.

Main inclusion criteria: Participants must be between 18 and 75 years old with advanced or metastatic soft-tissue sarcoma that either did not respond to anthracycline medications or cannot take them due to medical reasons. Patients must have confirmed high-grade soft tissue sarcoma (grade 2-3) of specific types including various liposarcomas, fibrosarcoma, leiomyosarcoma, rhabdomyosarcoma, angiosarcoma, synovial sarcoma, or undifferentiated sarcoma. Tumors must test positive for CD13 with a score of 1 or higher. Patients must have at least one measurable tumor and life expectancy of at least 3 months.

Main exclusion criteria: Patients outside the 18-75 age range or without confirmed soft-tissue sarcoma diagnosis are excluded. Those who have not received anthracycline therapy or who test negative for CD13 cannot participate. Women who are pregnant or breastfeeding, those without measurable disease, patients with severe heart, liver, or kidney problems, active uncontrolled infections, or other active cancers are excluded. Those with known allergies to study medications or unable to provide informed consent are also ineligible.

Focus of the trial: The study randomly assigns participants to receive either trabectedin alone or trabectedin plus tTF-NGR. Both medications are given through intravenous infusion. The treatment continues until disease progression or other stopping criteria are met. Regular imaging tests track tumor response, and overall health status is monitored throughout. The primary goal is to determine if adding tTF-NGR helps keep cancer under control for longer periods.

Investigational drugs: Trabectedin is a cancer medication that interferes with cancer cell growth and division. It is typically used when anthracycline-based treatments have failed. tTF-NGR is an experimental protein designed to target blood vessels in tumors and help trap trabectedin inside the tumor, making the treatment more concentrated and potentially more effective.

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

This trial evaluates a new treatment called patritumab deruxtecan for children with hepatoblastoma or rhabdomyosarcoma that has returned or not responded to previous treatments. The study is conducted in two parts, first determining safety and appropriate dosing, then assessing effectiveness.

Main inclusion criteria: Patients must have confirmed advanced or metastatic hepatoblastoma or rhabdomyosarcoma. The disease must have progressed after at least one prior treatment with no satisfactory alternative treatment options available. Patients must have recovered from previous treatment side effects to mild level or original state. Those with hormone-related side effects that are well-managed or moderate nerve damage may participate. Patients with history of Hepatitis B or C can participate under certain conditions.

Main exclusion criteria: Patients with cancer types not specified in the study or those with severe allergic reactions to similar medications cannot participate. Those with uncontrolled or serious medical conditions, pregnant or breastfeeding women, and those participating in another trial simultaneously are excluded. Patients who have not recovered from major surgery, have active infections requiring treatment, history of drug or alcohol abuse affecting study participation, or inability to comply with procedures are ineligible.

Focus of the trial: The treatment involves administering patritumab deruxtecan through intravenous infusion. The first stage determines the safe dose and monitors for side effects. The second stage focuses on how well the treatment works in controlling cancer. Patients are closely monitored for side effects and tumor response through regular assessments including blood tests and imaging scans.

Investigational drugs: Patritumab deruxtecan is designed to target and attach to a specific protein (HER3) found on cancer cells. Once attached, it delivers a powerful anti-cancer agent directly into the cancer cell, potentially stopping cancer growth and spread. This medication belongs to a class of drugs called antibody-drug conjugates.

Study of Trabectedin and Low-Dose Radiation Therapy for Adults and Young Adults with Advanced or Metastatic Soft Tissue and Bone Sarcomas

This study combines trabectedin chemotherapy with low-dose radiation therapy for patients with advanced sarcomas, including soft tissue sarcomas, bone tumors, and small round-cell sarcomas. The trial focuses on patients whose cancer has progressed despite previous treatments.

Main inclusion criteria: Patients must be between 16 and 75 years old with advanced or metastatic sarcoma that cannot be removed by surgery. Eligible tumor types include soft tissue sarcoma, bone tumors like osteosarcoma and chondrosarcoma, and small round-cell sarcomas including Ewing’s sarcoma and rhabdomyosarcoma. Patients must have performance status of 1 or less and measurable disease. They must have received at least one but no more than three previous treatments for advanced cancer. The disease must have worsened in the last 6 months.

Main exclusion criteria: Patients outside the age range or without measurable disease cannot participate. Those without confirmed diagnosis of eligible sarcoma types are excluded. Patients who are not adults or young adults as defined by the study or who belong to vulnerable populations requiring special protection are ineligible.

Focus of the trial: The treatment combines trabectedin administered through intravenous infusion with low-dose radiation therapy applied to specific tumor sites. Radiation doses vary by location: 45 Gy for extremities and 30 Gy for non-extremity areas. The study monitors tumor response, particularly in areas receiving radiation, and tracks how long the treatment remains effective. Regular assessments measure changes in tumor size, pain levels, and overall well-being.

Investigational drugs: Trabectedin interferes with cancer cell growth, slowing their spread in the body. It is used when other treatments have not been effective. Low-dose radiation therapy uses high-energy rays to target and destroy cancer cells while minimizing damage to surrounding healthy tissues.

Study to Find the Right Dose and Safety of Lutetium (177Lu) Edotreotide and Arginine-Lysine in Children with Somatostatin Receptor-Positive Tumors

This trial studies a targeted radiopharmaceutical therapy called lutetium Lu 177 edotreotide in children with tumors that test positive for somatostatin receptors. The study aims to determine the appropriate dose based on safety and how the body processes the treatment.

Main inclusion criteria: Children must be at least 2 years old and younger than 18 years with confirmed diagnosis of somatostatin receptor-positive tumors including neuroendocrine tumors, central nervous system tumors, or lymphoma. The tumor must have returned or not responded to at least one previous treatment. Patients must have Karnofsky score of 50 percent or higher (for those older than 16) or Lansky score of 50 percent or higher (for those 16 or younger). Life expectancy must be at least 12 weeks. Patients must have measurable disease and positive SSTR expression confirmed by immunohistochemistry.

Main exclusion criteria: Patients younger than two years old or with tumor types not SSTR-positive are excluded. Those who have not tried at least one other treatment before or who are not healthy enough to receive the study treatment cannot participate. Pregnant or breastfeeding patients, those with other medical conditions interfering with treatment, or those unable to follow study procedures are ineligible.

Focus of the trial: The treatment involves administering lutetium Lu 177 edotreotide through intravenous infusion along with an arginine-lysine solution to protect the kidneys. Regular monitoring assesses treatment response through measuring tumor size and evaluating overall health. The study aims to find the highest safe and effective dose with minimal side effects.

Investigational drugs: Lutetium Lu 177 edotreotide is a targeted therapy that delivers radiation directly to tumor cells expressing somatostatin receptors. This approach helps destroy cancer cells while minimizing damage to surrounding healthy tissue. The arginine-lysine solution provides kidney protection during treatment.

Study of Cobolimab and Dostarlimab for Children and Young Adults with Newly Diagnosed or Relapsed/Refractory Tumors

This trial studies two immunotherapy medications, cobolimab and dostarlimab, used together in children and young adults with newly diagnosed or relapsed tumors. The study is conducted in two parts, first assessing safety and determining appropriate dosage, then evaluating effectiveness in specific cancer types.

Main inclusion criteria: Participants must be within specific age ranges at time of consent, generally between 2 and 21 years depending on the study cohort. They must have advanced or metastatic solid tumors that have worsened after treatment with available therapies and have limited treatment options. Patients must have at least one measurable tumor lesion. Performance status must be 60 percent or higher on appropriate scales. Adequate organ function as shown by specific blood test results is required.

Main exclusion criteria: Patients who have not recovered from previous treatments or surgeries are excluded. Those with other serious uncontrolled illnesses, pregnant or breastfeeding women, and those with another cancer within the last 5 years (with certain exceptions) cannot participate. Patients who have had organ transplants, known allergies to study drugs, are taking other experimental drugs, have active infections requiring treatment, certain heart or lung conditions, or history of autoimmune diseases are ineligible.

Focus of the trial: The study evaluates cobolimab and dostarlimab administered together through intravenous infusion. The first part involves safety assessment and dose determination in patients with advanced solid tumors. The second part focuses on specific cancer types including melanoma and Hodgkin lymphoma to evaluate treatment effectiveness. Throughout the study, participants undergo regular monitoring including physical exams, laboratory tests, and imaging studies.

Investigational drugs: Cobolimab and dostarlimab are immunotherapy agents designed to help the immune system recognize and attack cancer cells more effectively. Cobolimab targets specific proteins to activate immune response, while dostarlimab blocks proteins that cancer cells use to evade the immune system.

Study of Lenvatinib for Children and Young Adults with Relapsed or Refractory Solid Tumors, Including High Grade Glioma and Rhabdomyosarcoma

This trial evaluates lenvatinib in young patients with solid tumors that have returned or are not responding to treatment. The study focuses on several cancer types including high grade glioma, rhabdomyosarcoma, and Ewing sarcoma, but excludes osteosarcoma.

Main inclusion criteria: Participants must be between 2 and 21 years old with high grade glioma, rhabdomyosarcoma, Ewing sarcoma, or other solid tumors (except osteosarcoma). The cancer must have returned or not responded to treatment, confirmed by tissue examination. Disease must be measurable using specific medical criteria. Participants must have appropriate activity level scores (Lansky Play Score for those 16 and under, Karnofsky Performance Status for those over 16). Brain-related symptoms must be stable for at least 7 days. Organs must function adequately, blood pressure must be controlled, and patients must have recovered from previous treatment side effects.

Main exclusion criteria: Patients outside the 2-21 age range or without one of the specified cancer types are excluded. Those with osteosarcoma cannot participate.

Focus of the trial: The treatment involves taking lenvatinib capsules by mouth. The study monitors tumor response over time, specifically looking at changes in tumor size and side effects. Regular check-ups and assessments track progress using specific criteria to evaluate tumor response. The primary goal is to determine effectiveness in shrinking tumors and improving patient condition.

Investigational drugs: Lenvatinib is a tyrosine kinase inhibitor that blocks certain proteins involved in tumor growth and blood vessel formation. By inhibiting these proteins, it aims to slow or stop cancer progression.

Study of Nivolumab with Chemotherapy for Children and Teenagers with Refractory or Relapsing Solid Tumors or Lymphoma

This trial explores combining nivolumab, an immunotherapy medication, with different metronomic chemotherapy regimens for children and teenagers with solid tumors that have not responded to treatment or have returned. The study tests three different chemotherapy combinations.

Main inclusion criteria: Patients must be less than 18 years old (with some exceptions) with confirmed solid malignant tumor or lymphoma that is progressive or refractory despite standard therapy. Disease must be measurable or evaluable by imaging. Performance status must be 70 percent or higher. Life expectancy must be at least 3 months. Patients must have adequate organ function including blood, heart, kidney, and liver function. Those who are sexually active must agree to use birth control. Females who can have children must have negative pregnancy test within 7 days before starting treatment.

Main exclusion criteria: Patients without progressive or refractory pediatric solid tumor are excluded. Those outside the age range, unable to follow procedures or take medications as required, with interfering medical conditions, who are pregnant or breastfeeding, or received certain interfering treatments cannot participate. Those with severe allergic reactions to study drugs, active infections, or other serious illness are ineligible.

Focus of the trial: The first stage determines which of three chemotherapy regimens is safe with nivolumab: cyclophosphamide and vinblastine; capecitabine alone; or cyclophosphamide, vinblastine, and capecitabine combined. The second stage focuses on the most promising regimen with or without nivolumab to prevent cancer worsening. Medications are given through intravenous or oral routes with regular monitoring.

Investigational drugs: Nivolumab is an immune checkpoint inhibitor helping the immune system recognize and attack cancer cells by blocking the PD-1 protein. Cyclophosphamide is an alkylating agent that damages cancer cell DNA. Vinblastine disrupts cell division by interfering with microtubules. Capecitabine is converted to 5-fluorouracil in the body, interfering with DNA and RNA of cancer cells.

Study on Ribociclib, Topotecan, and Temozolomide for Children and Young Adults (12 months – 21 years) with Relapsed or Refractory Neuroblastoma and Solid Tumors

This trial studies a combination of ribociclib, temozolomide, and topotecan for treating relapsed or refractory neuroblastoma and other solid tumors in children and young adults. The study is divided into two phases: determining the best dose of ribociclib and evaluating treatment effectiveness.

Main inclusion criteria: Participants must be between 12 months and 21 years old with specific solid tumors including neuroblastoma, medulloblastoma, high-grade glioma, malignant rhabdoid tumor, or rhabdomyosarcoma that have not responded to standard treatments. Those with central nervous system disease taking corticosteroids must be on stable doses for at least 7 days. Disease must be measurable according to specific criteria. Patients must be able to sit upright in wheelchair and have life expectancy of at least 12 weeks. Adequate bone marrow and organ function is required.

Main exclusion criteria: Patients who have not recovered from previous treatments or surgeries are excluded. Those with other serious health conditions interfering with the study, pregnant or breastfeeding women, those unable to follow procedures or attend visits, recent participation in another trial, allergies to study medications, uncontrolled infections, certain heart problems, or history of drug or alcohol abuse are ineligible.

Focus of the trial: Phase I determines the maximum tolerated dose of ribociclib when combined with temozolomide and topotecan, with medications administered orally and intravenously in 28-day cycles. Phase II evaluates the treatment’s effectiveness compared to placebo, focusing on overall response rate, progression-free survival, and duration of response. Regular assessments monitor patient response and health throughout the trial.

Investigational drugs: Ribociclib blocks proteins that cancer cells need to grow and divide. Topotecan interferes with cancer cell DNA, preventing growth and multiplication. Temozolomide damages cancer cell DNA, stopping their growth and spread. The combination aims to improve treatment outcomes for children with these challenging cancers.

Summary

These 10 ongoing clinical trials represent a diverse range of research efforts aimed at improving treatment options for rhabdomyosarcoma. Several notable patterns emerge from this overview.

The trials show strong geographic concentration in Western European countries, particularly France, which participates in 8 of the 10 studies, followed by Spain, Italy, and Germany with 6-7 trials each. This concentration suggests these countries have established pediatric oncology research networks and infrastructure supporting complex clinical trials.

A clear trend toward combination therapies is evident, with most trials testing multiple drugs together rather than single agents. Several studies explore combinations of traditional chemotherapy with newer targeted therapies or immunotherapies, reflecting current understanding that multi-pronged approaches may be more effective against aggressive cancers.

The trials predominantly focus on pediatric and young adult populations, typically ranging from infants to patients in their early twenties. This reflects the fact that rhabdomyosarcoma primarily affects children and adolescents. Most studies specifically target patients whose disease has relapsed or proven resistant to standard treatments, addressing an urgent medical need for these difficult-to-treat cases.

Several medications appear across multiple trials, including irinotecan, topotecan, temozolomide, and vincristine, suggesting these drugs are considered promising building blocks for new treatment regimens. The studies also investigate innovative approaches such as targeted radiopharmaceutical therapy and immunotherapy combinations that were not previously available for pediatric sarcomas.

These trials collectively offer patients across Europe access to cutting-edge treatment options and contribute to advancing medical knowledge about rhabdomyosarcoma treatment. Patients interested in participating should discuss eligibility with their healthcare team and consider factors such as trial location, treatment approach, and specific inclusion criteria when evaluating options.

Ongoing Clinical Trials on Rhabdomyosarcoma

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

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia Denmark France Germany Greece +6

  • Study to Find the Right Dose and Safety of Lutetium (177Lu) Edotreotide and Arginine-Lysine in Children with Somatostatin Receptor-Positive Tumors

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    France Italy Spain

  • Study of Trabectedin alone versus Trabectedin with tTF-NGR combination therapy in adults with metastatic or refractory soft tissue sarcoma who failed first-line treatment

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

  • Study of drug combinations including irinotecan, ifosfamide, vincristine, dactinomycin, regorafenib and other agents for children and adults with rhabdomyosarcoma

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia Denmark Finland France +10

  • Study of Crizotinib and Temsirolimus for Children with ALK, ROS1, or MET Positive Cancers, Including Neuroblastoma and Rhabdomyosarcoma

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Denmark Finland France Germany Italy The Netherlands +3

  • Study of Trabectedin and Low-Dose Radiation Therapy for Adults and Young Adults with Advanced or Metastatic Soft Tissue and Bone Sarcomas

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Spain

  • Indocyanine Green and Near-Infrared Fluorescence for Lymph Node Removal in Children with Renal Tumours or Paratesticular Rhabdomyosarcoma

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

  • Study on Ribociclib, Topotecan, and Temozolomide for Children and Young Adults (12 months – 21 years) with Relapsed or Refractory Neuroblastoma and Solid Tumors

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Denmark France Germany Hungary Italy +1

  • Study of Nivolumab with Chemotherapy for Children and Teenagers with Refractory or Relapsing Solid Tumors or Lymphoma

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study of Cobolimab and Dostarlimab for Children and Young Adults with Newly Diagnosed or Relapsed/Refractory Tumors

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia Denmark France Germany Italy Spain

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