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

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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 studying Rhabdomyosarcoma, a type of cancer that develops in muscle tissue. The study will test several cancer medications including regorafenib, irinotecan, cyclophosphamide, doxorubicin, temozolomide, ifosfamide, vincristine, dactinomycin, and vinorelbine in different combinations.

The main purpose is to find better treatment options for both newly diagnosed and relapsed rhabdomyosarcoma in children and adults. The study will evaluate different timing and doses of chemotherapy and radiation therapy. Some patients will receive standard treatment while others will receive new combinations of medications.

The trial will last several years, with an estimated completion date in 2030. Throughout the study, researchers will monitor how well the treatments work by measuring survival rates and checking if the cancer returns. They will also track side effects and complications from the treatments.

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 diagnosis and qualification

    You must have a confirmed diagnosis of rhabdomyosarcoma (except pleomorphic type)

    A medical assessment will determine if you qualify as High Risk (HR) or Very High Risk (VHR) patient

  2. Step 2

    Frontline treatment - initial phase

    Treatment will begin within 60 days after diagnostic biopsy/surgery

    You will receive one of two medication combinations:

    Option 1: Standard therapy with ifosfamide, vincristine, and actinomycin D (IVA)

    Option 2: New combination adding irinotecan to the standard therapy (IrIVA)

  3. Step 3

    Radiotherapy options

    If you are 2 years or older, you may receive radiation therapy

    The timing of radiation treatment depends on your specific situation:

    It may be given before surgery (pre-operative)

    It may be given after surgery (post-operative)

    The radiation dose will be determined based on your individual risk factors

  4. Step 4

    Maintenance treatment

    After initial treatment, you will receive maintenance therapy with vinorelbine and cyclophosphamide

    For High Risk patients: 6 cycles of treatment, with possibility of 6 additional cycles

    For Very High Risk patients: 12 cycles of treatment, with possibility of 12 additional cycles

  5. Step 5

    Treatment for disease relapse

    If the disease returns, you may receive one of these combinations:

    Option 1: vincristine, irinotecan, and temozolomide (VIrT)

    Option 2: vincristine, irinotecan, and regorafenib (VIrR)

Who can join the trial?

15 criteria

  • Confirmed diagnosis of rhabdomyosarcoma (a type of muscle cancer), except for the pleomorphic type
  • Age requirements vary depending on study group:
    • At least 6 months old for most study groups
    • Over 12 months and up to 25 years for some treatments
    • At least 2 years old for radiation therapy
    • No previous cancer treatment except surgery (for newly diagnosed patients)
    • Good liver function:
      • Normal or slightly elevated bilirubin levels
      • Liver enzymes less than 2.5 times the normal limit
      • Adequate blood counts:
        • White blood cells (neutrophils) at least 1.0 x 10^9/L
        • Platelets at least 80 x 10^9/L
        • Good kidney function with adequate creatinine clearance
        • Negative pregnancy test for females who can become pregnant
        • Agreement to use birth control during treatment and:
          • 12 months after treatment for females
          • 6 months after treatment for males
          • Must be healthy enough to receive treatment
          • Written informed consent from patient and/or parent/legal guardian

Who cannot join the trial?

12 criteria

  • Patients who do not have confirmed diagnosis of Rhabdomyosarcoma (a type of soft tissue cancer)
  • Patients younger than 6 months or older than 21 years of age
  • Patients with severe heart problems that would make chemotherapy unsafe
  • Patients with severe kidney or liver dysfunction that would interfere with treatment
  • Patients who have received any other cancer treatment within the last 30 days
  • Pregnant or breastfeeding women
  • Patients who are unable to follow the study protocol due to serious medical or psychological conditions
  • Patients who have participated in another clinical trial within the past 30 days
  • Patients who are allergic to any of the study medications (irinotecan, ifosfamide, vincristine, actinomycin D, vinorelbine, cyclophosphamide, regorafenib, or temozolomide)
  • Patients with active, uncontrolled infections
  • Patients who cannot undergo required imaging procedures
  • Patients who do not meet the required blood test values for safe treatment
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Investigated drugs

  • Irinotecan

    A chemotherapy medication used to treat various types of cancer. In this trial, it is being tested in combination with other medications for treating rhabdomyosarcoma.

  • Ifosfamide

    A chemotherapy medication that works by stopping cancer cells from dividing and growing. It is used as part of combination therapy for treating rhabdomyosarcoma.

  • Vincristine

    A chemotherapy medication that prevents cancer cells from dividing. It is commonly used in combination with other medications to treat various types of cancer, including rhabdomyosarcoma.

  • Actinomycin D

    A chemotherapy medication that works by interfering with cancer cell DNA. It is used as part of combination therapy for treating rhabdomyosarcoma.

  • Vinorelbine

    A chemotherapy medication that works by stopping cancer cells from separating into new cells. In this trial, it is being tested as part of maintenance therapy.

  • Cyclophosphamide

    A chemotherapy medication that works by slowing or stopping cell growth. In this trial, it is being used as part of maintenance therapy and can be given orally.

What is already known about the treatment

Based on the clinical trial data, here are the medications mentioned and their descriptions: Irinotecan - A chemotherapy medication administered intravenously that belongs to the topoisomerase I inhibitor class, used in the treatment of rhabdomyosarcoma. It works by interfering with cancer cell DNA replication, preventing tumor growth, and is often combined with other chemotherapy agents for enhanced effectiveness. Ifosfamide - An intravenous chemotherapy drug from the alkylating agent class that is used to treat various types of cancers, including rhabdomyosarcoma. It works by damaging the DNA of cancer cells, preventing them from dividing and multiplying. Vincristine - A plant-derived chemotherapy medication administered intravenously that belongs to the vinca alkaloid class. It works by stopping cancer cell division by blocking the formation of microtubules, and is commonly used in combination therapy for rhabdomyosarcoma. Actinomycin D - An intravenous antibiotic with anti-cancer properties that intercalates with DNA to prevent RNA synthesis in cancer cells. It is commonly used in combination with other chemotherapy drugs to treat rhabdomyosarcoma. Vinorelbine - A chemotherapy medication given intravenously or orally that belongs to the vinca alkaloid family. It works by preventing cancer cell division through disruption of microtubules and is used in maintenance therapy for rhabdomyosarcoma. Cyclophosphamide - An oral or intravenous chemotherapy medication from the alkylating agent class that works by damaging cancer cell DNA. It is used in maintenance therapy for rhabdomyosarcoma and is often combined with other chemotherapy drugs. Regorafenib - An oral targeted therapy medication that belongs to the multikinase inhibitor class. It works by blocking several proteins that promote cancer growth and blood vessel formation, and is being studied in combination with other drugs for relapsed rhabdomyosarcoma. Temozolomide - An oral chemotherapy medication from the alkylating agent class that can cross the blood-brain barrier. It works by damaging cancer cell DNA and is used in combination therapy for relapsed rhabdomyosarcoma.

Investigated diseases

Rhabdomyosarcoma - A rare type of cancer that develops in soft tissue, specifically in muscle cells that are in the process of developing. It most commonly occurs in children and teens, forming in muscles that are attached to bones. The disease can start in various parts of the body, including the head and neck area, arms, legs, trunk, or in internal organs. As the tumor grows, it can cause swelling, pain, or changes in the affected area. The cancer cells can spread to other parts of the body through the blood or lymph system if not addressed.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-510579-40-00Protocol codeRG_17-247Estimated enrolment1 585 patientsSponsorThe University Of Birmingham

sourced from the EU Clinical Trials Register and site verification

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