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Study on Atypical Teratoid/Rhabdoid Tumors in Children Using Dactinomycin, Carboplatin, and Cyclophosphamide

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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 a rare type of brain tumor in children known as atypical teratoid/rhabdoid tumors (ATRT). The study aims to evaluate the effectiveness of different treatment approaches for children with ATRT. The treatments being tested include high-dose chemotherapy (HDCT) and focal radiotherapy, which are used as consolidation therapies. Consolidation therapy is a treatment given after the initial therapy to strengthen its effects and help prevent the cancer from returning.

The trial involves several medications, including dactinomycin, carboplatin, cyclophosphamide, ifosfamide, etoposide, vincristine sulfate, thiotepa, doxorubicin hydrochloride, and methotrexate. These medications are administered through intravenous use, meaning they are given directly into a vein. The study is designed to compare the outcomes of children receiving these treatments to those who receive standard care, as well as to historical data from previous patients.

The purpose of the study is to determine if the new treatment approaches are as effective as, or better than, the current standard treatments. The trial will follow participants over a period of time to assess their overall survival and other health outcomes. This research is important for improving treatment options and outcomes for children diagnosed with ATRT.

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 a review of medical history and a series of tests such as MRI and CSF examination to evaluate the condition of the atypical teratoid/rhabdoid tumors (ATRT).

  2. Step 2

    Induction chemotherapy

    The first phase involves receiving three courses of induction chemotherapy. This treatment uses a combination of medications administered through intravenous infusion or injection. The specific drugs include dactinomycin, carboplatin, cyclophosphamide, ifosfamide, etoposide, vincristine sulfate, thiotepa, doxorubicin hydrochloride, and methotrexate.

    The purpose of this phase is to reduce the size of the tumors and assess the body's response to the treatment.

  3. Step 3

    Evaluation and review

    After completing the induction chemotherapy, another evaluation is conducted. This includes MRI and CSF examinations to determine the effectiveness of the treatment and to decide the next steps in the trial.

  4. Step 4

    Consolidation therapy

    Depending on the age and response to the initial treatment, the next phase involves consolidation therapy. This may include high-dose chemotherapy (HDCT) or focal radiotherapy (RT).

    For children aged 12-35 months, the trial evaluates the effectiveness of three courses of HDCT compared to RT. For children under 12 months, HDCT is used as a consolidation measure. For those aged 36 months or older, RT combined with conventional chemotherapy is assessed.

  5. Step 5

    Follow-up and monitoring

    Throughout the trial, regular follow-up appointments are scheduled to monitor health and response to treatment. This includes assessments of overall survival, neurocognitive outcomes, quality of life, and any adverse effects.

    These follow-ups occur at various intervals, including 2 and 5 years after the initial diagnosis, to ensure long-term health and well-being.

Who can join the trial?

32 criteria

  • Age at diagnosis must be less than 18 years.
  • For Part A: Must have an MRI and CSF (cerebrospinal fluid) examination after 3 courses of chemotherapy showing stable disease or better.
  • For Part A: Blood tests must show ALT or AST levels less than or equal to 3 times the upper limit of normal (ULN), and bilirubin levels less than or equal to 1.5 times the ULN.
  • For Part A: Creatinine levels must be less than or equal to 1.5 times the ULN, and kidney function (GFR) must be within normal age-related values.
  • For Part A: Heart function tests must show an ejection fraction (EF) of 50% or more, or a fractional shortening (FS) of 29% or more.
  • For Part B: Must be enrolled in the umbrella trial.
  • For Part B: Must have received 3 courses of induction chemotherapy according to the protocol.
  • For Part B: Radiotherapy is not allowed (for example, if under 12 months old or other reasons).
  • For Part B: Not eligible for the randomized trial in Part A (for example, if randomization is refused).
  • For Part B: Must have written informed consent or assent according to national laws.
  • For Part B: Central review must confirm the diagnosis of ATRT.
  • Pathology must be compatible with ATRT, and there must be a confirmed loss or deficiency of INI1, SMARCB1, or SMARCA4 by a local pathology lab.
  • For Part B: MRI and CSF examination after 3 courses of chemotherapy must show significant sensitivity to chemotherapy.
  • For Part B: Blood tests must show ALT or AST levels less than or equal to 3 times the ULN, and bilirubin levels less than or equal to 1.5 times the ULN.
  • For Part B: Creatinine levels must be less than or equal to 1.5 times the ULN, and kidney function (GFR) must be within normal age-related values.
  • For Part B: Heart function tests must show an ejection fraction (EF) of 50% or more, or a fractional shortening (FS) of 29% or more.
  • For Part C: Must be enrolled in the umbrella trial.
  • For Part C: Must have received 3 courses of induction chemotherapy according to the protocol.
  • For Part C: Must be aged 36 months or older, or high-dose chemotherapy (HDCT) is not possible, or not eligible for the randomized trial in Part A.
  • For Part C: Must have written informed consent or assent according to national laws.
  • For Part C: Central review must confirm the diagnosis of ATRT.
  • For Part C: MRI and CSF examination after 3 courses of chemotherapy must show stable disease or better.
  • Must have written informed consent or assent for study participation according to national laws.
  • For Part C: Blood tests must show ALT or AST levels less than or equal to 3 times the ULN, and bilirubin levels less than or equal to 1.5 times the ULN.
  • For Part C: Creatinine levels must be less than or equal to 1.5 times the ULN, and kidney function (GFR) must be within normal age-related values.
  • For Part C: Heart function tests must show an ejection fraction (EF) of 50% or more, or a fractional shortening (FS) of 29% or more.
  • Patient agrees to use effective contraception while on treatment.
  • For Part A: Must be enrolled in the umbrella trial.
  • For Part A: Must have received 3 courses of induction chemotherapy according to the protocol and show stable disease or better after induction.
  • For Part A: Expected age must be between 12-35 months at the time of consolidation therapy (radiotherapy or high-dose chemotherapy).
  • For Part A: Must have written informed consent or assent for randomization according to national laws.
  • For Part A: Central review must confirm the diagnosis of ATRT.

Who cannot join the trial?

5 criteria

  • Patients who are not diagnosed with atypical teratoid/rhabdoid tumors (ATRT). ATRT is a rare and fast-growing type of brain tumor.
  • Children who are not within the specified age range for the study. The study is for children aged 12 to 35 months for Part A, and children under 12 months for Part B.
  • Children who are 36 months or older for Part C.
  • Patients who are not eligible for randomization within Part A of the study protocol.
  • Patients who are not suitable for the specific treatment plans outlined in the study, such as high-dose chemotherapy (HDCT) or radiation therapy (RT).
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Investigated drugs

  • High-Dose Chemotherapy (HDCT)

    is a treatment that uses very high doses of powerful drugs to kill cancer cells. In this trial, it is used as a consolidation therapy, which means it is given after the initial treatment to help eliminate any remaining cancer cells and reduce the risk of the cancer coming back. The goal is to improve the chances of survival for children with atypical teratoid/rhabdoid tumors (ATRT).

  • Focal Radiotherapy (RT)

    is a treatment that uses high-energy rays to target and destroy cancer cells in a specific area of the body. In this trial, it is used as a consolidation therapy, similar to high-dose chemotherapy, to help ensure that any remaining cancer cells are destroyed after the initial treatment. This therapy is focused on a particular part of the body where the tumor was located.

  • Conventional Chemotherapy

    involves the use of standard cancer-fighting drugs that are given to patients to kill cancer cells or stop them from growing. In this trial, conventional chemotherapy is used alongside other treatments to help improve the overall effectiveness of the therapy and increase the chances of survival for children with ATRT.

What is already known about the treatment

High-Dose Chemotherapy (HDCT) – This medication is administered intravenously, meaning it is given through a vein, usually in a hospital setting. It is currently being studied in clinical trials for its effectiveness in treating atypical teratoid/rhabdoid tumors (ATRT) in children. The main therapeutic use of HDCT is to kill cancer cells by damaging their DNA, which prevents them from growing and dividing. It belongs to a class of drugs known as chemotherapy agents, which are used to treat various types of cancer by targeting rapidly dividing cells.

Investigated diseases

Atypical Teratoid/Rhabdoid Tumors (ATRT) – Atypical Teratoid/Rhabdoid Tumors are rare, fast-growing tumors that typically occur in the brain and spinal cord. They are most commonly diagnosed in young children. These tumors are characterized by their aggressive nature and can spread quickly within the central nervous system. ATRT is composed of a mix of different cell types, which can make it challenging to study. The progression of ATRT involves rapid growth and potential spread to other areas of the brain or spine. Symptoms often depend on the tumor's location and may include headaches, nausea, and changes in behavior or motor skills.
Trial detailsLast updated 2 Oct 2026
Age0-17PhasePhase IIITrial ID2022-501456-28-00Protocol codeSIOPEATRT01Estimated enrolment308 patientsSponsorGPOH gGmbH

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).