Ongoing Clinical Trials for Atypical Teratoid/Rhabdoid Tumour of CNS
There are currently 2 clinical trials underway for atypical teratoid/rhabdoid tumour of the central nervous system (CNS). These trials are testing different combinations of chemotherapy drugs and treatment approaches for children diagnosed with this rare and aggressive brain tumor. The studies are being conducted across multiple European countries and aim to improve treatment outcomes and survival rates for young patients.
Clinical trial locations
- Austria
- Belgium
- Czechia
- Denmark
- Finland
- France
- Germany
- Hungary
- Italy
- Netherlands
- Norway
- Spain
- Sweden
Study on Atypical Teratoid/Rhabdoid Tumors in Children Using Dactinomycin, Carboplatin, and Cyclophosphamide
This trial is designed for children under 18 years of age who have been diagnosed with atypical teratoid/rhabdoid tumors. The study evaluates different treatment approaches depending on the child’s age and response to initial therapy.
Main inclusion criteria: Children must be under 18 years old at diagnosis. The diagnosis must be confirmed through central review showing loss or deficiency of specific proteins (INI1, SMARCB1, or SMARCA4). After receiving three courses of initial chemotherapy, participants must have stable disease or better as shown by MRI and cerebrospinal fluid examination. Blood tests must show adequate liver function (ALT or AST less than 3 times the normal limit), kidney function (creatinine less than 1.5 times normal, with age-appropriate kidney filtering rate), and heart function (ejection fraction of 50% or more). Written informed consent from parents or guardians is required.
Main exclusion criteria: Children who do not have a confirmed diagnosis of atypical teratoid/rhabdoid tumors cannot participate. Those who fall outside the specific age ranges for each part of the study (Part A: 12-35 months, Part B: under 12 months, Part C: 36 months or older) are excluded. Children who are not suitable for the specific treatment plans outlined in the study, such as high-dose chemotherapy or radiation therapy, cannot enroll.
Focus and goals: The trial aims to determine if new treatment approaches are as effective as or better than current standard treatments. It compares outcomes for children receiving high-dose chemotherapy versus focal radiotherapy as consolidation therapy, which is treatment given after initial therapy to strengthen its effects and prevent cancer from returning. The study follows participants over time to assess overall survival, neurocognitive outcomes, quality of life, and any adverse effects at various intervals including 2 and 5 years after diagnosis.
Investigational drugs: The trial uses multiple chemotherapy medications including dactinomycin, carboplatin, cyclophosphamide, ifosfamide, etoposide, vincristine sulfate, thiotepa, doxorubicin hydrochloride, and methotrexate. These are administered intravenously (directly into a vein) in three courses as induction chemotherapy. Following this, the study evaluates high-dose chemotherapy and focal radiotherapy as consolidation treatments. The specific consolidation approach depends on the child’s age and response to initial treatment.
Study on the Effectiveness of Bevacizumab and Drug Combination for Children with Recurrent or Progressive Medulloblastoma, Ependymoma, and ATRT
This trial focuses on children whose tumors have returned or continued to grow after previous treatment. While the study includes three types of brain tumors (medulloblastoma, ependymoma, and atypical teratoid/rhabdoid tumor), it offers a treatment option for children facing recurrent or progressive disease.
Main inclusion criteria: Participants must have recurrent or progressive medulloblastoma, ependymoma, or atypical teratoid/rhabdoid tumor confirmed through tissue examination. There must be at least one site of disease that has not been treated again. Children can be male or female and must be under 20 years old at the time of original diagnosis. They must have normal organ and bone marrow function, including liver enzymes (ALT less than 5 times normal), kidney function (creatinine less than 1.5 times normal for age), white blood cells greater than 1000 per mm³, and platelets greater than 20,000 per mm³. A performance status score of at least 50 is required, indicating reasonable ability to perform daily activities. Written informed consent from patients and/or parents is necessary.
Main exclusion criteria: The specific exclusion criteria focus on ensuring participants meet the age requirements and are not part of vulnerable populations that might need special protection or care during the study.
Focus and goals: The study explores the effectiveness of a combination of medications that target the blood vessels supplying the tumor, known as anti-angiogenesis therapy. By cutting off the tumor’s blood supply, this approach aims to prevent it from receiving the nutrients needed to grow. The research team will evaluate how tumors respond to treatment, whether they shrink, remain stable, or grow, and monitor how long patients remain free of tumor growth. The trial also assesses safety, side effects, overall health, and quality of life during and after treatment.
Investigational drugs: The trial tests a combination of seven medications: Cytarabine (given as an injection into the intrathecal space around the spinal cord), Celecoxib (oral capsule), Cyclophosphamide (oral solution), Etoposide (intravenous infusion), Bevacizumab (intravenous infusion), Fenofibrate (oral capsule), and Thalidomide (oral capsule). This approach is called metronomic anti-angiogenic therapy, which involves giving lower doses of chemotherapy drugs more frequently to continuously attack cancer cells while minimizing side effects and cutting off the tumor’s blood supply.
Summary
Both clinical trials available for atypical teratoid/rhabdoid tumour of the CNS are focused exclusively on pediatric patients, reflecting the fact that this aggressive brain tumor primarily affects young children. The first trial is available across 12 European countries (Sweden, Germany, Netherlands, Denmark, Norway, Belgium, Italy, France, Finland, Spain, Hungary, and Czechia) and focuses on newly diagnosed cases, testing different treatment strategies based on the child’s age. The second trial operates in 7 countries (Sweden, Austria, Norway, Czechia, Denmark, France, and Spain) and specifically addresses recurrent or progressive disease.
The treatment approaches differ significantly between the two studies. The first trial uses intensive chemotherapy followed by either high-dose chemotherapy or radiotherapy as consolidation, while the second employs a metronomic anti-angiogenic approach designed to starve tumors of their blood supply. Both trials emphasize careful monitoring of participants’ health, organ function, and quality of life throughout treatment.
Families considering participation should note that both trials require extensive medical monitoring and documented normal organ function. The first trial may be particularly relevant for children with newly diagnosed tumors, while the second offers hope for those whose disease has returned or progressed after initial treatment.
