Clinical trial · Interventional
Combination Chemotherapy, Radiation Therapy, and an Autologous Peripheral Blood Stem Cell Transplant in Treating Young Patients With Atypical Teratoid/Rhabdoid Tumor of the Central Nervous System
Treatment of Atypical Teratoid/Rhabdoid Tumors (AT/RT) of the Central Nervous System With Surgery, Intensive Chemotherapy, and 3-D Conformal Radiation
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This phase III trial studies the side effects of combination chemotherapy, 3-dimensional conformal radiation therapy, and an autologous peripheral blood stem cell transplant, and to see how well they work in treating young patients with atypical teratoid/rhabdoid tumor of the central nervous system. Giving high-dose chemotherapy before an autologous peripheral blood stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as G-CSF, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy or radiation therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy or radiation therapy.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Childhood Atypical Teratoid/Rhabdoid Tumor | Childhood Atypical Teratoid/Rhabdoid Tumor | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (12)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| 3-Dimensional Conformal Radiation Therapy | Radiation | — | UNRESOLVED |
| Autologous Hematopoietic Stem Cell Transplantation | Procedure | — | UNRESOLVED |
| Carboplatin | Drug | Carboplatin | ALIAS |
| Cisplatin | Drug | Cisplatin | ALIAS |
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Etoposide | Drug | Etoposide | ALIAS |
| Filgrastim | Biological | Filgrastim | ALIAS |
| Laboratory Biomarker Analysis | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Arm I (chemotherapy, autologous PBSC, 3D-CRT)
- description
- Patients receive vincristine IV on days 1, 8, and 15; high-dose methotrexate IV on day 1; leucovorin calcium orally or IV; etoposide IV on days 4, 5, and 6; cyclophosphamide IV on days 4 and 5; cisplatin IV on day 6, and G-CSF IV or SC on day 7 until ANC recovers. Within 2-6 weeks after induction therapy or radiation therapy, patients receive high-dose carboplatin IV and high-dose thiotepa IV on days 1 and 2 and undergo autologous PBSC rescue on approximately day 4. Patients also receive G-CSF IV or SC once daily until ANC recovers. Treatment with consolidation therapy followed by stem cell rescue repeats every 28 days for 3 courses in the absence of disease progression or unacceptable toxicity. After consolidation therapy, patients undergo 3D-CRT to the brain (and the spine if needed) 5 days a week for 5-6 weeks.
- interventionNames
- Radiation: 3-Dimensional Conformal Radiation Therapy
- Procedure: Autologous Hematopoietic Stem Cell Transplantation
- Drug: Carboplatin
- Drug: Cisplatin
- Drug: Cyclophosphamide
- Drug: Etoposide
- Biological: Filgrastim
- Other: Laboratory Biomarker Analysis
- Drug: Leucovorin Calcium
- Drug: Methotrexate
- Drug: Thiotepa
- Drug: Vincristine Sulfate
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 21 Years
Show eligibility criteria text
Inclusion Criteria: * Diagnosis of CNS atypical teratoid/rhabdoid tumor (AT/RT) or tumors that have a mutation of the INI1 gene (even if the tumor does not have the usual histologic characteristics of AT/RT) * Patients with extra neural metastasis (M4) or renal rhabdoid tumors are not eligible * Patients with MRI evidence of spinal disease are eligible * Must have undergone definitive surgery in the past 31 days * Cranial MRI (with and without gadolinium) must be done pre-operatively * Post-operatively, cranial MRI (with and without gadolinium) must be done, preferably within 48 hours of surgery or 10-28 days after surgery * Entire spinal MRI must be obtained either pre-operatively (with gadolinium) or post-operatively (10-28 days after surgery), prior to study enrollment (with and without gadolinium) * Life expectancy \> 8 weeks * ANC \> 1,000/uL * Platelet count \> 100,000/uL (transfusion independent) * Hemoglobin \> 8 g/dL (RBC transfusions allowed) * Creatinine clearance (minimum of 12-24 hour urine collection) or radioisotope GFR \>= 60 mL/min * Total bilirubin =\< 1.5 times upper limit of normal (ULN) for age * AST and ALT \< 2 times ULN for age * Shortening fraction of \>= 27% by echocardiogram OR ejection fraction of \>= 47% by radionuclide angiogram * No evidence of dyspnea at rest * Pulse oximetry \> 94% on room air * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception * No prior radiotherapy or chemotherapy except for the following: * Patients enrolled on protocol ACNS0334 whose tumors demonstrate the INI1 gene mutation are eligible to transfer to this study even if they have received one course of induction therapy (these patients must be re-consented to treatment and restaged) * Prior corticosteroids allowed
References
Publications (2)
- DERIVEDMazzarella J, Heathcock JC. A Randomized Feasibility Study of Rehabilitation Targeting Upper Extremity Function and Participation Using Hippotherapy and the Equine Environment for Children with Cerebral Palsy and Autism Spectrum Disorder. J Integr Complement Med. 2025 Feb;31(2):196-208. doi: 10.1089/jicm.2024.0292. Epub 2024 Nov 8. PMID 39515374
- DERIVEDReddy AT, Krailo MD, Buxton AB, Strother DR, Huang A, Zhou T, Judkins AR, Burger PC, Pollack IF, Williams-Hughes C, Fouladi M, Ho B, Mazewski CM, Lewis VA, Vezina LG, Booth TN, Mahajan A. Reply to S.A. Upadhyaya. J Clin Oncol. 2020 Oct 1;38(28):3353-3354. doi: 10.1200/JCO.20.01573. Epub 2020 Jul 30. No abstract available. PMID 32730180