Clinical Trial: 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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Treatment of Atypical Teratoid/Rhabdoid Tumors (AT/RT) of the Central Nervous System With Surgery, Intensive Chemotherapy, and 3-D Conformal Radiation

Brief Summary: This phase III trial is studying giving combination chemotherapy together with 3-dimensional conformal radiation therapy and an autologous peripheral blood stem cell transplant to see how well it works 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.

Detailed Summary:

PRIMARY OBJECTIVES:

I. To determine the 6-, 12-, and 24-month event-free survival and overall survival of children (birth to 21 years of age) with atypical teratoid/rhabdoid CNS tumors (AT/RT), diagnosed based on histology, immunophenotyping, and modern molecular and immunohistochemical analysis of INI1, treated with surgery, intensive chemotherapy combined with stem cell rescue, and radiation therapy.

II. To compare the outcome of very young patients (under 3 years old) on this study whose histologic diagnosis is AT/RT with infants identified as having AT/RT on POG-9233 and CCG-9921.

SECONDARY OBJECTIVES:

I. To determine the feasibility and toxicity of the proposed chemotherapy regimen in combination with radiation therapy.

II. To contribute tumor samples from which biologic and gene expression data can be developed to yield prognostic indicators and provide direction for future treatment strategies.

III. To develop a clinical and biologic database on which future studies can be based.

OUTLINE: This is a multicenter study. Patients are stratified according to age and tumor histology (infants [< 36 months of age] with tumor histology and immunohistochemical [IH] analysis diagnostic of atypical teratoid/rhabdoid CNS tumors [AT/RT] [stratum 1] vs infants with INI1 mutation only-based diagnosis [i.e., histology is not consistent with AT/RT] vs older children [≥ 36 months of age] with tumor histology and IH analysis diagnostic of AT/RT vs older children with INI1 mutation only-based diagnosis).

INDUCTION THERAPY AND STEM CE
Sponsor: Children's Oncology Group

Current Primary Outcome:

  • Event-free Survival [ Time Frame: Up to 4 years after study enrollment ]
    Estimated 4-year EFS where EFS is calculated as the time from study enrollment to disease progression, disease relapse, occurrence of a second malignant neoplasm, death from any cause or last follow-up whichever occurs first. Kaplan-Meier method is used for estimation. Patients without an event are censored at last contact.
  • Overall Survival (OS) [ Time Frame: Up to 4 years after study enrollment ]
    Estimated 4-year survival, where survival is calculated as the time from study enrollment to death from any cause or last follow-up alive whichever occurs first. Kaplan-Meier method is used for estimation. Patients alive at last contact are censored.
  • Toxic Death [ Time Frame: During and after completion of study treatment up to 1 year after enrollment. ]
    The number of patients who experience death that is considered to be primarily attributable to complications of treatment.


Original Primary Outcome:

  • Event-free survival
  • Toxic death, defined as death primarily attributable to complications of treatment


Current Secondary Outcome: Non-hematological Toxicity Associated With Chemotherapy: Grade 3 or Higher During Protocol Therapy [ Time Frame: During protocol therapy up to 1 year after enrollment. ]

Number of Participants with Nonhematological Toxicity Associated With Chemotherapy: Grade 3 or Higher During Protocol Therapy.


Original Secondary Outcome:

  • Toxicity and safety
  • Survival


Information By: Children's Oncology Group

Dates:
Date Received: April 3, 2008
Date Started: December 2008
Date Completion:
Last Updated: February 13, 2017
Last Verified: February 2017