Human stem cell derived neuron

PNOC030: A Registry Study for Children with Atypical Teratoid Rhabdoid Tumor (ATRT)

This study will enroll subjects with newly diagnosed, progressive/recurrent/refractory or previously treated ATRT (Atypical Teratoid Rhabdoid Tumor).

 
 

The goal of this registry study is to collect more information to help identify clinical, biological, and imaging factors that can predict outcomes for ATRT.

The aim is to study how different treatments, clinical factors, and molecular features affect the chances of a patient's disease not worsening for six months after diagnosis (known as progression-free survival or PFS6).

Additionally, the goal is to build a detailed ATRT biorepository by collecting biological samples, medical images, and important clinical information. This will help with current and future research.

Inclusion Criteria
  • Participants must have a pathologic diagnosis of ATRT. The diagnosis must be supported by (1) immunohistochemistry demonstrating loss of INI1 or BRG1 or (2) identification of a pathogenic alteration in SMARCB1 or SMARCA4. Participants with extra-neural metastases/synchronous tumors (M4) are eligible. Participants of all ages are eligible.
  • Submission of tumor tissue, prior submission of tumor tissue to Children’s Brain Tumor Network (CBTN), or report from a CLIA (or equivalent) approved laboratory with molecular subgroup analysis is mandatory (tissue may be from initial diagnosis or relapse).
  • Co-enrollment on Protocol for Children and Young Adults with a Central Nervous System (CNS) Tumor to Assess Cognitive, Quality of Life (QOL), and Comprehensive Effects of Therapies (PNOC-COMP) is strongly encouraged when subjects are eligible.
  • Participants may be alive or deceased.
  • A legal parent/guardian or participant must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.
Exclusion Criteria
  • Participants without at least one of the following: tumor tissue available, tumor tissue previously submitted to CBTN, or an approved report with molecular subgroup determination from a either a CLIA certified laboratory or equivalent will not be eligible.

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