Clinical Trial
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. Subjects may be alive or deceased.
Additional data are needed to identify prognostic clinical, biologic and imaging variables for ATRT and large, accessible detailed data sets required for such analyses do not currently exist.
Primary Objective(s)
- To analyze the significance of treatment, clinical and molecular variables in predicting progression free survival (PFS) at 6 months (PFS6) from date of diagnosis.
- To create a well annotated ATRT biorepository for current and future studies through the collection of biospecimens, imaging and key clinical data.
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.
Support is provided by Team Beans (DFCI) and PNOC Foundation.
This study will enroll subjects with newly diagnosed, progressive/recurrent/refractory or previously treated ATRT. Subjects may be alive or deceased.
Additional data are needed to identify prognostic clinical, biologic and imaging variables for ATRT and large, accessible detailed data sets required for such analyses do not currently exist.
Primary Objective(s)
- To analyze the significance of treatment, clinical and molecular variables in predicting progression free survival (PFS) at 6 months (PFS6) from date of diagnosis.
- To create a well annotated ATRT biorepository for current and future studies through the collection of biospecimens, imaging and key clinical data.
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.
Support is provided by Team Beans (DFCI) and PNOC Foundation.
Sites Offering This Trial
How to Enroll
If you believe your child or patient is eligible for this trial, contact the closest participating site or email us for more information.