PNOC COMP: A Protocol for Children and Young Adults Diagnosed with a Central Nervous System (CNS) Tumor to Assess Cognitive, Quality of Life (QOL), and Comprehensive Effects of Therapies
This study aims to build a clearer picture of how brain tumors and their treatments affect children, teens, and young adults over time. We will track outcomes such as learning and memory, quality of life, and everyday functioning.
New therapies, like medicines that target specific tumor changes (for example, MEK or BRAF inhibitors) and immune-based treatments (such as checkpoint inhibitors, vaccines, oncolytic viruses, or CAR T-cell therapy), are becoming part of care for young patients. Because many participants are still growing and developing, it is especially important to understand how these treatments may impact the brain both in the short term and over many years.
By following patients in a structured way, this protocol will allow us to gather information about the effects of new therapies in a comprehensive and meaningful manner. We will also take into account the genetics of each patient’s tumor and, when available, genetic information inherited from their family background that is already collected as part of standard medical care. Linking these biological factors with real-world outcomes may reveal important patterns, particularly as advanced computer methods like machine learning become more widely available.
The ultimate goal is to create a resource that connects genetic information with patient experiences and outcomes, and to make this available to the wider research community. Enrollment is open to all children, teens, and young adults with brain tumors—not just those in PNOC treatment trials. However, patients who are enrolled in a PNOC therapeutic trial are required to also join this long-term follow-up study.
Primary Objectives:
- To longitudinally assess cognitive outcomes using a neurocognitive screening battery in children and young adults diagnosed with a CNS tumor.
- To longitudinally assess the impact of specific therapies on the QOL in children and young adults diagnosed with a CNS tumor.
- To longitudinally assess brain, spine, and vascular imaging to correlate these with treatment, cognitive, and QOL outcomes.
Exploratory Objectives:
- To assess how germline alterations impact tumor response, and cognitive outcomes in defined disease entities.
- To collect comprehensive genomic, epigenetic, transcriptomic, immune profiling, and phenotypic data on children and young adults diagnosed with a CNS tumor.
- To longitudinally assess endocrine, visual and auditory function in children and young adults diagnosed with a CNS tumor.
- To longitudinally assess measures of daily functional independence in children and young adults diagnosed with a CNS tumor.
- To assess patient and/or proxy satisfaction with study participating via patient-reported outcome (PRO) measures in the context of race ethnicity and other health related social risks.
- To assess on therapy toxicity in the context of race, ethnicity and other health related social risks.
Inclusion Criteria:
- Diagnosis: children and young adults with CNS tumor including brain and spinal cord tumors
- Patients with neurofibromatosis or other hereditary tumor predisposition syndromes are eligible for this trial if they have a CNS (brain or spinal cord) tumor
- Age: 0-39 years of age at the time of initial CNS tumor diagnosis
- Patients who are enrolled on a PNOC therapeutic trial will co-enroll in this study. These patients may be any age at the time of enrollment but must have been ≤ 39 at the time of diagnosis.
- Patients who are not enrolled in a PNOC therapeutic clinical trial are eligible but not required to participate in this study. These patients must be ≤ 39 at the time of enrollment.
- Patients without tissue available will still be eligible for the trial.
None
