Clinical Research Directory

Targeting Pediatric Brain Tumors and Relapsed/Refractory Solid Tumors With Sodium Glucose Cotransporter 2 Inhibitors (SGLT2i)

This is a pilot phase Ib study of the safety of dapagliflozin (in addition to standard of care treatment) for the treatment of pediatric patients with recurrent brain tumors and relapsed/refractory solid tumors. The primary hypothesis is that dapagliflozin is well-tolerated and safe to use in this patient population. The investigators also hypothesize that dapagliflozin will be efficacious as an adjunct to front-line chemotherapy assessed by decreased tumor markers mediated by its pleiotropic metabolic effects.

Intra-Tumoral Injections of Natural Killer Cells for Recurrent Malignant Pediatric Brain Tumors

This phase I trial tests the safety, side effects, and best dose of ex vivo expanded natural killer cells in treating patients with cancerous (malignant) tumors affecting the upper part of the brain (supratentorial) that have come back (recurrent) or that are growing, spreading, or getting worse (progressive). Natural killer (NK) cells are immune cells that recognize and get rid of abnormal cells in the body, including tumor cells and cells infected by viruses. NK cells have been shown to kill different types of cancer, including brain tumors in laboratory settings. Giving NK cells from unrelated donors who are screened for optimal cell qualities and determined to be safe and healthy may be effective in treating supratentorial malignant brain tumors in children and young adults.

Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas

The study evaluates CLR 131 in children, adolescents, and young adults with relapsed or refractory malignant solid tumors and lymphoma and recurrent or refractory malignant brain tumors for which there are no standard treatment options with curative potential.

High-Intensity Interval Exercise in Young Adult Survivors of Pediatric Brain Tumors: A Pilot Feasibility Study

The purpose of this study is to determine whether a 16-week virtual, home-based, high-intensity interval training (HIIT) exercise program will improve physical, cognitive, and emotional health among young adult survivors of pediatric brain tumors. The names of the study interventions involved in this study are/is: * High-Intensity Interval Training (HIIT)

HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors

This study is a clinical trial to determine the safety of inoculating G207 (an experimental virus therapy) into a recurrent or refractory cerebellar brain tumor. The safety of combining G207 with a single low dose of radiation, designed to enhance virus replication, tumor cell killing, and an anti-tumor immune response, will also be tested. Funding Source- FDA OOPD

GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults

This is a single-site, open-label Phase 1 clinical trial evaluating the feasibility, safety, and preliminary activity of autologous GPC2-targeted chimeric antigen receptor (CAR) T cells administered via intracerebroventricular (ICV) infusion in children and young adults with relapsed or refractory medulloblastoma or other eligible Central Nervous System (CNS) embryonal tumors.

A Multicenter Study to Assess the Feasibility of 5-Aminolevulinic Acid (5-ALA) in Pediatric Brain Tumor Patients

This clinical trial focuses on pediatric patients aged 2 up to 18 years of age with a new or recurrent pediatric brain tumor, suspected to be either a high-grade or low-grade glioma, and scheduled for surgical removal. 5-aminolevulinic acid (5-ALA) is FDA-approved for improving brain tumor visualization in adults during surgery through fluorescence, enabling more complete removal of the tumor. This study aims to evaluate the feasibility of administering 5-ALA to pediatric brain tumor patients and to assess the quality of tumor fluorescence during surgery in this patient population. For the clinical trial, the patient will orally ingest 5-ALA 6 to 12 hours before brain surgery. All study participants will be provided standard medical care for removal of the brain tumor. All children enrolled in the study will be closely monitored prior to, during, and after surgery to ensure there are no reactions to the study drug. 5-ALA can make the patient more sensitive to sunlight and direct indoor lighting, referred to as photosensitivity, and can cause a sunburn-type reaction. It is for this reason that patients will be kept in subdued light conditions for 48 hours following surgery. Study participation starts once the patient is enrolled in the study until 6-month post-surgery.

Survivors Journey+

The main goal of this pilot randomized controlled trial is to learn if an online program called "Survivors Journey" (SJ+) can help teens and young adults, ages 15-25, who are Pediatric Brain Tumor Survivors (PBTS), and their caregivers, manage everyday challenges better by using skills like problem-solving and coping skills. The main questions it aims to answer are: * Is the SJ+ program rated as feasible (\>50% enrollment rate and \>75% retention rate) and acceptable (\>80% satisfaction rate) by PBTS and their caregivers? * Does the SJ+ program have better outcomes in improving PBTS and caregiver wellbeing in comparison to an internet resource comparison (IRB) made for PBTS and their families? Participants will be randomized into two groups: one group will be given access to the online SJ+ program and receive weekly online coaching sessions, and the other group will be given access to an IRC. Outcomes will be assessed at baseline, treatment completion (\~ 3 months post-baseline), and at follow-up six months post-baseline. These outcomes include quality of life, internalizing symptoms, performance-based executive function skills, depression, and family impact.

Understanding the Late Effects of Surviving a Pediatric Brain Tumor

The goal of this study is to learn about the cognitive and behavioral functioning of children being treated for cancer.

Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan for Patients With High Grade Glioma

This is a 2 strata pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The study will use a new treatment approach based on each patient's tumor gene expression, whole-exome sequencing (WES), targeted panel profile (UCSF 500 gene panel), and RNA-Seq. The current study will test the efficacy of such an approach in children with High-grade gliomas HGG.

Evaluation of the Neurologic Assessment in Pediatric Neuro-Oncology (pNANO)

This study aims to investigate a neurologic exam scale to provide an objective and more standard way to assess tumor response in pediatric patients with brain and spinal cord tumors.

Non-Helium Magnetoencephalography in Pediatric Brain Tumors

This observational study aims to evaluate the effectiveness of magnetoencephalography (MEG) in the preoperative assessment of pediatric brain tumors, particularly in determining the extent of resection, and compare it with computed tomography (CT) and magnetic resonance imaging (MRI). The main questions this study aims to answer are: What is the consistency between MEG and CT/MRI in localizing pediatric brain tumors? What is the clinical efficacy of MEG in determining the extent of resection of pediatric brain tumors preoperatively? Participants will: Undergo both MEG and CT/MRI examinations for preoperative assessment, and the extent of tumor resection will be determined by the comprehensive results of the above examinations. Be followed up with MEG and CT/MRI at 1, 3, and 6 months after surgery, along with neurological and quality-of-life assessments.

The FLaME Cognitive Rehabilitation Study for Childhood Brain Tumour

Medical treatments have improved survival rates for children with brain tumours. However, most children experience long-term difficulties with 'cognition' (thinking skills such as memory and paying attention) and cognitive fatigue (excessive mental tiredness) after treatment. Thinking difficulties and fatigue can affect a child's ability to learn, and their social and emotional wellbeing. National guidance recommends treatment called 'cognitive rehabilitation' which teaches skills to improve or manage cognitive difficulties. Families often request this, but it is not usually available due to little research. Fatigue may also get in the way of children using and benefiting from cognitive rehabilitation. No research study has offered a fatigue treatment for children recovering from brain tumours. The study aims to see if it is practical and helpful to families to provide cognitive rehabilitation for children affected by brain tumours. The treatment focuses on strategies to help cognition. The investigators will see if adding strategies to manage fatigue helps. The study will include thirty-six 7-17-year-olds who have been treated for brain tumour at Great Ormond Street Hospital. All participants will have had an assessment describing cognitive strengths and weaknesses as part of usual care. Participants will be randomly allocated to one of three groups: 1) cognitive rehabilitation with fatigue management (12 weeks), 2) cognitive rehabilitation only (6 weeks), or 3) usual care. Each child and their carer will complete questionnaires before, during, and after the treatment, and an interview at the end of the treatment. This information will help the researchers see if families find the treatment helpful and practical to take part in, and if adding fatigue strategies is beneficial. Researchers will look at information such as the number of appointments attended, feedback about the treatment, and information about fatigue levels, cognition, and wellbeing. The findings will be used to develop a UK-wide study.

Feasibility/Acceptability of Attentional-Control Training in Survivors

This is a multicenter pilot randomized controlled trial, with an active control condition, of the feasibility, acceptability, and preliminary efficacy of EndeavorRx in a cohort of survivors of acute lymphoblastic leukemia or brain tumor ages 8-16 who are \> 1 year from the end of therapy.