Recurrent Medulloblastoma

The purpose of this study is to find out whether 177Lu-DPTA-omburtamab is a safe treatment for children and adolescents with recurrent/refractory medulloblastoma or another type of cancer with CNS metastases.

The primary objective will be to demonstrate the manufacturing feasibility and safety, and to determine the maximum tolerated dose (MTD) of RNA-LP vaccines in pediatric patients with recurrent/progressive Medulloblastoma (MB)

Children with medulloblastoma, an aggressive brain tumor, require prolonged and complex multimodal therapy. Their primary parental caregivers bear the main responsibility for ensuring treatment adherence, yet the psychological toll on these caregivers may undermine their ability to follow prescribed regimens. This study investigates whether multidimensional parental distress predicts overall treatment adherence in this population, and which specific distress domains are most strongly associated with poor adherence. Parental distress-including depression, anxiety, and posttraumatic stress-is common among parents of children with cancer and may interfere with their ability to manage complex treatment regimens. However, no study has specifically examined whether parental distress predicts treatment adherence in children with recurrent medulloblastoma, a devastating brain tumor with a poor prognosis and no standard treatment protocol. This prospective longitudinal cohort study aims to investigate whether parental distress is a significant predictor of treatment adherence in children with recurrent medulloblastoma. A total of 450 parent-child dyads will be enrolled across 4 tertiary pediatric oncology centers. Children must have a confirmed diagnosis of recurrent medulloblastoma and be receiving active treatment. Parents (primary caregivers) will complete validated questionnaires at baseline assessing depression, anxiety, stress, and trauma-related distress using the DASS-21 and IES-R. Treatment adherence will be monitored over a 12-month follow-up period using electronic medication monitoring caps, clinic attendance records, and parent-reported medication logs. The primary outcome is the proportion of prescribed chemotherapy doses taken (treatment adherence rate). Secondary outcomes include trajectories of adherence over time and the relationship between specific dimensions of parental distress (depression, anxiety, stress, posttraumatic stress) and adherence patterns. The study will also examine whether child clinical factors (e.g., molecular subgroup, prior treatment history) and family demographic factors moderate this relationship. Findings from this study may inform the development of targeted psychosocial interventions to support distressed parents and improve treatment adherence-and ultimately clinical outcomes-in this vulnerable pediatric population. Participants can expect to be enrolled in the study for approximately 12 months.

This study will address the question of whether targeting CMV antigens with PEP-CMV can serve as a novel immunotherapeutic approach in pediatric patients with newly-diagnosed high-grade glioma (HGG) or diffuse intrinsic pontine glioma (DIPG) as well as recurrent medulloblastoma (MB). PEP-CMV is a vaccine mixture of a peptide referred to as Component A. Component A is a synthetic long peptide (SLP) of 26 amino acid residues from human pp65. The SLPs encode multiple potential class I, class II, and antibody epitopes across several haplotypes. Component A will be administered as a stable water:oil emulsion in Montanide ISA 51. Funding Source - FDA OOPD

This study will evaluate the safety and efficacy of Lutathera (177Lu-DOTATATE) in patients with progressive or recurrent High-Grade Central Nervous System (CNS) tumors and meningiomas that demonstrate uptake on DOTATATE PET. The drug will be given intravenously once every 8 weeks for a total of up to 4 doses over 8 months in patients aged 4 to \<12 years (Phase I) or 12 to \</=39 years (Phase II) to test its safety and efficacy, respectively. Funding Source - FDA OOPD (grant number FD-R-0532-01)

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.

This study is a prospective single-center clinical study, which aims to observe and evaluate the efficacy and safety of apatinib combined with temozolomide and oral etoposide in the treatment of recurrent medulloblastoma in children.