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Diffuse intrinsic pontine gliomas (DIPGs), which diffusely occupy the pons of brainstem, are the deadliest primary brain cancer in children. Biopsy for pathology plus radiotherapy remains the current standard-of-care treatment that is minimal effective. Thus, the median overall survival after diagnosis is just 10 months. Recent studies have identified a lysine 27-to-methionine (K27M) somatic mutation at histone H3 variant (H3.3), as a feature mutation in DIPGs. Several preclinical studies have already demonstrated H3.3-K27M as a promising target for immunotherapy. The researched vaccine is a cancer-treatment vaccine containing an ...
Study to investigate the safety and activity of NEO-PTC-01 in patients with unresectable or metastatic melanoma. NEO-PTC-01 is an autologous personalized T cell (PTC) product for adoptive cell therapy that is manufactured ex vivo and targets neoantigens displayed on the patient's tumor and the tumor microenvironment. The study will be conducted in two parts, Part 1 (Dose Finding) and Part 2 (Dose Expansion). The dose-finding part of the study will test two doses of NEO-PTC-01 and will be structured according to a 3+3 dose escalation design. After the highest tolerated NEO-PTC-01 dose is identified, 2 additional evaluations in Part...
This research study is studying the drugs called NeoVax (a new type of personalized neoantigen vaccine) in combination with CDX-301 and Nivolumab or Pembrolizumab as a possible treatment for melanoma. The names of the study drugs involved in this study are: - Personalized Neoantigen peptides (which combined with poly-ICLC make the vaccine NeoVax) - Poly-ICLC (Hiltonol) - CDX-301 - Nivolumab (Opdivo) - Pembrolizumab (Keytruda)
Background: Neurobehavioral functions and quality of life (QoL) are the important outcome measurements after radiotherapy in patients with brain tumors and even head/neck cancers. However, few studies have focused on neurobehavioral functions and QoL after anti-cancer treatment particularly brain radiotherapy for pediatric/adolescent patients with brain tumors. This study thus aims to prospectively evaluate those functions in pediatric or adolescent patients with brain or head/neck tumors in order to provide useful information about their clinical outcomes. Methods: A total of 72 pediatric/adolescent patients, who are diagnosed...
Medical scientists want to find better ways to treat neuroblastoma and to find ways to prevent the tumor from growing back. To do this, they need more information about the characteristics of neuroblastoma cells. Therefore, they want to study samples of neuroblastoma tissues and neuroblastoma and normal cells in the blood and bone marrow that may be related to the growth of neuroblastoma cells. Doctors and other medical scientists also want to find better ways to detect and measure neuroblastoma to improve the ability to follow the response of tumor cells to therapy.
Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter, study for patients with neuroblastoma in remission. In this study subjects will receive 730 Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1, 2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study. This study will focus on the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to prevent recurrence.
The goal of this multicenter prospective longitudinal study is to study the long-term impact of multimodal treatment (chemotherapy, radiotherapy and surgery) in adult brain and base of skull tumors on neurocognitive functioning. All included patients will complete a self-report inventory (subjective cognitive functioning, QoL, confounders), a cognitive test battery, an advanced MR at multiple timepoints. Moreover, toxicity will be scored according to the CTCAEv5.0 in these patients over time.
Congenital central hypoventilation syndrome (CCHS) is a rare disorder of autonomic and respiratory regulation that frequently alters oxygen delivery to the brain. In CCHS, neurocognitive function has been of great concern because of the potential for repeated hypoxemia and hypercarbia in activities of daily living in addition to hypoventilation with related hypoxemia and hypercarbia during sleep. As the world's leading referral center for CCHS, the Center for Autonomic Medicine in Pediatrics (CAMP) is engaged in ongoing research to identify factors that impact neurocognitive performance in patients with CCHS in order to optimize...
This phase II trial studies the neurological function in patients with multiple brain metastases undergoing stereotactic radiosurgery (SRS) or stereotactic body radiation therapy (SBRT). Stereotactic body radiation therapy uses special equipment to position a patient and deliver radiation to tumors with high precision. This method can kill tumor cells with fewer doses over a shorter period and cause less damage to normal tissue. Assessment of neurocognitive function may help show that SRS preserves neurological function in patients with multiple brain metastases better than SBRT.
This will be the first in-depth study to evaluate pretreatment and 12 months post-treatment, neurocognitive and psychological outcomes of children with brain tumor and blood cancer in Pakistan. The investigators will also determine the socioeconomic burden of pediatric brain tumors in low middle-income region and the association of micro RNA and protein markers with neurocognitive outcomes in PBT and blood cancer children. A prospective cohort study with a follow-up of 12 months at the Aga Khan University Hospital, Karachi, Pakistan and Jinnah postgraduate Medical Centre , Karachi, Pakistan will be conducted. After taking consent/ ...
