Hemangioblastoma

Researchers are evaluating the optimization of 18F-DOPA PET/CT imaging in specific patient groups including pediatric patients with congenital hyperinsulinism or neuroblastoma, pediatric and adult patients with neuroendocrine tumors or brain tumors, and adults suspected of Parkinson's disease or Lewy body dementia. The study aims to improve image quality using a new digital PET/CT scanner and intravenous furosemide, while also exploring gallbladder activity patterns related to dopaminergic degeneration. Participants will receive an intravenous injection of 18F-DOPA, with some also receiving a single intravenous dose of furosemide. The study includes a primary objective of assessing image quality improvements in the pelvis area and a secondary objective examining gallbladder activity patterns using dynamic imaging in a subgroup. Imaging data will be compared to previous scans with older technology. During the study, participants will undergo PET/CT scans, with measurements of lesion size and activity, bladder activity, and image artifact scoring. A questionnaire will screen for gallbladder disease history. The research team will analyze the imaging results and gallbladder activity patterns, with follow-up assessments occurring within one to three months. The total planned enrollment is 800 patients over approximately five years, with detailed monitoring of image optimization and gallbladder activity.

Researchers are evaluating the diagnostic value of a new protein-specific probe called 18F-T2 in PET/CT imaging for people with solid tumors that are likely to express high levels of CAIX protein. The study will also assess how safe and tolerable the 18F-T2 injection is, as well as measure its radiation dosage. This research is important to better understand how well 18F-T2 can detect these tumors compared to standard imaging techniques. Participants with tumors suspected to express high levels of CAIX will receive an intravenous injection of 18F-T2. About an hour after the injection, PET/CT imaging will be performed to capture detailed images of the tumors. Within one week, participants will also undergo a whole-body PET/CT scan using 18F-FDG, a commonly used imaging agent, to allow comparison between the two imaging methods. During the study, participants will be monitored for any adverse events within 24 hours after the 18F-T2 injection to evaluate safety and tolerability. Researchers will measure the diagnostic sensitivity and specificity of 18F-T2 PET/CT for detecting CAIX-positive tumors. They will also assess uptake values in tumors on both 18F-T2 and 18F-FDG scans, analyze the correlation between 18F-T2 uptake and CAIX expression in tissue samples, and evaluate radiation dosimetry. The study will continue until one month after completion for outcome assessments.

Researchers are evaluating the use of Gallium-68-DOTATATE PET/MRI in patients with somatostatin receptor-positive (SSTR-positive) central nervous system (CNS) tumors, mainly focusing on meningioma but also including other tumor types such as esthesioneuroblastoma, hemangioblastoma, medulloblastoma, paraganglioma, pituitary adenoma, and SSTR-positive systemic cancers metastatic to the brain. The study aims to assess the diagnostic usefulness of this imaging technique, especially in distinguishing tumor recurrence from post-treatment changes, with a particular interest in cases where tumor location limits surgical removal or where patients have higher-grade disease or previous radiation treatment. This is a Phase 4 interventional study sponsored by Weill Medical College of Cornell University.

Researchers are evaluating a new dual-target imaging agent called 68Ga-RM26-RGD for PET/CT scans in patients with breast, brain, and prostate cancers that express specific receptors (GRPR and integrin αvβ3). This study aims to improve tumor detection by comparing this agent with conventional 18F-FDG and single-target imaging agents, addressing limitations of current imaging methods in detecting tumors with low 18F-FDG uptake or variable receptor expression. Participants will undergo PET/CT scans using intravenous injections of 68Ga-RM26-RGD and one of the comparator agents (18F-FDG, 68Ga-RM26, or 68Ga-RGD) within a two-week period. The dosages for these agents are approximately 1.8-2.2 MBq/kg. Each patient receives scans with both agents to compare imaging performance directly. During the study, researchers will assess diagnostic accuracy and dosimetry of 68Ga-RM26-RGD over about one year. Participants will have multiple PET/CT scans and related evaluations to monitor uptake of the imaging agents in tumors. The study includes safety and diagnostic performance monitoring throughout the participation period.

Researchers are evaluating a new clinical fMRI software designed to map critical brain functional networks and cerebrovascular reactivity in patients with brain tumors. The goal is to improve presurgical planning by integrating resting-state fMRI (rs-fMRI), task-based fMRI (tb-fMRI), and cerebrovascular reactivity (CVR) mapping. This observational study aims to validate the accuracy of rs-fMRI compared to tb-fMRI using intra-operative direct cortical stimulation as the gold standard, and to test whether CVR mapping enhances confidence in identifying important brain areas. The study involves developing and optimizing software to map resting-state networks and CVR, then testing its use in patients undergoing neurosurgical tumor removal. The research includes retrospective review of prior clinical presurgical fMRI studies and a prospective phase where patients will have presurgical fMRI and direct cortical stimulation as part of their standard care. The software development will be done in partnership with a commercial MRI software producer to create robust clinical tools. Participants will undergo presurgical fMRI scans, including resting-state and task-based imaging, as part of their usual care, followed by direct cortical stimulation during surgery. Researchers will analyze these imaging data to validate the software's mapping accuracy. The primary outcome is to confirm the ability of rs-fMRI to identify language and motor areas reliably. The study will continue through to completion, averaging about one year per participant, with no investigational treatments given, focusing on imaging and surgical assessments.

Researchers are evaluating whether sitagliptin, an investigational drug, can improve the immune response against glioblastoma by targeting myeloid-derived suppressor cells (MDSCs), which suppress the body's natural defense against cancer. Glioblastoma patients have increased MDSCs, and reducing these cells may help reverse immunosuppression. This phase 2 study focuses on patients with progressive grade 4 gliomas who are undergoing surgery and chemotherapy. The investigation builds on prior findings that MDSCs rely on an enzyme called DPP-4, which sitagliptin inhibits. Participants will be divided into two groups: one will receive presurgical sitagliptin followed by post-surgical sitagliptin and chemotherapy, and the other will start sitagliptin only after surgery along with chemotherapy. Sitagliptin is taken orally at doses ranging from 25 mg to 100 mg daily, self-administered by participants. The study includes a "window of opportunity" period before surgery to assess the drug's biological impact, followed by ongoing treatment until disease progression. During the study, participants will undergo tumor resection surgery and receive laboratory tests to monitor organ function and blood counts. Researchers will measure immune cell changes in tumor tissue, focusing on CD8+ T cells, and track progression-free and overall survival at specified intervals. Safety will be assessed by recording adverse events up to 30 days after treatment ends. The total duration of participation varies depending on individual treatment response and disease progression.

Diffuse gliomas are tumors affecting the brain and spinal cord, especially aggressive in people with mutations in the IDH1 or IDH2 genes. This trial evaluates zotiraciclib, a study drug being tested for effectiveness in people aged 15 and older with recurrent diffuse gliomas that have these gene mutations. The research includes a phase I/II design to assess safety, dosing, and how well the drug works compared to existing data. Participants will take zotiraciclib capsules by mouth on specific days (1, 4, 8, 11, 15, and 18) during 28-day treatment cycles, for up to 18 cycles. Dose levels may be adjusted based on tolerance, and some participants undergoing surgery will receive a single pre-treatment dose before tumor biopsy or removal. The study includes different groups, including dose escalation, recommended dose estimation, and a surgical cohort. Before joining, participants will have physical exams, blood and urine tests, heart function tests, and brain MRIs. They will keep medication diaries to track dosing. Clinic visits occur about once a month for exams and tests, with brain MRI scans every 8 weeks. Researchers will monitor progression-free survival over 12 months and evaluate safety and longer-term outcomes. Total participation may last up to 1.5 years or 18 treatment cycles.

Researchers are investigating the safety and effectiveness of Bevacizumab (BEV) with or without microbubble-mediated focused ultrasound (FUS) using the NaviFUS System in patients with recurrent glioblastoma multiforme (rGBM). This pivotal, randomized, open-label study compares standard care BEV alone to BEV combined with FUS in patients who have previously undergone surgery, radiotherapy, and chemotherapy with temozolomide. BEV is considered the best current treatment choice for these patients after prior therapies. Participants will be randomly assigned to one of two groups. One group will receive BEV alone via intravenous infusion at a dose of 10 mg/kg over 30-90 minutes every two weeks. The other group will receive the same BEV treatment followed by administration of microbubbles (SonoVue®) at 0.1 mL/kg and focused ultrasound exposure controlled by the NaviFUS System. Treatments will continue every two weeks for up to 34 weeks or until disease progression, intolerable side effects, non-compliance, or withdrawal. During the study, participants will be monitored through regular assessments including MRI scans, quality of life questionnaires, cognitive tests (Mini-Mental State Examination), and evaluation of corticosteroid use and adverse events. The primary outcome is progression-free survival at six months. Secondary outcomes include survival rates up to two years, tumor response, local disease control, and performance status. The total study participation can last up to 36 months with ongoing safety and efficacy monitoring.

Researchers are comparing two treatments, niraparib and temozolomide (TMZ), in adults with newly diagnosed glioblastoma multiforme (GBM) that has an unmethylated MGMT promoter. This Phase 3 trial aims to find out if niraparib can improve overall survival compared to the current standard treatment, TMZ. The trial will enroll 450 adult participants and is sponsored by the Ivy Brain Tumor Center. Participants will be randomly assigned to receive either niraparib or TMZ. Niraparib is taken orally once daily starting with radiation therapy (RT) for 6 to 7 weeks and continued as an adjuvant treatment until cancer progression or discontinuation. The TMZ group receives the drug daily during RT, followed by a rest period and then up to 6 cycles of adjuvant TMZ. Radiation therapy is given as standard care to 60 Gy in 30 fractions. During the study, participants will complete scheduled visits and keep a diary to record medication use. Researchers will monitor overall survival over 24 months as the primary outcome. Secondary measures include progression-free survival, response rates, quality of life assessments, neurocognitive function tests, and tracking of adverse events. Safety and treatment effects will be closely observed throughout the trial period, which continues until March 2028.

Researchers are evaluating the safety of avutometinib as a treatment for children and young adults aged 3 to 30 years with advanced or recurrent solid tumor cancers, including pediatric tumors with specific genetic alterations affecting the MAP kinase pathway and certain neurofibromatosis conditions. This phase 1 study aims to find the highest dose of avutometinib that is safe and causes few or mild side effects. Participants receive oral avutometinib twice a week, once daily, in cycles of three weeks on treatment followed by one week off, with each cycle lasting 28 days. The study uses a dose-escalation design, starting at Dose Level 1 and adjusting the dose based on the number of dose-limiting toxicities (DLTs) observed, to identify the maximum tolerated dose (MTD). During the study, participants will be closely monitored for safety over up to 12 months. Researchers will assess side effects and tolerability, and participants will undergo regular evaluations including clinical assessments and laboratory tests. The study carefully tracks adverse events and adjusts dosing to ensure participant safety throughout the trial.