Medulloblastoma

Researchers are evaluating the use of 18F-Fluciclovine positron emission tomography (PET) as a biomarker to measure the response or progression of brain metastasis in participants treated with stereotactic radiosurgery (SRS). This pilot imaging study focuses on adults diagnosed with brain metastases who are planned to receive SRS treatment. The study is sponsored by Baptist Health South Florida and is a Phase 1 clinical trial. Participants will undergo an 18F-fluciclovine PET scan at the time of their SRS planning magnetic resonance imaging (MRI). They will then receive a single dose of SRS as part of their standard care. A second 18F-fluciclovine PET scan will be performed approximately 8 weeks after the SRS treatment, with a window of plus or minus 2 weeks. During PET scans, participants receive a 5-mCi dose of 18F-fluciclovine through intravenous injection, with imaging data collected up to 25 minutes post-injection. Throughout the study, participants will be monitored using PET imaging to evaluate changes in standardized uptake values (SUV) over 8 weeks, which helps assess tumor response. Tumor control will also be evaluated at 12 months. The study involves standard imaging, including MRI and PET scans, and tracks participant safety and treatment progress. Total participation timelines include initial scans at SRS planning and follow-up imaging about 8 weeks later, with tumor control assessed after one year.

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 the safety and effectiveness of a modified herpes simplex virus called recombinant oncolytic herpes simplex virus type 1 (R130) in patients with advanced solid tumors. This early phase 1, open, single-arm clinical trial aims to study the treatment in people with various cancers such as sarcoma, carcinoma, digestive cancer, breast cancer, lung cancer, brain cancer, melanoma, gynecologic cancer, head and neck cancer, and kidney cancer. The study focuses on patients who have not responded to standard treatments or who choose not to receive other antitumor therapies. Participants will receive injections of 1 to 2 milliliters of R130 at a concentration of 1x10^8 plaque-forming units per milliliter into their tumors or abdominal cavity every 7 to 14 days. This approach allows the virus to be delivered directly to the cancer site. The study involves only one treatment group receiving the R130 virus, and no placebo or comparison group is used. During the trial, researchers will monitor participants for adverse events and laboratory abnormalities up to 6 months and assess their immune response. Disease control and response duration will be evaluated every 10 weeks for up to 12 months, while quality of life assessments will occur every 6 weeks for the same period. Participants will undergo regular laboratory tests and clinical evaluations to track safety and treatment impact. The total study duration for each participant may extend up to one year with ongoing monitoring.

Many children with cancer experience emotional distress, fatigue, and difficulties in relationships. Their parents also face increased responsibilities and may feel more distressed and tired. While psychological interventions for these families have shown promise in improving social skills, coping, and well-being, further research is needed. Hypnosis is commonly used in pediatric oncology to reduce pain and distress during procedures and has also been effective in enhancing well-being in adults with cancer. This trial explores the feasibility and potential benefits of combining self-care and hypnosis in a group setting for children with cancer and their parents. The intervention involves six monthly group sessions, each lasting two hours, where participants learn self-hypnosis exercises and discuss self-care techniques like understanding personal needs, self-respect, assertiveness, and managing negative thoughts. Homework assignments are given to encourage positive changes. Two groups participate: one including children with cancer and their siblings, and another for their parents. Data are collected before and after the intervention through questionnaires and interviews to assess its impact. Participants will complete assessments measuring changes in children's quality of life, fatigue related to cancer, and parents' perceptions of their child's quality of life and their own fatigue. Secondary outcomes include the impact of cancer on the family, parents' emotional distress, and coping strategies. These are evaluated before the program starts and immediately after its conclusion at six months. The study aims to improve understanding of how this combined self-care and hypnosis intervention may enhance the well-being of children with cancer and their families.

Sleep plays a vital role in a child's development, affecting brain function, emotional health, and overall recovery. Children undergoing intensive cancer treatments often experience sleep problems such as difficulty falling or staying asleep, shorter sleep duration, or poor sleep quality. These issues, reported in a significant portion of pediatric cancer survivors, can impact treatment adherence, daily life, and social interactions, highlighting the need for better sleep management in this group. Researchers are evaluating the Dreamcatchers Programme, a nurse-led, multi-component intervention designed to improve sleep quality in children with cancer. The program involves sleep hygiene education, progressive muscle relaxation (PMR), and breathing exercises, delivered through group sessions and weekly follow-ups over four weeks. The intervention group receives these targeted strategies, while the control group continues routine hospital support without sleep-specific content, with access to the program after the study. Participants will attend initial education sessions, practice relaxation techniques, and keep sleep diaries to track habits and progress. Nurses will monitor sleep quality and overall life quality at three months using validated tools. Data will be collected securely and confidentiality maintained. This pilot study aims to assess feasibility and provide preliminary effectiveness results to guide future pediatric oncology sleep care.

Researchers are evaluating the use of the drug SGT-53 in combination with radiation therapy and the drug Nivolumab in children with brain tumors that have returned, worsened, or not improved after previous treatments. This early-phase clinical trial aims to find the right dose of SGT-53 that can be safely given alongside radiation and Nivolumab. The study also investigates the side effects of SGT-53, how the drug moves through the body, and how much of it reaches the tumor tissue in a small group of patients. During the first treatment cycle, participants receive SGT-53 twice a week, Nivolumab every two weeks, and radiation therapy during the second week. In cycles two through six, SGT-53 is given once a week during even cycles and twice a week during odd cycles, while Nivolumab continues every two weeks. From cycle seven onward, both SGT-53 and Nivolumab are given every two weeks. In a special early part of the study, four participants will have genetic testing on tumor samples after receiving SGT-53 to compare with skin samples. Participants will be closely monitored throughout the study with evaluations of side effects related to SGT-53 from enrollment to three years after treatment ends. Researchers will assess the drug's safety, how it is processed in the body, and its presence in tumor tissue. The study includes clinical and radiological assessments, genetic testing, and ongoing safety monitoring to understand the treatment's effects over time.

Researchers are evaluating the safety, tolerability, and efficacy of CBA-1205, an anti-DLK1 monoclonal antibody, in patients with advanced solid tumors including hepatocellular carcinoma, malignant melanoma, and certain pediatric cancers. This first-in-human, Phase I, multi-center, non-randomized, open-label study is designed in five parts to assess these outcomes in different patient groups, especially those who have no standard treatment options or are intolerant or non-responsive to existing therapies. The study treatment involves intravenous administration of CBA-1205 at varying doses depending on the study part. In Part 1, doses from 0.1 to 30 mg/kg are given every two weeks in 28-day cycles to patients with solid tumors. Parts 2 through 5 administer doses ranging from 10 to 30 mg/kg every two weeks in similar 28-day cycles to patients with hepatocellular carcinoma, malignant melanoma, and pediatric cancers, continuing until criteria for treatment discontinuation are met. Participants will undergo safety and tolerability evaluations, including monitoring for dose-limiting toxicity during the first 28 days and adverse events up to 12 months. Pharmacokinetic and immunogenicity analyses will be conducted from Day 1 to Day 43 or until treatment discontinuation. Efficacy will be assessed at screening and regular intervals during treatment. The study includes detailed laboratory tests and performance status assessments, with the total participation duration varying by individual response and treatment continuation.

Researchers are evaluating the SYSUCC-RMS regimen for children with rhabdomyosarcoma, a type of pediatric cancer. This study aims to understand how effective and safe this treatment is, while also exploring how combining radiotherapy with chemotherapy affects survival rates across different risk groups: low, medium, high, and very high risk. The study is a Phase 3 clinical trial led by Yizhuo Zhang. Participants receive one of several combination chemotherapy regimens based on their risk level: low risk patients receive VAC, intermediate risk patients receive VAC/VII, high risk patients receive CAV/IE, and very high risk patients receive CAV/VIP. The study follows a single-arm design and evaluates the treatments over various risk groups, focusing on the impact of concurrent therapies on survival. During the study, researchers will monitor participants for up to 10 years, assessing outcomes such as objective response rate, disease control rate, progression-free survival, and overall survival. Safety of the SYSUCC-RMS regimen will also be tracked throughout this period. Participants’ health status will be regularly evaluated to understand the long-term effects and treatment outcomes.

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 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.