Chordoma

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.

Spinal cord gliomas are rare malignant tumors that develop within the spinal cord, often appearing around age 35 and slightly more common in males. These tumors typically cause symptoms like axial pain and tend to grow in a lateral, infiltrative pattern. Treatments are limited mainly to surgery, and the choice of surgical approach can affect neurological outcomes and quality of life. This research compares two surgical methods to find the best approach for tumor removal and patient recovery. The study compares the dorsolateral sulcus (DLS) approach and the traditional posterior median sulcus (PMS) approach for spinal cord tumor removal. The DLS approach is thought to better preserve neurological function by avoiding critical midline structures, while the PMS approach offers direct midline access but risks more damage. Patients with laterally located tumors, confirmed by imaging and pathology, undergo surgery via one of these two methods. Outcomes will be assessed over time to evaluate pain, neurological function, quality of life, tumor removal extent, and survival. Participants will be evaluated before surgery and followed up at 1, 3, 6, and 12 months after surgery with clinical exams, imaging, and questionnaires like the Visual Analog Scale for pain and SF-36 for quality of life. Neurological function is measured using established grading scales, and tumor progression and survival are monitored up to 24 and 36 months, respectively. The study aims to determine which surgical approach best balances effective tumor removal with preservation of neurological health.

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.

Researchers are investigating a new treatment approach for pediatric and young adult patients with relapsed or refractory solid tumors. The study focuses on using allogeneic stem cell transplantation that removes alpha/beta T cells and CD19+ B cells to reduce complications like graft-versus-host disease and improve outcomes. This approach is combined with zoledronic acid to potentially enhance the immune system's ability to fight tumors and improve survival rates. Participants will receive a stem cell transplant processed with the Miltenyi CliniMACS Prodigy® system to remove specific immune cells. Zoledronic acid will be given intravenously on days 28, 56, 84, 112, and 140 after transplant. The study includes a phase Ib dose-finding segment and a phase II segment to evaluate the safest and most effective dose of zoledronic acid. During the study, participants will be closely monitored for disease-free survival at 1 year post-transplant and the incidence of acute graft-versus-host disease over 2 years. Additional survival rates will be assessed at 100 days, 1 year, and 2 years. Assessments include clinical evaluations and safety monitoring over several years, with the total participation lasting until 2 years post-transplant to evaluate long-term effects and outcomes.

Researchers are creating a secure electronic health record (EHR)-based registry to collect clinical and research data on rare diseases, specifically bone and soft tissue tumors. This observational study aims to help identify clinical and molecular features related to prognosis and therapy response, encourage collaboration between institutions, and standardize biospecimen collection for biomarker analysis in patients treated and monitored at Ohio State University. Participants will not receive experimental treatments but will be observed as part of routine care. They will complete quality of life questionnaires, and their medical records will be reviewed. Blood samples may be collected for up to two years to support biomarker research. The study facilitates long-term data collection and collaboration to better understand these rare tumors. During the study, participants will provide information through questionnaires and allow access to their medical records. Researchers will monitor outcomes such as overall survival over five years. The study includes ongoing collection of clinical data and biospecimens to support research. Participation duration varies, and there is no interventional treatment provided as this is an observational registry.

Researchers are evaluating proton therapy in patients with chordoma, chondrosarcoma, Ewing sarcoma, and osteosarcoma who require two radiation beams for treatment. This study aims to compare the toxicity of proton therapy delivered using a single daily beam versus the conventional approach of two daily beams. The latest Proteus®One proton therapy equipment with a compact rotating gantry enables precise targeting of tumors from different angles, potentially reducing treatment side effects. Participants are randomly assigned to receive either one daily beam, which delivers a doubled dose in each session, or two daily beams delivered according to the standard treatment plan. Both beams are conventionally delivered in the same session but require repositioning the patient before each beam, which takes extra time. This study evaluates whether delivering only one beam daily can maintain treatment effectiveness while reducing toxicity and saving time over the 2 to 7 week treatment period. During the treatment and up to five years of follow-up, patients will be monitored for side effects, quality of life, and treatment effectiveness. Researchers will measure the frequency of toxicities and assess quality of life throughout treatment and follow-up. They will also evaluate the time saved by using a single daily beam. The total participant involvement includes initial treatment and long-term follow-up visits to assess outcomes and safety.

Researchers are investigating the wide range of molecular features found in people receiving care within a large community healthcare system who are at risk of or diagnosed with cancer. The study aims to understand the genetic factors behind premalignant and malignant conditions across various cancer types and stages. This research helps advance knowledge of cancer biology and supports the discovery of biomarkers linked to clinical outcomes. Participants will undergo comprehensive molecular profiling, including somatic tumor testing from tissue and/or blood samples using next-generation sequencing. Some samples may also receive whole exome or transcriptome sequencing for research purposes. Pharmacogenomic testing will help explore how individuals respond differently to medications, and participants may optionally provide microbiome samples. Participants may also consent to store biological samples in a biobank and allow their de-identified data to be used for future research. During the study, electronic health records will be reviewed both retrospectively and prospectively to connect clinical data with genomic findings. Researchers will measure how many patients undergo molecular profiling, are referred for genetic testing or targeted clinical trials, and have therapy changes based on molecular or pharmacogenomic results. The study spans five years, with long-term follow-up and data collection to support ongoing research and collaboration in cancer studies.

Chordoma is a rare type of bone cancer that occurs in the skull base or spine. Researchers want to better understand chordoma by studying its clinical course, appearance on imaging scans, and how it responds to different therapies. This natural history study focuses on children and adults with chordoma to help design improved treatments and supportive care in the future. Participants must have a confirmed diagnosis of chordoma and be enrolled in the NCI protocol 19-C-0016 for rare solid tumors. The study involves documenting medical histories and following participants over time to observe disease progression, patterns of recurrence, responses to therapies, and tumor growth rates. This study does not involve new treatments but collects detailed clinical, epidemiologic, and biological data. During the study, participants have visits to assess their disease, including physical and neurologic exams, CT scans, and MRIs. Every 6 to 12 months, participants complete tests such as smell tests, surveys about their emotional and physical well-being, and cognitive function assessments. Researchers also gather ongoing information about disease status from participants or their doctors. Some participants may be asked to attend additional clinic visits. The main outcome measured is the natural history of chordoma over 10 years, focusing on clinical presentation and disease progression.

This research aims to explore the factors that influence decision making for patients with new or recurrent central nervous system (CNS) tumors and their families. The focus is on understanding challenges related to treatment planning and enrolling in clinical trials. The study involves patients of various ages, including children, young adults, and their caregivers, to gather diverse perspectives on these decisions. Participants will take part in qualitative interviews designed to identify specific barriers to treatment and trial enrollment, such as transportation, childcare, and other related expenses. For children 12 years old and younger, only caregivers will be interviewed. Patients aged 13 to 17 may participate along with their caregivers if they choose, while patients 18 years and older can include their caregivers in interviews. The study will assess the impact of community and hospital resources on decision making through follow-up interviews. Throughout the study, participants will engage in interviews that are both structured and open-ended to fully capture their experiences and challenges. Researchers will measure how these resources affect treatment decisions and will identify barriers to trial enrollment within 12 months. The study aims to improve support for patients and families facing CNS tumor treatment decisions, with total participation lasting as needed for follow-up assessments.

Researchers are evaluating three treatment approaches—carbon ion therapy, surgery (with or without photon/proton therapy), and proton therapy alone—for patients with pelvic bone sarcomas. The study aims to compare how these treatments affect disease control and quality of life, focusing on whether carbon ion therapy offers better patient-reported health outcomes and fewer side effects compared to surgery, as well as improved local control compared to proton therapy. Participants receive one of the three treatments and complete quality of life questionnaires at several points: before any therapy, between 2 to 4 months, and again at 5 to 9 months after treatment completion, followed by annual assessments for up to five years. Researchers also review patients' medical records to gather detailed clinical data. This observational study involves collecting patient-reported outcomes and medical information without assigning treatments. Throughout the study, patients are asked to complete questionnaires taking about 20 minutes to assess their health-related quality of life. These assessments occur at baseline and various follow-up points up to five years after treatment. Researchers monitor disease progression, local control of the sarcoma, survival outcomes, and treatment-related toxicities. The study's follow-up and data collection span several years to better understand long-term effects and outcomes of the different treatment approaches.