Neuroblastoma

The LuDO-N Trial is a phase II multi-center clinical study investigating the use of 177Lu-DOTATATE in children with recurrent or relapsed high-risk neuroblastoma. This trial aims to assess how well this treatment works by measuring the response at 1 and 4 months after treatment ends. Secondary goals include evaluating survival rates, treatment-related side effects, and examining how tumor characteristics relate to treatment response. Participants receive two doses of 177Lu-DOTATATE administered intravenously, with at least two weeks between doses. The first dose is based on the patients weight, while the second dose is adjusted using scans to control radiation exposure, particularly to avoid kidney toxicity. Patients must be prepared for autologous stem cell transplantation as part of this intensified treatment approach. During the study, participants will undergo various scans and laboratory tests before starting treatment and at scheduled times to monitor tumor response and side effects. Researchers will assess treatment effectiveness using established neuroblastoma response criteria and will follow patients for up to five years to monitor survival and any long-term effects. The total participation time includes treatment administration and extended follow-up for safety and outcomes.

Researchers are evaluating the optimization of 18F-DOPA PETCT 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 Parkinsons disease or Lewy body dementia. The study aims to improve image quality using a new digital PETCT 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 PETCT 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 use of the radiopharmaceutical 18F-mFBG in PETCT imaging compared to the established 123I-mIBG scintigraphy SPECTCT scans for children with neuroblastoma. The study aims to assess how well 18F-mFBG can identify neuroblastoma lesions and compare its diagnostic performance with that of 123I-mIBG imaging. Participants include children with either a confirmed or presumed diagnosis of neuroblastoma based on clinical, imaging, and laboratory findings. Participants will undergo both 18F-mFBG PETCT and 123I-mIBG scintigraphy scans within a 7-day window, without receiving chemotherapy, immunotherapy, or radiotherapy between these imaging procedures. The 18F-mFBG imaging is performed using a large field of view total body PETCT scanner. Two independent teams, each consisting of a nuclear medicine physician and a pediatric radiologist, will review both imaging types while blinded to results. Discrepancies in lesion detection between the two scans will be reviewed by an expert panel. During the study, participants will have two diagnostic imaging sessions spaced closely in time. The study will evaluate lesion detection and imaging consistency through expert blinded readings. The primary outcome focuses on the ability of 18F-mFBG PET to identify neuroblastoma lesions within 60 minutes. Secondary outcomes compare the 18F-mFBG results with the clinical 123I-mIBG scans performed within seven days. The study involves informed consent and careful monitoring but does not involve treatment interventions.

This research aims to evaluate the use of 18F-metafluorobenzylguanidine 18F-MFBG positron emission tomography PET in diagnosing neuroblastoma, a cancer that often expresses norepinephrine transporter NET. The current imaging methods using radioactive iodine-labeled compounds have limitations such as low resolution, long procedures, and difficulty detecting small lesions. This study will look at the safety, image quality, and diagnostic ability of 18F-MFBG, which can be used shortly after injection and shows promise based on preliminary data. Participants will receive a single intravenous dose of 18F-MFBG at 2-5 MBqkg. After 60 minutes, patients will undergo a PETCT or PETMR scan to visualize the tumor. This approach allows for quicker imaging compared to prior methods and aims to better detect neuroblastoma lesions. The study will compare this imaging techniques performance with other images and assess its value for predicting progression-free survival in high-risk patients. During the study, patients will be monitored for diagnostic efficacy over an average of 1.5 years, with secondary outcomes tracked for up to 3-4 years in some cases. Assessments include image quality, tumor burden evaluation, and safety observations. Participants will be involved in the injection and scanning procedures, with follow-up to assess prognosis and imaging performance. The total participation duration varies depending on outcome tracking needs.

Researchers are studying the use of 18F-metafluorobenzylguanidine 18F-MFBG positron emission tomography PET to evaluate neuroendocrine tumors, mainly pheochromocytoma, paraganglioma PPGL, and neuroblastoma NB. These tumors express norepinephrine transporter NET, which is targeted by imaging substances like 123I131I-MIBG. However, current imaging methods have limitations such as low resolution and long waiting times, which affect diagnosis. This study aims to assess the safety, image quality, diagnostic accuracy, and tumor burden detection of 18F-MFBG PET in these neural crest tumors.

Researchers are evaluating a new 4SCAR-T cell therapy targeting three surface antigens GD2, PSMA, and CD276 in patients with relapsed or refractory neuroblastoma NB, a severe childhood tumor with poor survival despite current treatments. This study aims to assess the feasibility, safety, and effectiveness of this multi-targeted CAR-T cell approach. The research also seeks to understand how these modified T cells function and persist in patients over time. The investigational treatment involves gene-modified T cells designed to recognize and kill tumor cells expressing GD2, PSMA, and CD276. Each patients tumor will be tested to identify which antigens are highly expressed, allowing a personalized combination of two or three targets for the 4SCAR-T therapy. The CAR-T cells are infused intravenously at doses ranging from 1x106 to 1x107 cells per kilogram of body weight. This approach is intended to overcome the limited success of single-target CAR-T treatments in neuroblastoma. Participants will undergo evaluations to measure side effects, anti-tumor effects, CAR-T cell expansion, cytokine secretion, and survival over a period of up to three years. Tumor antigen expression is determined before treatment using immunohistochemistry or flow cytometry. Researchers will monitor patient health through physical exams, cardiac and liver function tests, blood counts, and oxygen levels. The study involves ongoing safety assessments and effectiveness monitoring to better understand treatment impact and patient outcomes.

Researchers are evaluating the use of Gallium-68-DOTATATE PETMRI 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.

Neuroblastoma is the most common extracranial tumor in children, occurring at a rate of about 10.2 cases per million yearly. This study is a phase II pilot trial designed to assess the use of a new imaging method, 68Ga-DOTATATE PETCT, for detecting neuroblastoma and compare it to the existing 123I-MIBG scan. Researchers aim to evaluate the feasibility and safety of 68Ga-DOTATATE and how accurately it identifies neuroblastoma lesions compared to 123I-MIBG. Participants will first undergo a 123I-MIBG scan followed by a 68Ga-DOTATATE PETCT scan a few days later. The 68Ga-DOTATATE is a radiopharmaceutical injected into the patient, and imaging is done approximately one hour post-injection, lasting about two hours. This single-arm study includes children and young adults up to 21 years old who have biopsy-proven or suspected neuroblastoma. The study monitors adverse events within 24 hours after the 68Ga-DOTATATE injection. During the study, researchers will collect clinical data from the imaging scans and participants medical records for up to two years, including demographics, treatments, medications, pathology, and laboratory tests. Outcome measurements focus on the rate of participant enrollment and any side effects from the 68Ga-DOTATATE injection, as well as lesion detection and differences between the two imaging methods. Participants health status will be followed regularly to assess the imaging techniques effectiveness and safety.

Researchers are studying tumors that have somatostatin receptors SSTRs on their surface, which include tumors in the lung, head and neck, digestive tract, kidneys, and adrenal glands. This trial tests a drug called 212PbVMT-Alpha-NET, which is designed to target these receptors and potentially help shrink tumors that have spread and cannot be removed surgically. The study focuses on people aged 18 and older with these specific types of tumors and aims to determine the maximum tolerated dose and safety of the drug. Participants receive 212PbVMT-Alpha-NET through a vein on the first day of four 8-week cycles. Some participants will also receive a related imaging drug, 203PbVMT-Alpha-NET, a few days before the first two treatment cycles to allow whole-body scans that track how the drug spreads in the body. After each infusion, participants stay in the hospital for a few nights and have weekly blood tests during each treatment cycle. The study includes dose escalation to find the best dose and then dose expansion at that dose level. Throughout the study, participants undergo physical exams, blood and urine tests, imaging scans including PETCT and SPECTCT, heart function tests, and may provide tumor tissue samples if needed. Follow-up visits occur about 30 days after treatment ends, then every 12 weeks for up to 3 years, with annual check-ins continuing up to 6 years after the last treatment. Researchers monitor safety, response to treatment, and survival outcomes during this time.

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 childrens quality of life, fatigue related to cancer, and parents perceptions of their childs 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.