Neuroblastic Disorder

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 patient's 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 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 use of the radiopharmaceutical 18F-mFBG in PET/CT imaging compared to the established 123I-mIBG scintigraphy SPECT/CT 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 PET/CT 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 PET/CT 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 MBq/kg. After 60 minutes, patients will undergo a PET/CT or PET/MR 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 technique's 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 123I/131I-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.

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 PET/CT, 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 PET/CT 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.

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.

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 observing patients with high-risk neuroblastoma, a common childhood cancer typically diagnosed under age 5, to further evaluate the safety and effects of dinutuximab beta, a monoclonal antibody treatment. This study aims to gather detailed information on survival, pain levels, and the occurrence of side effects such as neurotoxicity, visual problems, capillary leak syndrome, heart events, and allergic reactions. It is a non-interventional, multinational registry designed to follow patients over time. The study collects information from patients being treated with dinutuximab beta either in standard clinical practice or as part of clinical trials where the drug is authorized. Data on treatment dosing, pain management, and side effects are gathered during up to five treatment cycles, each lasting 35 days. Researchers will also monitor long-term safety and patient outcomes through follow-up visits after treatment completion. Participants, who are children aged 1 to 18 years with high-risk neuroblastoma, will have their medical data recorded at baseline, during treatment, and at follow-up visits. This includes assessments of pain, side effects, disease progression, and survival. Data is collected electronically from medical records, and patients will be followed for up to 10 years to provide comprehensive safety and effectiveness information.

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.