Neuroendocrine disorder

This large Phase 3 trial tests whether VE303, a capsule containing 8 harmless bacteria, can prevent repeat Clostridioides difficile (C. diff) infections. About 852 people who have had at least one prior C. diff infection will take VE303 or a placebo for 14 days. The main goal is …

This phase II trial is testing whether giving a lower dose of radiation after chemotherapy can effectively treat children and young adults with central nervous system germinomas, a type of brain tumor. The study will enroll 240 participants aged 3 to 29. After initial chemotherap…

This early-phase study tests whether adding chemotherapy directly into the spinal fluid (intrathecal topotecan) followed by maintenance pills is feasible for children under 6 with high-risk embryonal brain tumors. About 15 children will receive this treatment after standard high-…

This phase 1 trial tests a drug called 177Lu-DTPA-omburtamab, which delivers radiation directly to tumor cells in children and teens with recurrent or refractory medulloblastoma or other central nervous system (CNS) tumors that have spread. The drug targets a protein called B7-H3…

This early-stage trial tests whether personalized immune cells (T-cells) can safely treat young children with aggressive brain tumors. The T-cells are designed to attack unique markers found on each child's tumor. Up to 12 children under 5 with embryonal brain tumors or recurrent…

This phase 2 trial tests a personalized approach for children and young adults with a rare brain tumor called non-germinomatous germ cell tumor (NGGCT). Patients first receive chemotherapy, and then the type and amount of radiation they get depends on how well the tumor responds.…

This early-phase trial is testing a combination of three treatments for children whose brain tumors have come back or not responded to standard care. The experimental drug SGT-53 delivers a normal TP53 gene to tumor cells, and is given alongside the immunotherapy Nivolumab and ra…

This early-phase study tests whether a new type of immunotherapy, called B7-H3 CAR T cells, can be safely given directly into the brain or spinal fluid of children and young adults with aggressive brain tumors like DIPG, DMG, or other recurrent central nervous system cancers. The…

This study tests a new way to treat children with gut-brain disorders like cyclic vomiting and functional dyspepsia. The therapy uses a mild electrical nerve stimulation to the ear, personalized based on each child's nerve activity. The goal is to improve quality of life and redu…

This early-phase trial tests a new treatment for children and young adults with medulloblastoma or similar brain tumors that have come back or not responded to standard therapy. The treatment uses the patient's own immune cells, modified to recognize and attack tumor cells, deliv…

This early-stage study tests an oral drug called ONC206 in adults with brain tumors that have come back. The main goal is to find the safest dose and check for side effects. About 102 participants will take the drug once or several times a week.

This early-phase trial is testing whether a single dose of a hormone pill can help diagnose oxytocin deficiency in young people with hypopituitarism. Researchers will give the pill to 20 participants and measure oxytocin-related substances in blood and saliva. The goal is to deve…

This study compares a new diagnostic test using mannitol infusion to the standard hypertonic saline test for diagnosing the cause of polyuria-polydipsia syndrome (excessive urination and thirst). Researchers will enroll 144 adults to see if the mannitol test is as accurate and mo…

This pilot study is testing whether an oxytocin nasal spray can help with anxiety, depression, and social functioning in adults with arginine-vasopressin deficiency, a rare hormone disorder. In the first part, 30 participants will receive single doses of oxytocin or placebo and c…

This study tests whether a nasal spray containing oxytocin can help reduce anxiety and improve emotional well-being in adults with central diabetes insipidus, a condition where the body lacks a key hormone. Participants will receive either oxytocin or a placebo spray for several …

This study tests a small device worn on the ear that stimulates the vagus nerve, which helps control nausea and vomiting. Researchers want to see if it can make cyclic vomiting episodes less frequent, shorter, and less severe in 120 children. Half the kids will get an active devi…

This phase 2 trial tests whether oxytocin nasal spray can improve sexual well-being in people with central diabetes insipidus, a condition that also causes oxytocin deficiency. The study includes 42 participants (patients and healthy controls) and uses a double-blind, placebo-con…

This study uses a non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) to compare brain activity in people with cyclic vomiting syndrome (CVS) and healthy volunteers. The goal is to see if there are differences in cortical excitability—how easil…

This study is testing whether taking estrogen pills (estradiol valerate or ethinylestradiol) can safely trigger the release of oxytocin, a hormone important for social bonding and childbirth. Researchers will measure oxytocin levels in 28 healthy volunteers and patients with AVP …

This study looks at how radiation to the brain affects memory in children who had a brain tumor. Researchers will test memory and thinking skills in 130 children ages 4 to 12, starting 2 years after radiation and again 2 years later. The goal is to learn which parts of the brain …

This study looks at whether measuring a substance called copeptin in the blood can help doctors diagnose central diabetes insipidus (CDI) in children after brain surgery. CDI causes extreme thirst and frequent urination due to a hormone imbalance. The study will track copeptin le…

This study tests whether music enhanced with sonic augmentation technology can increase oxytocin (a hormone linked to social bonding) with few side effects. Researchers will compare the modified music to regular music in 22 healthy adults and patients with AVP-deficiency. The goa…

This study is following 2000 adults with functional digestive disorders like irritable bowel syndrome, dyspepsia, and constipation. Researchers will collect medical data and biological samples (biopsies, blood, urine, stool) during routine care. The goal is to find markers that c…

This study is collecting data from 1,650 people with various brain tumors to see if the updated 2021 WHO classification system helps doctors group patients more accurately and choose better treatments. Researchers will track responses to therapy, survival, and tumor features over…

This international registry collects medical data and biological samples from children and adults with rare brain tumors like medulloblastoma and ependymoma. By gathering information from patients not in clinical trials, researchers hope to improve treatment recommendations and i…