Adrenal gland disorder

This early-stage trial tests whether a single infusion of donated mesenchymal stem cells is safe for children with cerebral adrenoleukodystrophy (cALD), a rare and life-threatening brain disease. The stem cells are given as a temporary bridge to buy time before a more definitive …

This phase 2 trial is testing a combination of two immunotherapy drugs, nivolumab and ipilimumab, in 798 people with over 50 types of rare cancers. The goal is to see if these drugs can shrink tumors by helping the immune system attack cancer cells. Participants receive the drugs…

This clinical trial is testing whether dimethyl fumarate, a drug already used for multiple sclerosis, can improve balance and walking in adults with adrenomyeloneuropathy (AMN), a rare nerve disease. Forty participants will take either the drug or a placebo daily for 36 months, w…

This study looks at how the drug crinecerfont works in babies under 2 years old with congenital adrenal hyperplasia (CAH), a condition where the body can't make certain hormones properly. The goal is to measure drug levels in the blood and check for side effects. Only 7 infants a…

This phase 2 trial tests the drug cabozantinib in children and young adults with rare cancers like sarcomas, Wilms tumor, and others that have come back or not responded to treatment. Cabozantinib works by blocking enzymes that help tumors grow and form blood vessels. The study a…

This study tests whether adding cabergoline to osilodrostat works better than osilodrostat alone for controlling Cushing's disease. About 50 adults with active Cushing's disease will receive either the combination or the single drug. The goal is to see if the combo can lower cort…

This study tests whether adding olaparib to the standard chemotherapy temozolomide helps control advanced pheochromocytoma or paraganglioma—rare neuroendocrine tumors that have spread or can't be removed surgically. About 46 adults with these cancers will be randomly assigned to …

This study tests a drug called crinecerfont in adults with classic congenital adrenal hyperplasia (CAH), a genetic condition that requires lifelong steroid treatment. The goal is to see if crinecerfont can safely reduce the daily steroid dose needed while keeping hormone levels u…

This phase 2 trial tests whether giving heated chemotherapy directly into the abdomen during surgery can help people with a rare and aggressive adrenal cancer that has spread within the belly. About 30 participants will have surgery to remove visible tumors, followed by a wash of…

This study tests whether the drug eplerenone works better than amlodipine for controlling high blood pressure in Black adults who carry a specific gene variant (LSD1 risk allele). About 300 participants will follow a controlled diet and take escalating doses of one of the two dru…

This study looks at the long-term safety of relacorilant, a drug for people with Cushing syndrome. It includes 125 adults who previously completed a relacorilant study and may benefit from continued treatment. Researchers will track side effects over time to see if the drug is sa…

This study follows 200 adults in Japan with severe growth hormone deficiency who are taking Sogroya as part of their normal care. Researchers will monitor side effects and changes in body composition over 2 to 5 years. Participants will also fill out quality-of-life questionnaire…

This study looks at whether injecting special stem-like cells called Muse cells directly into the ovaries can help women aged 28 to 70 who are in perimenopause. The cells may repair tissue, reduce inflammation, and improve hormone balance. Researchers will track safety, hormone l…

This phase II trial tests the immunotherapy drug pembrolizumab in 39 people with advanced adrenocortical carcinoma that cannot be removed by surgery. The study aims to see if the drug can shrink or control the cancer. Participants receive pembrolizumab and are monitored for tumor…

This phase 2 study tests two drugs, sunitinib and temsirolimus, in 137 people with advanced rare tumors that have no cure. The goal is to see if these drugs can shrink tumors or slow their growth. Participants take one of the two drugs, and researchers measure how long the cancer…

This Phase II trial tests whether combining lenvatinib and everolimus can shrink or slow advanced carcinoid tumors that cannot be removed by surgery. The study enrolls 36 adults with confirmed unresectable carcinoid tumors. Both drugs are taken orally and work by blocking enzymes…

This study tests a drug called lanreotide in 10 people with advanced or metastatic paraganglioma or pheochromocytoma, rare tumors that release hormones. The goal is to see if lanreotide can slow tumor growth compared to before treatment. Participants receive injections every 4 we…

This phase II trial tests the drug axitinib in 6 adults with metastatic or inoperable pheochromocytoma or paraganglioma, rare tumors of the adrenal glands or nerve tissue. Axitinib works by blocking blood vessel growth that feeds tumors. The main goal is to see how many patients'…

This early-phase study tests a new drug called E7386 in about 70 people with advanced solid tumors, including colorectal cancer. The main goal is to check safety and find the right dose. Participants have tumors that no longer respond to standard treatments.

This study tested a drug called pembrolizumab in 157 people with rare cancers that had spread or couldn't be removed. The drug works by helping the body's immune system attack the cancer. The main goal was to see how many patients were alive and their cancer hadn't grown after 27…

This phase 2 trial tests two drugs, pembrolizumab and lenvatinib, in 30 adults with advanced adrenal cortical carcinoma that has worsened after standard chemotherapy. The goal is to see if the combination can shrink or slow the cancer. Participants receive the drugs intravenously…

This study tests whether combining two immunotherapy drugs, nivolumab and ipilimumab, can shrink tumors in people with rare cancers of the urinary or reproductive systems. About 100 adults with advanced disease that cannot be removed by surgery are taking part. The main goal is t…

This early-phase trial tests a gene therapy called BBP-631 for classic congenital adrenal hyperplasia (CAH), a genetic condition that forces patients to take daily steroids. The therapy uses a harmless virus to deliver a working copy of the faulty gene. Only 8 adults are enrolled…

This study tests a new device called the Nectero EAST System in 46 people with abdominal aortic aneurysms (AAA). The goal is to see if it is safe and can stop the aneurysm from growing, reducing the need for major surgery. Participants receive the device through a catheter, and d…

This study follows 206 patients with Cushing's syndrome who are taking osilodrostat (Isturisa) to see how safe and effective it is over time. Researchers will track side effects and how well the drug controls cortisol levels. No new treatment is given—just real-world monitoring.…

This study tests whether eating wild blueberry powder every day can lower blood pressure and improve blood vessel health in middle-aged and older men and postmenopausal women with slightly high blood pressure. Researchers will measure artery function and stiffness over time. The …

This phase 2 trial is testing whether a combination of two drugs, talazoparib and temozolomide, can shrink tumors in people with rare cancers that have spread. The study includes 14 participants with advanced rare solid tumors, such as pheochromocytoma or paraganglioma, who have …

This study follows children with growth hormone deficiency who are starting Ngenla for the first time. Researchers will monitor their growth and any side effects over several years, until November 2027. The goal is to understand the long-term safety and effectiveness of this dail…

This phase 2 trial tests whether metyrapone, a drug that lowers cortisol production, is safe and effective for people with mild autonomous cortisol secretion (MACS). About 30 participants will take the drug for a period, and researchers will track side effects and cortisol levels…

This phase 1 trial is testing a new imaging tracer called 68Ga-FAPI-46 to see where it collects in normal and cancerous tissues. About 30 people with various solid tumors or blood cancers will receive the tracer and then undergo a PET/CT scan. The goal is to understand how the tr…

This study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…

This early study tests a new radioactive tracer called 68Ga-FAPI-46, which targets a protein (FAP) found around many tumors. About 26 people with various cancers will get a PET/CT scan using this tracer to see where it goes in the body. The goal is to learn if this tracer can hel…

This study tests a new DNA-reading technology called ultra-long read sequencing to find hidden genetic changes that standard tests miss. Researchers will analyze blood samples from 15 patients with known or suspected genetic variants in difficult-to-read DNA regions. The goal is …

This study is testing whether whole body MRI scans can help find cancers early in children and adults with Li-Fraumeni syndrome, a genetic condition that greatly raises cancer risk. About 150 participants will receive annual whole body MRI scans for four years to see if they keep…

This study compares a new, non-invasive PET-MRI scan to the standard invasive test (IPSS) for finding pituitary tumors in people with Cushing's disease. About 43 adults with confirmed Cushing's will get both tests to see which is more accurate. The goal is to make diagnosis safer…

This study tests a special PET scan (11C-Metomidate) to better identify adrenal tumors that cause high blood pressure and can be cured with surgery. About 100 adults with suspected adrenal-related hypertension will receive the scan, which is non-invasive and may replace the curre…

This study aims to see if a simple, inexpensive saliva test can accurately diagnose Cushing's syndrome, a condition where the body makes too much cortisol. Researchers will compare two automated saliva test methods in 380 adults who are already being checked for the condition. Th…

This study explores whether ultrasound and PET/CT scans can more accurately diagnose and monitor polymyalgia rheumatica (PMR), a condition causing muscle pain and inflammation. Researchers will follow 98 adults over 50 suspected of having PMR, using imaging at key points during t…

This study is testing a new AI-based system called AIMAS that aims to assess motor skills in children aged 6 to 12. The goal is to see if the AI can reliably identify kids with developmental coordination disorder (DCD) compared to standard clinical methods. Researchers will enrol…

This study tests whether a short, modified exposure therapy given soon after rape can prevent post-traumatic stress disorder (PTSD) in women. About 166 women who have experienced sexual assault will be randomly assigned to either the brief therapy or standard care. The goal is to…

This study looks at whether pramipexole, a drug used for restless leg syndrome, can ease leg symptoms in women with X-linked adrenoleukodystrophy (ALD). About 24 women will take the drug or a placebo for two months. The goal is to see if it improves sleep, walking, and quality of…

This study tests a small device that delivers mild electrical stimulation to the perineum (the area between the scrotum and anus) to help men who leak urine after prostate removal surgery. Thirty men who are scheduled for robotic prostate surgery and already use at least one pad …

This study tests a 6-week group program called Working Out Dads (WOD) to see if it helps reduce mental health struggles in fathers of children aged 0-4. About 293 dads with mild to moderate distress will either join the WOD peer-support group or receive usual care. The goal is to…

This study was designed to look back at the health of people with Cushing's disease in Western France between 1990 and 2015. The goal was to find out how many patients went into remission and how many had their disease come back after their first surgery. However, the study was w…

This study follows 400 adults with growth hormone deficiency who are already taking Sogroya (somapacitan) as part of their normal care. Researchers will monitor side effects and effectiveness over 5 to 10 years. Participants will also fill out questionnaires about their well-bein…

This study follows 600 people with Albright hereditary osteodystrophy, a rare genetic condition affecting bones and hormones. Researchers are looking at how growth hormone treatment affects height and weight in those with a related hormone problem, and also studying thinking and …

This study aims to find genetic and metabolic markers that explain why some people develop diabetes, high blood pressure, or bone loss from glucocorticoids while others do not. Researchers will analyze blood and urine samples from up to 600 participants, including those with exce…

This study is not testing a new drug, but rather doing the early science needed to develop one. Researchers are looking for the right targets (antigens) on adrenocortical carcinoma cells and studying how the tumor suppresses the immune system. The goal is to find a way to create …

This study looks at whether a 25-minute hydration education session plus a self-assessment worksheet can help wildland firefighters and other active people drink more fluids and stay hydrated. About 38 participants who are underhydrated will be split into two groups: one gets the…

This study follows 400 people with adrenocortical carcinoma who are taking mitotane as their first treatment. The goal is to carefully record all side effects, including ones that are not well understood. By collecting this data, researchers hope to improve how side effects are m…

This study looks at how many people with Addison's disease still produce small amounts of adrenal hormones like cortisol, even years after diagnosis. Researchers will test 200 adults in Norway and Sweden using blood and urine samples. The goal is to see if this leftover function …

This study evaluates a program that aims to improve diets by making healthy foods like fish, chicken, and eggs more available and affordable for low-income households in Mozambique. Researchers will survey over 5,000 men and women to see if the program increases how often they ea…

This study gathers health questionnaires from up to 1,500 people with multiple endocrine neoplasia (MEN1 or MEN2) and their close relatives. The goal is to build a database that helps researchers better understand these rare inherited conditions. No new treatments or procedures a…

This 10-year observational study will follow 500 children with growth hormone deficiency who are receiving the once-weekly injection somapacitan as part of their routine care. Researchers will track side effects, medication errors, and new cases of cancer or type 2 diabetes. The …

This study looks back at medical records from 1,000 people with adrenocortical carcinoma (ACC), a rare and serious cancer. Researchers want to see how factors like age, sex, race, and tumor burden relate to survival and treatment response. No new treatments are given; instead, th…

This study looks at how diet and exercise affect the epigenome—chemical marks on DNA—in sperm of overweight Hispanic men. Researchers will compare sperm from 20 healthy active men and 80 obese inactive men before and after a 12-week program of diet, exercise, or both. They will a…

This study looks at a specific genetic change found in some people with high blood pressure. Researchers want to see if this change is linked to a condition called primary aldosteronism, which can sometimes be cured with surgery. About 76 men from a larger Swedish health study wh…

This pilot study aimed to see if a self-paced virtual education video could help adolescents and young adults (ages 16-22) with congenital adrenal hyperplasia (CAH) become more independent in managing their health. Participants would have been split into two groups: one watching …

This study follows 100 adults aged 35-70 with mildly elevated blood pressure to see how their aldosterone hormone system changes over time. Participants will eat special high-salt or low-salt diets for a few days each year to test how their body responds. The goal is to better un…

This study planned to test whether an 8-week course of a flavored drink containing essential amino acids plus arginine could reduce liver fat and improve metabolism in obese children aged 7-10. The researchers intended to measure changes in liver fat, blood fats, insulin sensitiv…

This study watches 200 people with lung, gut, or hormone diseases over time using special brain scans (PET). It aims to see how these diseases change brain activity and whether those changes can predict tumor return, spread, or survival. No new treatment is tested—just observatio…

This study follows 150 patients with functional adrenal tumors to see how their heart and blood pressure change before and after surgery. Researchers will use heart ultrasounds and blood pressure monitoring to understand recovery and risks. The goal is to help doctors choose bett…

This study looks at over 3,600 people with adrenal tumors found by chance. Researchers want to see if mild cortisol excess from these tumors is linked to higher risk of death or heart problems like heart attack and stroke. Participants had a simple overnight dexamethasone test to…