Polyendocrinopathy

This phase II trial tests whether intensive blood pressure management (targeting systolic BP below 120 mmHg) is better than standard care (below 140 mmHg) for patients with advanced kidney or thyroid cancer starting anti-angiogenic TKI therapy. The study enrolls 61 participants 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 phase 2 trial tests whether giving selpercatinib before standard surgery can shrink tumors in people with RET-altered thyroid cancer. About 30 participants with advanced or recurrent disease will receive the drug and then undergo surgery. The goal is to see if the drug impro…

This study tests an experimental drug called KL590586, taken as a capsule, in people with advanced solid tumors that have specific RET gene changes. The trial has two phases: Phase I checks safety and the right dose, while Phase II looks at how well the drug shrinks tumors. Up to…

This early-phase trial tests an investigational drug called ABBV-706, alone or combined with other drugs, in about 288 adults with advanced solid tumors like small cell lung cancer, high-grade brain tumors, and neuroendocrine cancers. The main goals are to check safety, find the …

This study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…

This early-phase study tests a radioactive drug (177Lu-PP-F11N) that seeks out and attaches to cancer cells in people with advanced medullary thyroid cancer and certain other neuroendocrine tumors. The goal is to see if the drug can help image tumors and deliver targeted radiatio…

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 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 stem cell transplant using a milder chemotherapy-like regimen to treat children and young adults (up to age 28) with various immune system disorders. The goal is to help the donor stem cells settle in the body with fewer side effects. The study involves 20 part…

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 II trial tests a stem cell transplant from unrelated or partially matched family donors for children and young adults with severe immune deficiencies. The stem cells are specially processed to remove certain immune cells, aiming to reduce complications. The goal is to …

This early-stage trial is testing an oral drug called LOXO-260 in about 70 people with advanced cancers that have a specific change in the RET gene. The cancers have stopped responding to other targeted treatments. The study aims to find the safest dose and see if the drug can sh…

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 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 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 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 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 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 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…