Hereditary sensory and autonomic neuropathy

This study tests a drug called tivozanib in people with bile duct cancer that cannot be removed by surgery and has already been treated with chemotherapy. The goal is to find the safest dose and see if the drug can shrink tumors or slow the cancer. Participants take tivozanib by …

This study tests an experimental oral drug called ROC-101 in 40 adults with pulmonary arterial hypertension (PAH) or pulmonary hypertension linked to interstitial lung disease (ILD-PH). Participants take the drug alongside their standard care for 24 weeks, with a long-term follow…

This trial is testing an investigational drug called ARV-6723, given as a pill, in people with advanced solid tumors. The drug is designed to help the immune system recognize and attack cancer cells. The study will test ARV-6723 alone and in combination with pembrolizumab, an imm…

This study tests an experimental drug called ART0380, taken as a pill, in people with advanced or metastatic solid tumors (cancers that have spread). The drug targets a protein that helps cancer cells repair their DNA. Researchers want to find the safest dose when given alone or …

This early-stage trial is testing an experimental oral drug, HPB-092, in people whose acute myeloid leukemia (AML) has returned or not responded to standard treatments. The drug is designed to block two proteins, FLT3 and IRAK4, that help leukemia cells grow and survive. The stud…

This study tests a new drug called STX-478 in people with advanced solid tumors that have a specific genetic change (PI3Kα mutation). The trial has two phases: first, it tests STX-478 alone to find safe doses; then, it combines STX-478 with other cancer drugs like fulvestrant or …

This study tests two experimental drugs, sapanisertib and serabelisib, combined with standard therapy for people with HR+/HER2- advanced or metastatic breast cancer. The goal is to find safe doses and see if the combination can shrink tumors or slow the disease. About 32 adults w…

This study tests a new daily pill, ETX-636, for people with advanced solid tumors (including breast cancer) that have a specific genetic change called PIK3CA. The trial has two parts: first, finding a safe dose of ETX-636 alone or with another drug (fulvestrant); second, checking…

This study is testing whether a lower dose of radiation can effectively treat metastatic tumors that have a specific genetic change called an ATM mutation. The goal is to find the smallest effective dose to reduce side effects. About 45 adults with advanced cancer and an ATM muta…

This early-stage trial tests a new drug called TOS-358 in women with advanced HR+ HER2- breast cancer that has a specific mutation in the PIK3CA gene. The study aims to find the safest dose and see if the drug works alone or with standard treatments like fulvestrant and CDK4/6 in…

This study tests a combination of two targeted drugs, dabrafenib and trametinib, in people with rare solid tumors that have a specific genetic change called BRAF V600E. The goal is to see how well the drugs shrink or control the tumors. The study includes both children (age 1 and…

This early-stage trial tests an experimental drug called ACR-2316 in 100 people with advanced solid tumors that have spread or come back. The main goals are to check the drug's safety, find the best dose, and see if it can shrink tumors. It is for patients who have had up to 3 pr…

This early-phase trial is testing a new drug called AZD2962, which blocks a protein called IRAK4 involved in cancer growth. The study includes about 72 people with certain blood cancers (myelodysplastic syndromes or chronic myelomonocytic leukemia) that have not responded to prio…

This phase 2 trial tests alpelisib, a drug originally developed for cancer, in 20 people aged 2 to 40 with MCAP syndrome—a rare condition causing an enlarged brain, blood vessel issues, and developmental delays. Participants receive either alpelisib or a placebo for 6 months, the…

This study tests a new combination of drugs (sapanisertib and serabelisib, together called PIKTOR) plus standard chemotherapy (paclitaxel) in people with advanced or recurrent endometrial cancer that has not been cured by prior treatments. The goal is to see if this combo can shr…

This early-phase study tests a three-part drug combination for people with advanced pancreatic cancer who haven't had chemotherapy yet. The drugs include elraglusib (a targeted agent), retifanlimab (an immunotherapy), and a standard chemo regimen called mFOLFIRINOX. The main goal…

This Phase 3 study is testing whether markers in pancreatic fluid and blood can detect early pancreas cancer in people with high genetic risk. Participants receive a secretin injection to collect fluid during an endoscopy. The goal is to improve early detection in those with here…

This study tests a device called Walkasins, worn on the lower legs, that vibrates to give sensory feedback about foot pressure. The goal is to see if using it daily for six months improves balance and walking in 200 adults aged 55+ with peripheral neuropathy. Participants will do…

This study is a large registry that collects health and genetic information from people with a personal or family history of pancreatic tumors. The goal is to find the genes and other factors that cause pancreatic cancer to run in some families. By studying thousands of participa…

This international online study collects medical, behavioral, and developmental information from people with rare genetic changes that are linked to autism and other neurodevelopmental disorders. By partnering with families, researchers aim to build a detailed database to improve…

This study aims to learn more about rare inherited disorders that affect the brain, spinal cord, muscles, and nerves. Researchers will collect medical history, perform exams, and run genetic tests on up to 3,500 participants. No new treatments are tested; the goal is to better un…

This study is following 600 people in Germany with a type of advanced lung cancer (non-squamous non-small cell) who are starting a three-drug treatment: tremelimumab, durvalumab, and platinum chemotherapy. Researchers want to see how well the treatment works in real life, especia…

This study aims to see if a quick bedside sensory test can predict which painkiller—pregabalin, duloxetine, or a placebo—works best for people with nerve pain in their feet. About 190 adults with diabetic, chemotherapy-related, or idiopathic nerve pain will try each treatment for…

This study is collecting information from 4,000 adults with peripheral neuropathy (nerve damage) to better understand the condition. Researchers will combine standard tests with nerve ultrasound to see if it helps diagnose and track the disease. No new treatments are being tested…

This study looks at how a standard immunotherapy drug (anti-PD1) changes the appearance and biology of atypical moles in people with advanced melanoma. Researchers will track 30 patients to see if the drug alters mole size, color, and cellular features. The goal is to better unde…

This study is collecting nerve samples and biofluids from 400 people with ALS or peripheral neuropathies to find biological markers for early diagnosis and disease tracking. By analyzing individual cells, researchers hope to identify molecular changes that happen before symptoms …

This study follows people with familial dysautonomia (FD) to learn how the disease changes over time. Researchers will collect routine medical test results and optional blood samples to find biological markers that could help diagnose and treat FD better. The study is observation…

This study is looking at 50 adults with a rare genetic nerve disorder called RFC1-ataxia or CANVAS, which causes problems with balance, coordination, and sensation. Researchers will take blood samples and skin biopsies over 12 months to measure markers of cell stress and damage. …