Erdheim-Chester disease

This study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…

This trial is testing two drugs, vemurafenib and cobimetinib, already approved for melanoma, in adults with other rare cancers that have a specific genetic change called BRAF V600 mutation. The goal is to see if the combination can shrink tumors or stop them from growing. About 3…

This early-stage trial is testing a new drug called Q702 in people with various blood cancers and related disorders that have not responded to standard treatments. Q702 is designed to help the immune system attack cancer cells and help the bone marrow make healthy blood cells. Th…

This phase 2 trial is testing the drug mirdametinib in 40 people aged 2 and older with Langerhans cell histiocytosis or similar disorders. The goal is to see if this oral medication works better and has fewer side effects than current treatments. Participants take the drug twice …

This phase 2 study tests the drug cobimetinib in children and adults with Langerhans cell histiocytosis and related disorders that have come back or not responded to standard treatment. Cobimetinib blocks a growth signal that goes haywire in these diseases. The study will enroll …

This study tests a new type of ultrasound called super-resolution ultrasound (SRUS) to see if it can better tell the difference between benign (non-cancerous) and malignant (cancerous) swollen lymph nodes. About 779 adults with abnormal lymph nodes will receive an injection of ul…

This study surveys 250 informal caregivers (family or friends) of people with Erdheim-Chester disease and similar conditions. Participants complete online questionnaires about their supportive care needs, mood, and sense of purpose. The goal is to identify what caregivers need mo…

This study aims to learn more about how diseases related to blood clots, the immune system, and blood vessels start and change over time. Researchers will enroll up to 1,000 people aged 5 and older, including those with these conditions, their healthy relatives, and healthy volun…

This study looks for better ways to detect lung inflammation and infections early in people who have received a stem cell transplant from a donor. About 40 participants aged 5 to 70 will get regular lung scans, breathing tests, blood draws, and a procedure where a tube collects f…

This study follows 6,000 people with histiocytic disorders (like Langerhans cell histiocytosis) to understand long-term health problems, pain, mental health, and causes of death. Researchers compare participants to healthy controls to identify factors that lead to new health issu…

This study is testing a tool called B-COMPASS that uses a 22-question survey to predict whether people will follow their prescribed treatments. Researchers will enroll 3100 adults with cardiovascular, endocrine, immune, nerve, cancer, or rare diseases across Europe. The goal is t…

This study aims to create a better way to predict long-term outcomes for people with Erdheim-Chester disease (ECD), a rare condition that causes abnormal cells to build up in bones and organs. Researchers will analyze data from 1000 patients to identify factors that affect surviv…

This study is creating a large registry of 800 people receiving biologic medications for immune conditions like autoimmune diseases and primary immune deficiencies. Researchers will collect blood samples and health data to better understand how these treatments work and who respo…

This early-phase study is testing a new imaging agent called [68Ga]-pentixafor to see how well it shows up on PET/CT scans in people with certain blood cancers (like multiple myeloma and non-Hodgkin lymphoma) and rare immune cell tumors. About 30 participants will get the agent a…

This study creates a registry for people with Erdheim-Chester disease and related histiocytoses. Researchers will collect medical information and patient experiences over time to better understand these rare conditions. Up to 500 participants will be enrolled, and some may be ask…

This study at Mayo Clinic will collect medical histories and blood, fluid, or tissue samples from 500 people with histiocytic disorders. Researchers will analyze these samples for molecular markers like BRAF V600E and PD-L1 to better understand the diseases. The goal is to gather…