Erdheim-Chester disease

This trial tests whether two approved drugs, dabrafenib and trametinib, can treat rare cancers that have a specific genetic change called BRAF V600. These drugs are already used for melanoma and lung cancer, but researchers want to see if they help people with other cancer types.…

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 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…

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 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 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 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…