Langerhans cell histiocytosis

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 study tests a drug called tovorafenib (DAY101) in children and young adults (ages 6 months to 22 years) with Langerhans cell histiocytosis that has returned or not responded to treatment. The drug works by blocking enzymes that help cancer cells grow. Researchers will check …

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 study tests a targeted drug called trametinib in 75 children with Langerhans cell histiocytosis (LCH), a rare disease where immune cells grow out of control. The goal is to see if the drug can safely stop the disease from getting worse or coming back. Children with new, rela…

This study tests whether adding the targeted drug luvometinib to standard chemotherapy improves outcomes for children with multisystem Langerhans cell histiocytosis (LCH), a rare immune cell disorder. About 120 children aged 0-18 with newly diagnosed, untreated LCH will be random…

This study tests a new daily oral drug called luvometinib in children aged 0-18 with Langerhans cell histiocytosis (LCH) that affects certain bones. LCH is a rare condition where immune cells build up and can cause bone damage. The drug targets a faulty cell signal found in nearl…

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 a combination of low-dose cytarabine (a chemotherapy drug) and thalidomide in 50 adults with untreated Langerhans cell histiocytosis (LCH), a rare disease where immune cells build up and damage tissues. Participants receive up to 24 cycles of treatme…

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 phase 3 trial compares two chemotherapy approaches for children and young adults with Langerhans cell histiocytosis (LCH), a rare cancer that can damage tissues. One group gets the standard two-drug combo (vinblastine/prednisone), while the other gets a single drug called cy…

This study tests whether telepresence robots can help children aged 5-17 with cancer attend school remotely when they can't be there in person. The robots let kids see, hear, and talk in class from their hospital or home. Researchers will track school attendance, social well-bein…

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 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…