Familial polycythemia

This early-stage study tests a new drug called casdatifan (AB521) alone or combined with other cancer drugs in people with advanced clear cell kidney cancer or other solid tumors. The main goals are to check safety and find the right dose. About 362 participants will take part.

This phase 3 trial tests whether adding the drug sapablursen to standard treatment helps people with polycythemia vera, a blood disorder causing too many red blood cells. About 250 participants who currently need regular blood removal (phlebotomy) will receive either sapablursen …

This study tests whether a new pill (gecacitinib) plus a weekly injection (pegylated interferon) can help people with polycythemia vera, a condition where the body makes too many red blood cells. The goal is to bring blood counts back to normal and ease symptoms. Thirty adults wi…

This early-stage study tests a new drug, PRT12396, in about 100 people with polycythemia vera or myelofibrosis—rare blood cancers. The main goals are to find a safe dose and check for side effects. Researchers will also look for signs that the drug controls the disease, but this …

This study tests a new daily pill called JBI-802 in people with certain blood cancers (myeloproliferative neoplasms and MDS/MPN) who have too many platelets. The trial has two phases: first, finding a safe dose in about 30 adults, then checking if it works. Participants take the …

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 early-stage study tests an experimental drug, INCA036978, in about 218 adults with myeloproliferative neoplasms (a group of blood cancers). The main goal is to check the drug's safety and find the best dose, either alone or with standard treatment. Participants must have a l…

This study tests an experimental drug called DISC-3405 in 60 adults with polycythemia vera, a condition where the body makes too many red blood cells. The goal is to see if the drug can safely lower the need for phlebotomies (blood removal) and control blood counts. Participants …

This study tests an experimental drug called DISC-0974 in about 150 adults with myelofibrosis or myelodysplastic syndrome (MDS) who also have anemia. The goal is to see if the drug can reduce the need for blood transfusions and increase red blood cell levels. The study is in earl…

This early-phase study tests a new drug called INCB160058 in adults with myeloproliferative neoplasms, a group of rare blood cancers. The main goal is to check the drug's safety and how the body processes it. About 186 participants will receive the drug to find the right dose and…

This study tests whether ruxolitinib works better than current standard therapies (hydroxycarbamide or interferon alpha) for people with high-risk polycythemia vera, a blood cancer that increases red blood cells. About 586 adults aged 18 and older with high-risk features will be …

This study tests whether testicular tissue frozen before puberty can be transplanted back into adult men to restore sperm production. Five men who had fertility-threatening treatments as children will receive their own preserved tissue. The goal is to see if sperm can be found in…

This study is checking whether unlicensed cord blood units are safe to use for stem cell transplants in children and adults with blood disorders. Researchers will closely watch for any infusion-related problems. The goal is to see if these units can be a safe option for patients …

This study follows about 400 people with certain blood cancers (like essential thrombocythemia and myelofibrosis) who are already taking bomedemstat and benefiting from it. The goal is to collect more information on the drug's long-term safety and how well it controls the disease…

This phase 3 trial compares the experimental drug givinostat to the standard drug hydroxyurea in 220 adults with high-risk polycythemia vera, a blood cancer that increases red blood cells and raises clot risk. The study measures how well each drug controls blood counts and spleen…

This study tests whether the drug lusutrombopag can help restore platelet levels faster in patients who have had a stem cell transplant for blood disorders. About 45 adults will receive the drug after transplant, and doctors will check platelet counts at 21 days. The goal is to i…

This phase 3 trial is testing whether direct oral anticoagulants (DOACs) like apixaban or rivaroxaban are better than low-dose aspirin at preventing blood clots in people with JAK2V617F-positive myeloproliferative neoplasms (blood cancers). The study plans to enroll 1308 high-ris…

This study tests whether giving a chemotherapy drug (decitabine) plus a targeted therapy (ruxolitinib, fedratinib, or pacritinib) before a stem cell transplant helps people with advanced myeloproliferative neoplasms (MPN) get to transplant and have better outcomes. About 25 adult…

This study tests a new drug called flonoltinib maleate in 60 people with polycythemia vera (a condition where the body makes too many red blood cells) who cannot take or no longer respond to standard treatments. The goal is to see if the drug safely reduces red blood cell levels …

This study follows 36 Italian patients with Polycythemia Vera who responded well to Ropeginterferon alfa-2b in an earlier trial. Researchers will check every 6 months whether the treatment continues to keep their red blood cell levels normal and prevent the disease from getting w…

This study tests low-dose methotrexate, a common arthritis drug, in 54 people with three rare blood cancers: polycythemia vera, essential thrombocythemia, and myelofibrosis. Researchers want to see if it can shrink spleens, improve symptoms, and lower blood counts safely. If succ…

This study is for adults with blood disorders who are getting a stem cell transplant from a matched donor. Researchers want to see if giving a special potato starch along with a drug that lowers iron (deferasirox) can help prevent serious complications like graft-versus-host dise…

This early-phase trial is testing whether adding abemaciclib to the standard drug ruxolitinib is safe and effective for people with myelofibrosis, a type of bone marrow cancer. The study will enroll 18 adults who have been on ruxolitinib for at least 12 weeks. Researchers will mo…

This program provides early access to the drug ruxolitinib for people with serious or life-threatening conditions like certain blood cancers, severe COVID-19, or graft-versus-host disease. It is for patients who have no other treatment options and cannot join a clinical trial. Th…

This phase 2 trial tests a two-step, lower-intensity transplant method for people with various blood cancers. The goal is to see if this gentler approach reduces the chance of dying from the transplant itself. About 63 participants will receive a combination of radiation, chemoth…

This study tests whether adding navtemadlin to the standard drug ruxolitinib helps people with myelofibrosis who aren't getting enough benefit from ruxolitinib alone. About 600 adults with myelofibrosis will first take ruxolitinib, then those with a poor response will be randomly…

This study aims to develop a non-invasive method to detect and track fibrosis (scarring) in myelofibrosis, a serious blood disorder. Currently, doctors must perform a bone marrow biopsy to check fibrosis. The researchers will test whether a blood test and a special PET scan can i…

This study looks at whether doing breathing exercises before orthopedic surgery (like knee, hip, or fracture repair) can help your lungs work better after the operation. It involves 36 adults who have used tobacco and are at risk for breathing problems after surgery. Participants…

This phase 2 trial tests whether a curcumin supplement (C3 complex with piperine) can reduce inflammation and improve symptoms in people with certain blood disorders, including CCUS, low-risk MDS, and MPNs. Thirty participants will receive either curcumin or a placebo for 12 mont…

This study tests N-Acetylcysteine (N-AC), an antioxidant, in 27 adults with myeloproliferative neoplasms (MPN) like essential thrombocythemia, polycythemia vera, or myelofibrosis. The goal is to find the best dose that safely reduces symptom burden, measured by a symptom score. P…

This study tests whether a program of exercise and ergonomics training can reduce muscle and joint pain in teachers. About 80 teachers who have had pain for at least three months will be randomly assigned to receive the training or not. Researchers will measure changes in pain, d…

This study tests a web-based app called IAMABLE that provides evidence-based rehabilitation strategies for people with chronic conditions like heart disease, arthritis, or neurological disorders. Fifty adults aged 45 to 75 will use the app for 4 months to set goals and learn abou…

This phase 2 trial is testing whether the drug ruxolitinib (Jakafi) can reduce symptoms like fatigue and itching in people with low-risk essential thrombocythemia (ET) or polycythemia vera (PV). About 60 participants will take the pill daily, and researchers will measure symptom …

This study follows about 200 adults with polycythemia vera (a blood disorder) who do not have an enlarged spleen and are starting treatment with ropeginterferon alfa-2b. Researchers will track symptoms, blood cell counts, and side effects over 36 months in real-world clinics. The…

This study tests whether extra support for case managers helps veterans leaving the military better manage pain and reduce risky substance use. About 1800 veterans will take part. The program focuses on non-drug pain treatments and counseling.

This pilot study tests whether a 12-week nutrition and exercise program is feasible for patients with myeloproliferative neoplasms (MPNs), a group of blood cancers. Thirty participants will take virtual and in-person classes to learn healthier eating and physical activity habits.…

This study collects blood stem cells and immune cells from healthy volunteers and patients with primary immune deficiencies or blood disorders. The cells are used in the lab to develop new gene and cell therapies. Up to 850 adults aged 18-70 will participate. The goal is to advan…

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 at how a simple monitor called the Pleth Variability Index (PVI) changes during laparoscopic-assisted vaginal hysterectomy. Researchers will track PVI at six key moments when the patient's body position changes, such as tilting the head down or raising the legs. …

This research study creates a registry of older adults with cancer to collect information about their health, treatments, and outcomes. Participants complete a geriatric assessment and allow researchers to follow their medical records. The goal is to better understand how cancer …

This study aims to learn more about rare skeletal disorders by collecting medical records, blood samples, and genetic data from 100 participants. People with known or suspected bone conditions, as well as their healthy family members, can join either remotely or in person. The go…

This study collects blood, bone marrow, urine, and tissue samples from up to 6,000 people with solid tumors, blood cancers, or non-cancerous blood disorders, as well as from their healthy family members who are stem cell donors. The samples are stored and used in research to bett…

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 translates and tests several health questionnaires—covering pain, arm and leg function, mood, and daily activities—for use with Albanian-speaking people. Researchers will check if the translated versions are reliable and accurate by giving them to about 300 participant…

This study tests a new tool called the PRISM Dashboard, which gives physiotherapists feedback on how their decisions compare to their peers. The goal is to see if this feedback helps reduce differences in care for people with muscle and joint pain. About 60 physiotherapists and t…

This study is testing a new technique called optical genome mapping (OGM) to find genetic changes in blood cancers like polycythemia vera, essential thrombocythemia, and myelofibrosis. Researchers will compare OGM with standard genetic tests in 300 patients who need a bone marrow…

This study looks at how the immune system reacts to myeloproliferative neoplasms (MPN), a group of blood cancers. Researchers will collect blood and bone marrow samples from 150 adults with or without MPN to find special immune cells (T cells) that recognize and attack MPN cells.…

This study creates a nationwide registry and biobank for children diagnosed with myeloproliferative neoplasms (MPNs)—rare blood cancers that cause overproduction of blood cells. By collecting medical data and blood samples from up to 500 children across Germany, researchers hope …

This study is a large registry that collects health information from up to 200,000 people with blood disorders such as hemophilia, thrombosis, and sickle cell disease. Participants are seen at Hemophilia Treatment Centers across the U.S. The goal is to gather real-world data to h…

This study aims to understand exactly how the drug interferon attacks cancer stem cells in people with myeloproliferative neoplasms (MPNs), a group of blood cancers. Researchers will analyze blood samples from 80 adults to see why some patients respond well while others do not. T…

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 follows 252 adults with muscle, bone, or joint problems (like arthritis, back pain, or after surgery) to see how well they do their prescribed home exercises. Researchers will track who sticks with the program and why, looking at factors like pain, mood, and support. T…

This study looks at how gut bacteria and the immune system interact in children and adults receiving stem cell transplants or CAR-T therapy. Researchers will collect stool and blood samples over time to find patterns linked to complications like infections or graft-versus-host di…

This study is for adults with polycythemia vera, essential thrombocythemia, or pre-myelofibrosis who are starting their first treatment. Researchers want to find out why some patients respond well to standard drugs while others do not. They will look at genetic and immune system …

This study tests whether using a virtual reality (VR) headset during shoulder exercises helps patients feel more engaged and motivated. Fifty adults with shoulder problems will do rehab exercises in a VR environment and then answer questionnaires about how easy and immersive the …

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 study aims to create a secure national database for muscle and joint (musculoskeletal) conditions by collecting routine data from community clinics and GP practices. About 22,000 adults will have their information anonymously gathered to help develop a dashboard that tracks …

This long-term observational study is enrolling 5,000 adults with myeloproliferative neoplasms (MPNs)—rare blood cancers like polycythemia vera and myelofibrosis. Researchers will collect health records, patient surveys, and insurance data over at least five years to track sympto…

This study watches 2000 people having hip or knee replacement surgery to see how long spinal anaesthesia lasts. The goal is to learn how the amount of anaesthetic affects timing, helping doctors decide if patients can safely go home the same day. No new treatments are tested—just…

This study follows 102 women who had fertility preservation before cancer treatment to see what happens long-term. Researchers want to know if they get pregnant naturally, use stored eggs or tissue, or decide not to have children. The goal is to improve support and care for femal…

This study is looking for better ways to predict blood clots in people with myeloproliferative neoplasms (MPNs), a group of blood cancers. Researchers will measure specific markers in the blood of 300 participants to see if they can tell who is at higher risk for clots. The goal …

This study is collecting long-term safety and effectiveness data on treatments for various blood disorders, including hemophilia, sickle cell disease, and clotting disorders. It aims to enroll 3000 people of any age with these conditions. Researchers will monitor side effects, co…

This study is creating a collection of blood samples from 2,000 people with various blood disorders. The samples will be stored and used for future research approved by an ethics board. No treatments or drugs are being tested, and participants continue their normal care.

This study looks at how changing the heel cushion on an ankle-foot brace (AFO) affects walking in people who use one daily. Forty adults with leg injuries or nerve problems will try four different heel wedges—tall or short, soft or firm—while walking at controlled speeds. Researc…

This study examines why some patients with essential thrombocythemia (ET) later develop polycythemia vera (PV), a related blood condition. Researchers will review medical records of 678 adults to identify risk factors for this change and compare outcomes between those who progres…

This study looks at how 3D computer simulation and printing can help surgeons better plan and perform bone correction surgeries. Researchers will compare the planned corrections to the actual results in 100 children and young adults with rare bone deformities. The goal is to make…

This study looks at a type of immune cell called monocytes in people with certain blood cancers (polycythemia vera, essential thrombocythemia, and myelofibrosis). Researchers will analyze blood samples from 70 newly diagnosed patients to find unique patterns in these cells. The g…

This study looks at families who carry a change in the DDX41 gene, which may raise the risk of blood cancers like leukemia. Researchers will collect health questionnaires and saliva samples from up to 910 people to track who develops blood problems and when. The goal is to better…

This study follows 4,000 children who receive orthopedic implants (like rods or screws) during standard care for bone fractures, deformities, or hip problems. Researchers will track how long the implants last, any complications, and how well they work over time. The goal is to ga…

This study looks at how thick the blood is in 160 people with polycythemia (a condition with too many red blood cells). Researchers want to see if thicker blood causes more symptoms like headaches or dizziness, and how it differs between types of polycythemia. No new treatments a…

This study is building a registry of people with rare bone diseases (skeletal dysplasias) in Italy. Researchers will collect medical history, genetic data, and treatment details over time to better understand how these conditions progress. No experimental treatments are given; th…

This study looks at using a needle placed into the bone marrow (intraosseous infusion) to give fluids and medicines quickly to critically ill patients with blood diseases. Researchers will enroll 52 adults aged 18-65 to see how often the first attempt works and how safe it is. Th…

This study follows 144 adults with muscle or bone problems who are already scheduled to receive treatment with Chattanooga Intelect devices (like TENS or ultrasound). Researchers will measure pain, muscle strength, and movement range to confirm the devices are safe and work as ex…

This study collects blood, tissue, and body fluid samples from up to 15,000 people with blood cancers or related disorders. The samples are stored in a central lab for future research on diagnosis, prognosis, and immune monitoring. Participants do not receive any treatment as par…

This study is a registry that tracks patients with blood diseases who receive stem cell transplants at a hospital on the Qinghai-Tibet Plateau. Researchers will collect information on recovery, complications, and costs over time. The goal is to understand how high altitude affect…

This study aims to uncover inherited (germline) genetic factors that make some people more likely to develop myeloproliferative neoplasms (MPNs), a group of blood cancers. Researchers will analyze DNA from 313 patients diagnosed at a young age or with a family history of blood ca…

This study looks at whether a quick ultrasound of the thigh muscle can detect muscle loss (sarcopenia) in people who have finished cancer treatment. Researchers will compare the ultrasound results with a standard whole-body scan (DEXA) in 55 adults aged 18-74 with certain cancers…