Aplastic anemia

This phase 3 trial tests whether adding pelabresib to the standard drug ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a type of bone marrow cancer. About 460 adults who have not taken JAK inhibitors before will receive either the combination or a …

This study tests a stem cell transplant using blood stem cells from a half-matched family donor (haploidentical) for people with severe aplastic anemia, related MDS, or PNH. Participants receive chemotherapy and a single radiation dose before the transplant, plus a drug to preven…

This study tests whether a new cord blood stem cell product, Omidubicel, can improve transplant outcomes for people with severe aplastic anemia or a related bone marrow disorder. Participants receive a combination of stem cells from a family member and specially processed cord bl…

This study tests an oral drug called pacritinib in people aged 12 and older with myelodysplastic syndromes (MDS) or related blood cancers (MDS/MPN). The goal is to find the best dose and see if it can shrink or control the disease. Participants take capsules twice daily for up to…

This study tests a new way to do stem cell transplants for people with severe aplastic anemia or other bone marrow failure diseases. Doctors give a smaller number of donor white blood cells along with the stem cells to try to reduce serious side effects. Up to 120 patients aged 4…

This study tests two experimental drugs, pozelimab and cemdisiran, together in 35 adults with paroxysmal nocturnal hemoglobinuria (PNH) whose red blood cell destruction is not well-controlled by current C5 inhibitors. The goal is to see if the combination can lower hemolysis (bre…

This study follows about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking pegcetacoplan (Aspaveli/Empaveli) as part of their routine care. Researchers will track blood markers, symptoms, and side effects for up to 3 years to see how well the drug works out…

This study follows about 665 people with myelodysplastic syndromes, beta-thalassemia, or myelofibrosis who have already taken luspatercept in earlier trials. Researchers want to see how safe the drug is over many years by tracking side effects, disease progression, and survival. …

This study tests a new type of stem cell transplant for children with severe blood disorders like sickle cell disease or aplastic anemia. The transplant uses stem cells from a parent or other half-matched family donor, which are specially processed to remove certain immune cells.…

This study tracks 8 Korean patients with paroxysmal nocturnal hemoglobinuria (PNH) who are taking Danicopan alongside standard C5 inhibitors like eculizumab or ravulizumab. The goal is to monitor safety and check if the drug works as expected in everyday medical practice. It is a…

This study tests a drug called pegcetacoplan in 12 to 17-year-olds with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug aims to control the disease by blocking part of the immune system. The trial will check safety, how the…

This study is monitoring the safety and effectiveness of the drug iptacopan (Fabhalta) in 21 Korean adults with paroxysmal nocturnal hemoglobinuria (PNH) or C3 glomerulopathy (C3G). Researchers will track side effects and lab results from routine medical records. The goal is to s…

This trial tests a gene therapy for Fanconi anemia, a rare genetic disorder that causes bone marrow failure and increases cancer risk. Participants receive their own stem cells that have been genetically corrected with a lentiviral vector to fix the faulty FANCA gene. The study e…

This Phase 2 trial is testing a stem cell transplant from a donor for people with various blood cancers and bone marrow disorders. The transplant uses strong chemotherapy or radiation to wipe out the diseased marrow, then replaces it with healthy donor cells. A new combination of…

This early-phase study is testing a new drug called rebecsinib in 28 people whose secondary acute myeloid leukemia or high-risk myelofibrosis has come back or not responded to treatment. The drug is given by IV infusion four times per 28-day cycle for up to six cycles. The main g…

This early-stage trial is testing whether adding roginolisib to the standard drug ruxolitinib is safe and tolerable for adults with myelofibrosis whose disease no longer responds to JAK inhibitors. The study will enroll 26 participants and monitor side effects, heart changes, and…

This Phase 2 trial tests a combination of horse anti-thymocyte globulin, cyclosporine, steroids, and growth factors in 140 adults with aplastic anemia or low-risk myelodysplastic syndrome (MDS). The goal is to see if this immune-suppressing and blood-cell-boosting approach can im…

This phase 2 trial tests the drug pacritinib in 30 people with myelofibrosis, a rare bone marrow cancer, who also have low platelet counts. The goal is to see if pacritinib can reduce scarring (fibrosis) in the bone marrow and improve symptoms. Participants take pacritinib pills …

This study is testing the long-term safety and effectiveness of two experimental drugs, pozelimab and cemdisiran, taken together for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 202 participants who completed a prior study will continue treatment to see…

This study tests an experimental drug called ALN-CFB in people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cells despite standard treatment. The goal is to see if adding ALN-CFB is safe and can improve anemia. About 24 participants will receive eit…

This study tests a new drug called ADX-038 in healthy volunteers and people with PNH who still have low red blood cells despite treatment. The goal is to check safety and how the drug works in the body. It is an early-stage trial with 50 participants.

This early-phase study tests whether adding a new drug called pelabresib to the standard treatment ruxolitinib is safe and helpful for Japanese adults with myelofibrosis, a type of bone marrow cancer. Only 6 people will take part, and the main goal is to check for side effects an…

This study is testing whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant, can prevent graft-versus-host disease (a common complication where donor cells attack the patient's body) while still being effective. The trial involves 260 adults with b…

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 provides unlicensed cord blood units for transplant to children and adults with blood cancers, immune disorders, and other serious diseases. The goal is to see how well patients recover their white blood cells after the transplant. By making more cord blood units avail…

This program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant extravascular hemolysis (EVH) despite treatment with Soliris or Ultomiris. Participants must be 18 or older, have anemia and high reticulocyte counts, an…

This phase 2 trial tests a stem cell transplant from a donor for people with high-risk sickle cell disease, thalassemia, and other red blood cell disorders. Participants receive drugs and low-dose radiation before the transplant to help the new cells take hold. The study will tra…

This early-phase study tests whether adding pegylated interferon alfa-2a after a stem cell transplant can help control myelofibrosis, a type of bone marrow cancer. About 18 adults with primary or secondary myelofibrosis will receive the drug after their transplant to find the saf…

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 phase 2 trial tests a bone marrow transplant using lower doses of chemotherapy and radiation to prepare the body, along with a drug called cyclophosphamide after transplant to prevent graft-versus-host disease. It enrolls 60 people with severe aplastic anemia or related bone…

This early-phase study tests whether adding hyperbaric oxygen therapy (breathing pure oxygen in a pressurized chamber) to standard stem cell transplant preparation is safe and helps recovery. It involves 24 adults with various blood cancers, such as leukemia and myelofibrosis. Th…

This study looks at how well the drug iptacopan works for adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will track changes in hemoglobin levels and other health outcomes in 80 patients across China. The goal is to see if iptacopan impro…

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 early-stage trial is testing a drug called tasquinimod, either alone or with the standard drug ruxolitinib, in 33 people with myelofibrosis (a type of bone marrow cancer). The goal is to see if the combination is safe and can help control the disease, especially in patients …

This study tests a drug called afatinib in 25 adults with Fanconi anemia who have advanced head and neck cancer that cannot be removed by surgery. Afatinib is a pill that blocks a protein helping cancer grow. The goal is to see if it can shrink tumors and control the disease. Res…

This early-phase study tests an oral drug called PMD-026 in 18 adults with myelofibrosis, a type of bone marrow cancer. Participants must have already tried a JAK inhibitor without success. The main goals are to check safety, find the best dose, and see if the drug can shrink the…

This early-stage trial tests the safety of a new drug called EA5 in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The drug is an antibody designed to block part of the immune system that attacks blood cells. The goa…

This early-stage trial tests a one-time gene therapy called APR-2020 in 4 children with a severe form of Diamond-Blackfan anemia. The treatment uses the child's own blood stem cells, modified to correct the genetic defect, and given back as an infusion. The goal is to see if it i…

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 study tests an experimental drug called flonoltinib maleate in about 64 people with a rare bone marrow cancer (myelofibrosis) that is moderate to high risk and has not responded to or stopped responding to standard JAK inhibitor therapy. Participants take a daily pill on an …

This early-stage study tests an experimental oral drug, AJ1-11095, in about 76 adults with myelofibrosis (a type of bone marrow cancer) whose disease has stopped responding to standard JAK2 inhibitor therapy. The main goals are to check the drug's safety, find the best dose, and …

This phase II trial tests different drug combinations to prevent graft-versus-host disease (GVHD) in 400 blood cancer patients receiving stem cell transplants from unrelated donors. Participants will receive either thymoglobulin or a combination of cyclophosphamide with other imm…

This phase 2 study tests the drug reparixin in 10 people with myelofibrosis, a type of bone marrow cancer. Participants have advanced disease and cannot take or have not responded to standard JAK inhibitor therapy. They take reparixin pills three times daily for 6 months, with th…

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 nelfinavir, a drug originally used for HIV, can help people with myelofibrosis who have anemia (low red blood cells). The drug aims to restore iron delivery to the bone marrow. Ten adults with myelofibrosis and low hemoglobin will take nelfinavir twice da…

This early-phase trial tests a combination of two drugs, tagraxofusp and pacritinib, in about 20 adults with myelofibrosis (a bone marrow disorder) who have already tried standard JAK inhibitors or cannot use them. The main goals are to see if the combo can shrink an enlarged spl…

This early-stage study tests a new oral drug, LP-118, in up to 100 adults with blood cancers like lymphoma, leukemia, or myeloma that returned or didn't respond to prior therapy. The main goals are to find the safest dose and see if the drug can shrink tumors or slow the disease.…

This study tests an experimental pill called nuvisertib for people with myelofibrosis, a rare bone marrow disorder. It is for patients whose disease is moderate to high risk and who have not responded well to standard treatments. The goal is to see if the drug is safe and can shr…

This study tests a stem cell transplant method for adults aged 40 to 60 with severe aplastic anemia that has not improved or has returned after standard drug therapy. The approach uses a special drug after transplant to lower the risk of graft-versus-host disease, a common and se…

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 small study tests a device called MEX-CD1 that removes excess iron from the blood during a special type of dialysis. It is for people with myelodysplastic syndrome or myelofibrosis who have iron overload from blood transfusions but cannot take standard chelation therapy. Eac…

This phase 2 study tests whether combining two drugs, momelotinib and luspatercept, can help people with myelofibrosis who need regular blood transfusions. About 68 adults with primary or secondary myelofibrosis will receive the combination orally or by injection. The main goal i…

This study is for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells, leading to serious infections and bleeding. It tests a bone marrow transplant from a half-matched family donor, followed by a drug to reduce complications…

This early-stage trial tests a new drug called INCA033989, alone or with an existing drug (ruxolitinib), in people with myeloproliferative neoplasms—a group of rare blood cancers that cause the bone marrow to produce too many cells. The study aims to find safe doses and check for…

This study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…

This study tests a medicine called iptacopan in children aged 2 to under 18 who have a rare blood disease called PNH. The goal is to see how the drug works in their bodies and if it is safe. About 12 children will take the medicine, and doctors will monitor side effects and blood…

This program offers eltrombopag to patients with serious or life-threatening thrombocytopenia (low platelet counts) who have no other treatment options and cannot join a clinical trial. A licensed physician must request the drug, and the potential benefit must outweigh the risks.…

This study tests whether a once-weekly injection (romiplostim) can help platelets recover faster and more safely than a daily injection (rhTPO) after a donor stem cell transplant. It includes 66 adults with MDS or severe aplastic anemia. The goal is to reduce bleeding risk and tr…

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 early-phase study tests a new automated method (CliniMACS Prodigy) to select CD34 stem cells for patients with blood cancers who need a stem cell boost or second transplant. The goal is to reduce the risk of severe graft-versus-host disease (GVHD) while improving blood cell …

This study tests a new drug called HS-10542 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The goal is to see if the drug can safely raise hemoglobin levels and reduce the need for blood transfusions. About 50 partic…

This study tests a device that removes specific immune cells from donated stem cells before transplant. The goal is to lower the risk of graft-versus-host disease, where donor cells attack the patient's body. Up to 90 children and young adults (ages 0-30) will receive these treat…

This early-stage trial tests a new drug, INCA035784, in 120 adults with myeloproliferative neoplasms (a group of blood cancers). The main goal is to check safety and find the right dose. Participants must have a specific genetic mutation (CALR) and have tried other treatments bef…

This early-stage study is testing a drug called TQ05105 in people with mild or moderate liver impairment and healthy volunteers. The goal is to see how the liver affects the drug's levels in the body and check for safety. 24 participants will receive a single dose and be monitore…

This study tests whether adding ulixertinib to the standard drug ruxolitinib is safe and helpful for people with myelofibrosis, a type of bone marrow cancer. About 37 adults who are already on a stable dose of ruxolitinib will receive different doses of ulixertinib. The goal is t…

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 study tests whether adding umbilical cord blood to a half-matched stem cell transplant can help people with aplastic anemia, a serious condition where the bone marrow stops making enough blood cells. About 224 participants aged 14 and older will receive either the combined t…

This early-stage trial tests the safety and best dose of the drug momelotinib when given before, during, and after a stem cell transplant for people with myelofibrosis, a serious bone marrow disorder. About 28 participants will receive momelotinib pills daily for up to 13 cycles.…

This early-stage trial is testing a new drug called INCA033989, alone or with an existing drug ruxolitinib, in 225 people with myeloproliferative neoplasms (a group of blood cancers). The main goal is to check safety and find the best dose. Participants must have a specific genet…

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 new drug combinations can better prevent graft-versus-host disease (GVHD) in people with blood cancers who receive a stem cell transplant from a mismatched unrelated donor. About 358 participants will get either the standard GVHD prevention or one of two …

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 whether giving high-dose ruxolitinib and busulfan before a stem cell transplant can reduce spleen size and help the transplant work better in people with myelofibrosis. About 30 adults aged 18 to 75 with an enlarged spleen and a matched donor will take part. The …

This study tests whether starting two oral medications (cyclosporine and eltrombopag) earlier than usual is safe and feasible for people with severe aplastic anemia, a rare condition where the immune system attacks bone marrow. Participants aged 3 and older will take these pills …

This phase 3 trial tests a new drug called flonoltinib maleate in people with intermediate- or high-risk myelofibrosis, a type of bone marrow cancer. The study compares flonoltinib to an existing drug, ruxolitinib, to see if it can reduce spleen size and improve symptoms. About 1…

This program offers early access to the investigational drug selinexor for people with certain advanced cancers, such as multiple myeloma, lymphoma, sarcoma, and others, who have no other treatment options. Patients must not qualify for any ongoing clinical trial and must have ex…

This expanded access program offers a special stem cell purification technique (CD34+ selection) for patients undergoing stem cell transplants. The method removes many T-cells from the donated stem cells, which lowers the risk of a serious complication called graft-versus-host di…

This study is tracking 10 children aged 6 to 17 with aplastic anemia, a condition where the bone marrow stops making enough blood cells. The children are receiving eltrombopag (Revolade) for the first time, combined with anti-thymocyte globulin. Researchers are monitoring for ser…

This phase 2 trial tests whether adding the drug ruxolitinib to standard care can prevent graft-versus-host disease (GVHD) in older patients with myelofibrosis or MDS/MPN overlap syndromes who receive a donor stem cell transplant. About 50 participants will get ruxolitinib before…

This study tests a bone marrow transplant from a partially matched donor (like a parent or sibling) for people newly diagnosed with severe aplastic anemia, a condition where the bone marrow stops making enough blood cells. Participants receive chemotherapy and immune-suppressing …

This Phase 2 trial tests the drug selinexor in people with myelofibrosis who have not taken JAK inhibitors and have low to normal platelet counts. The main goal is to see if selinexor can shrink the spleen by at least 35% after 24 weeks. Researchers will also check if it improves…

This study tests a new drug called elritercept (KER-050) for people with myelofibrosis, a rare bone marrow disorder. The drug is given alone or with the standard treatment ruxolitinib. The main goals are to check safety and see if it helps with anemia and other symptoms. About 13…

This study is observing how well a double umbilical cord blood transplant works for 40 adults with blood cancers like leukemia or lymphoma. The transplant uses two units of cord blood from unrelated donors to help rebuild the patient's blood system. Researchers are tracking survi…

This phase 2 trial tests a special stem cell transplant for people with inherited bone marrow failure disorders like Fanconi anemia. The transplant uses a technique to remove certain immune cells, aiming to reduce the need for long-term immune-suppressing drugs and lower infectio…

This study tests a new way to treat Fanconi Anemia, a rare genetic disorder that causes bone marrow failure. Doctors will give patients donor stem cells that have been specially filtered to remove certain immune cells, along with an experimental antibody called JSP191. The goal i…

This study tracks 50 children and adults with non-malignant disorders like immune deficiencies and anemias who receive a stem cell transplant after a reduced-intensity chemotherapy regimen. The goal is to see if this approach improves survival and reduces severe graft-versus-host…

This study is for people with myelofibrosis, a type of bone marrow cancer. It tests whether giving a JAK inhibitor drug before, during, and after a half-matched stem cell transplant can lower the risk of graft-versus-host disease (a common complication where donor cells attack th…

This early-stage trial tests a new way to do a stem cell transplant for people with severe aplastic anemia whose disease has come back or not improved with standard treatments. The approach uses a milder conditioning regimen and removes certain immune cells from the donor cells t…

This early-stage trial tests two drugs—imetelstat and ruxolitinib—together in people with myelofibrosis, a type of bone marrow cancer. The study has two parts: first, finding a safe dose of the combination, and second, checking if it improves symptoms like fatigue and spleen disc…

This study tests two drugs—axatilimab and ruxolitinib—together in people with myelofibrosis or chronic myelomonocytic leukemia. The goal is to find a safe dose and see if the combination helps control the disease. About 66 adults who have tried other treatments or are new to ther…

This early-phase study tests a combination of two drugs, selumetinib and azacitidine, in adults with higher-risk blood cancers like MDS and myelofibrosis. The main goal is to find the safest dose of selumetinib when given with standard azacitidine and to monitor side effects. Par…

This phase 3 study tests whether adding danicopan to standard PNH medications (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria who still have significant anemia from extravascular hemolysis. Six participa…

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 tests whether giving emapalumab early can help children with newly diagnosed severe aplastic anemia recover their blood cell counts within 6 weeks. About 35 children will receive the drug before standard treatments. The goal is to see if this approach improves treatmen…

This study is testing a less intense chemotherapy and radiation regimen before a stem cell transplant for children and young adults up to age 55 with non-cancerous blood disorders like immune deficiencies, anemias, and metabolic diseases. The goal is to see if this gentler prepar…

This study tests whether combining two experimental drugs (TQ05105 and TQB3617) can safely shrink the spleen and improve symptoms in people with intermediate- or high-risk myelofibrosis, a type of bone marrow cancer. About 92 adults will take the pills daily, and researchers will…

This program provides early access to the drug iptacopan for people with serious or life-threatening C3 glomerulopathy (C3G) or paroxysmal nocturnal hemoglobinuria (PNH) who have no other treatment options. It is designed for patients who cannot join a clinical trial or have exha…

This study tests whether a tailored exercise program combined with a dietary supplement called nicotinamide riboside (NR) can improve muscle mass, strength, and fitness in adolescents and young adults who had a bone marrow transplant for leukemia or other blood disorders. About 8…

This phase 2 trial is testing a stem cell transplant using a milder chemotherapy regimen to treat people with severe immune deficiencies and inherited bone marrow failure. The goal is to see if donor cells can safely take over and rebuild a healthy immune system. Up to 27 partici…

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 program offers extra stem cells from a half-matched family donor to patients who are getting a cord blood transplant for serious blood cancers or immune disorders. The goal is to help the bone marrow recover more quickly while the cord blood cells take over permanently. It i…

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 study tests a stem cell transplant approach for people with Fanconi Anemia who don't have a perfectly matched donor. The goal is to see if using lower doses of chemotherapy before the transplant can reduce serious side effects. About 70 participants will receive a transplant…

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 is testing whether umbilical cord blood stem cells from unrelated donors can safely and effectively restore blood cell production in people with serious conditions like leukemia, immune deficiencies, and inherited blood disorders. The main goal is to see how quickly th…

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 study tests a specific order of antibiotics for cancer patients who develop fever due to low white blood cells (febrile neutropenia). Twenty adults with blood cancers will receive a carbapenem first, then add vancomycin or linezolid if fever persists, and finally an antifung…

This early-phase study tests whether Darzalex Faspro can safely lower donor-specific antibodies (DSAs) in 8 adults with blood cancers or bone marrow failure who are at high risk of rejecting a stem cell transplant. The goal is to reduce DSA levels enough to allow the transplant t…

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 Phase 2 trial tests a new drug called RVU120, alone or with the standard drug ruxolitinib, in 230 adults with intermediate or high-risk myelofibrosis. The main goal is to see if the drug can shrink the spleen and improve symptoms. Participants must have not responded well to…

This study explores whether MRI scans can safely and accurately detect cancerous or precancerous mouth lesions in people with Fanconi Anemia, a rare genetic condition that raises cancer risk. Researchers will compare MRI results to standard biopsies in 80 adult participants. If s…

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 is testing whether a special type of PET/MRI scan, using a tracer called 18F-FAPI, can accurately diagnose and track myelofibrosis. Researchers will compare the scan results with standard bone marrow biopsies in 57 patients. If the scan works well, it could offer a les…

This study tests whether an artificial intelligence (AI) algorithm can screen for coronary artery disease (CAD) by analyzing facial images. Researchers will enroll 1,392 high-risk adults (those with diabetes, high blood pressure, or over 65) and use the AI to classify them as hig…

This early-phase trial tests a vaccine (CMV-MVA Triplex) given to both stem cell donors and recipients to boost immunity against cytomegalovirus (CMV) and prevent active infection after a half-matched stem cell transplant. About 46 people with various blood cancers will take part…

This study tests whether moxibustion, a traditional therapy that burns herbs near the skin, can prevent hemorrhagic cystitis (painful bladder inflammation) in 266 patients receiving stem cell transplants. Participants are randomly assigned to receive either standard care or stand…

This phase 2 trial tests a vaccine called CMV-MVA Triplex, given to stem cell donors before they donate. The goal is to boost the donor's immunity against cytomegalovirus (CMV), which can then be passed to the transplant patient. Researchers hope this will prevent CMV infection i…

This study tests whether a personalized facial rehabilitation program can help people with systemic sclerosis who have facial tightness and mouth problems. About 60 adults with this rare autoimmune disease will either receive the rehab program or standard care. The goal is to see…

This study is testing whether hypnosis can lower anxiety in people having facial surgery under local anesthesia. Sixty-six adults who are at least mildly anxious will either get standard care or extra hypnosis support. Researchers will measure anxiety levels before and after surg…

This study tests whether a special cooling cap can prevent hair loss in children receiving chemotherapy for non-cancerous conditions or solid tumors. The cap is worn before, during, and after chemo to keep the scalp cool. Researchers want to see if it is safe and tolerable for 40…

This early-stage study tests a new drug called APG-5918 in healthy volunteers and people with anemia (low red blood cells). The main goal is to check safety and how the body handles the drug, while also seeing if it can raise hemoglobin levels. About 105 participants will take pa…

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 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 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 looks at anxiety related to cancer screenings in adults with Fanconi anemia, a rare condition that raises cancer risk. Researchers will observe 20 participants during their regular screening visits, noting body language, worries, and the clinic environment. No treatmen…

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 at how well the drug iptacopan works for people with paroxysmal nocturnal hemoglobinuria (PNH) in everyday medical practice. Researchers will track 50 Italian patients for 12 months to see if their anemia improves and if they need fewer blood transfusions. The go…

This study follows people with acquired or inherited bone marrow failure for up to 20 years to learn how the diseases and treatments affect the body over time. Participants, aged 2 and older, will have regular check-ups and tests every 1 to 3 years. The goal is to better understa…

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 aims to discover new genes that may cause certain cancers to run in families. Researchers will collect blood samples and health information from 1,500 people in families where multiple members have had cancer, especially childhood cancers. The goal is to build a regist…

This study collects blood samples and health information from 375 people with non-cancerous blood disorders like aplastic anemia, sickle cell disease, or thalassemia who are receiving a stem cell transplant or gene therapy. The goal is to create a biobank that helps researchers u…

This natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…

This study follows people with Fanconi anemia, a rare inherited condition that raises the risk of certain cancers, especially squamous cell carcinoma. Researchers will screen 200 participants aged 12 and older every year for up to 10 years using exams, blood tests, and imaging to…

This study collects blood, bone marrow, and other samples along with health information from up to 1,716 people with non-cancerous blood diseases and their family members. Researchers will analyze the participants' genes to find new genetic causes of these conditions and understa…

This study tests a new communication tool called 'Let's Get REAL' that helps children and teens (ages 8-17) and their families talk together about stem cell transplant or cellular therapy decisions. The goal is to see if the tool is easy to use and helpful for families. About 60 …

This study is following 200 people in Germany who have myelofibrosis (a type of bone marrow cancer) and anemia (low red blood cells). Researchers will track which treatments patients receive, how their disease responds, and how they feel over time. The goal is to gather real-worl…

This early-stage study tests a new drug called ZE74-0282 in 40 healthy adults aged 18-55. The main goal is to check its safety and how the body handles it, not to treat any disease. Participants receive either the drug or a placebo, and researchers monitor side effects and drug l…

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 uses a non-invasive brain imaging technique called fNIRS to see how anemia affects thinking and memory. Researchers will measure brain activity in 323 adults with different types of anemia and compare it to cognitive test scores. The goal is to identify which brain reg…

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 checks if people with Diamond Blackfan Anemia (ages 3-30) can produce enough blood stem cells for future gene therapy. Participants receive two drugs to move stem cells from bone marrow into the blood, then researchers measure the cell count. No actual stem cell collec…

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 is building a large database of people with Diamond Blackfan anemia, a rare inherited bone marrow condition that causes anemia. Researchers will collect medical information from up to 900 participants to better understand how the disease develops and progresses. The go…

This study looks at how the Shingrix vaccine (for shingles) works in people with psoriasis or myelofibrosis who are taking drugs that weaken their immune system. Researchers will compare their immune response to that of similar patients not on these drugs. The goal is to understa…

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 follows 75 pregnant women who have taken Ultomiris (ravulizumab) for conditions like PNH or aHUS. Researchers will track pregnancy outcomes and baby health up to one year after birth. The goal is to gather safety information, not to test a new treatment.

This early-stage study tests a new drug called CMS-D017 in 88 healthy adults to see if it is safe and how the body processes it. The drug is being developed for two rare conditions: paroxysmal nocturnal hemoglobinuria (a blood disorder) and complement-mediated kidney disease. Par…

This research study collects blood, bone marrow, and tissue samples from people with cancer, blood disorders, and healthy volunteers. The goal is to study how the immune system changes during different treatments, including immunotherapy and chemotherapy. By comparing samples fro…

This study tests a telehealth program that helps older adults (60+) with blood cancers like leukemia or MDS talk to their doctors about their care wishes. The program also helps them fill out advance directive forms. Researchers want to see if it improves communication and reduce…

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 watches 200 people with aplastic anemia who are getting ATG/ALG treatment for the first time. Researchers will track side effects like serum sickness, organ damage, and infusion reactions to learn how common and severe they are. The goal is to create better guidelines …

This study looks at how specific genetic changes (ASXL1 mutations) affect the course of myelofibrosis, a rare bone marrow cancer. Researchers will analyze blood and bone marrow samples from 50 adults to identify different patterns of these mutations. The goal is to better predict…

This study looks at whether doctors at Siriraj Hospital follow a new guideline for treating anemia in patients before major surgery. Anemia is common in surgical patients and can lead to more blood transfusions and worse outcomes. The researchers will track 93 anemic patients to …

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 looks at whether changes in the level of a specific gene mutation (CALR) in the blood can help predict when certain blood cancers (myeloproliferative neoplasms) get worse. Researchers will collect blood samples from 260 adults diagnosed between 2006 and 2020 and track …

This study is creating a large database of people in China with various blood diseases, such as leukemia, multiple myeloma, and hemophilia. Researchers will collect information from medical records and follow participants over time to learn about disease patterns, treatments, and…

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 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 is an observational study that will follow about 617 people with myelofibrosis across Europe. Researchers will collect information from medical records to see how the disease progresses and how patients respond to standard treatments. No experimental drug or intervention is …

This study looks at how metabolism in cancer and immune cells influences the bone marrow environment and treatment resistance in blood cancers like leukemia and myelofibrosis. Researchers will analyze samples from 350 participants to measure metabolites, lipids, and gene activity…

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 is observing 1000 adults having moderate- to high-risk surgery to see if the type and severity of anemia they have before surgery affects their risk of complications like heart injury, kidney injury, or stroke. Researchers will compare patients with iron-deficiency ane…

This study aims to create a prediction tool for pregnant women with aplastic anemia, a rare blood disorder that raises risks of bleeding, infection, and complications like preterm birth. Researchers will follow 300 women to identify factors that lead to poor outcomes for mother a…

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 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 testing whether an artificial intelligence (AI) tool can help match cancer patients with the right clinical trials. Researchers will enroll up to 50,000 people with various cancers to see if the AI improves trial enrollment rates and patient outcomes. The goal is to…

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…

This early-stage study is testing a single dose of the drug HSK39297 in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH) who have mild or moderate liver impairment, compared to those with normal liver function. The goal is to see how the liver affects drug levels and safe…