Aplastic anemia

This phase II trial tests a vaccine designed to protect stem cell transplant recipients from cytomegalovirus (CMV), a common virus that can cause serious illness in people with weakened immune systems. The vaccine uses a modified, harmless virus to deliver three CMV proteins, tra…

This study tests a vaccine called Triplex to prevent cytomegalovirus (CMV) infection in adults who have received a stem cell transplant. CMV is usually harmless but can be life-threatening for people with weakened immune systems. The vaccine uses harmless virus pieces to train th…

This study follows patients who have already been taking ruxolitinib alone or with panobinostat in earlier trials and are still benefiting. Researchers will track side effects over time to see how safe these drugs are for long-term use. About 296 participants with conditions like…

This phase 2 study tests the drug selinexor against standard treatments in 112 people with myelofibrosis who have already tried JAK inhibitors. The main goal is to see if selinexor can shrink the spleen and improve symptoms. Participants are randomly assigned to receive either se…

This study looks at the long-term safety and tolerability of iptacopan in adults with paroxysmal nocturnal hemoglobinuria (PNH) who have already completed earlier phase 2 or 3 studies with the drug. About 208 participants will continue taking iptacopan and be monitored for side e…

This early-phase trial tests whether adding specially grown umbilical cord stem cells to a standard bone marrow transplant can improve outcomes for people with severe aplastic anemia, a rare condition where the bone marrow stops making enough blood cells. About 40 participants ag…

This study monitors about 200 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking iptacopan (Fabhalta). Researchers will use registry data to count infections caused by certain bacteria. The goal is to better understand the real-world safety of this medic…

This phase II trial tested whether adding veliparib (a targeted drug) to standard chemotherapy (topotecan and carboplatin) helps people with advanced myeloproliferative disorders, acute myeloid leukemia, or chronic myelomonocytic leukemia. The study enrolled 25 adults whose cance…

This study is testing the long-term safety and effectiveness of an experimental drug called XH-S003 in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that can cause fatigue, blood clots, and other serious problems. About 26 people who have already ta…

This study tests a stem cell transplant method for people with acquired or inherited bone marrow failure. Donor stem cells are specially processed to remove certain immune cells, which may lower the risk of graft rejection and graft-versus-host disease. The goal is to see if this…

This early-phase trial tests a reduced-intensity stem cell transplant using a half-matched donor and a drug called cyclophosphamide after the transplant to prevent rejection. The goal is to see if this approach is safe enough for patients with advanced myelofibrosis, a serious bo…

This study provides extended access to the drug momelotinib for 237 people with a type of bone marrow cancer called myelofibrosis. Participants had already been taking momelotinib in earlier studies and had not seen their disease get worse. The goal is to see how safe the drug is…

This phase 3 trial tests a drug called imetelstat against the best available treatments for people with advanced myelofibrosis whose disease has stopped responding to JAK inhibitors. The study includes 327 participants and aims to see if imetelstat can help them live longer and f…

This study follows about 80 people with paroxysmal nocturnal hemoglobinuria (PNH) who have already taken danicopan in a previous trial. They will continue taking danicopan as an add-on to their standard C5 inhibitor therapy. The goal is to see if the combination remains safe and …

This study tracks 132 people with PNH who are taking Fabhalta to see how safe it is and what side effects occur. Researchers will monitor infections and check for serious red blood cell breakdown if the drug is stopped. The goal is to better understand the drug's risks in everyda…

This early-phase trial tests whether adding special immune cells called veto cells to a stem cell transplant can help donor cells grow in the patient without causing severe graft-versus-host disease (GVHD). The study includes 16 people with various blood cancers or bone marrow fa…

This study follows 64 Polish adults with paroxysmal nocturnal hemoglobinuria (PNH) who are taking ravulizumab as part of their routine care. Researchers will track blood markers like LDH and how many patients need blood transfusions over time. The goal is to see if the drug works…

This early study tests whether elotuzumab can help people with a specific type of myelofibrosis, a serious bone marrow disorder. The drug targets a protein on cancer cells to try to improve blood counts, shrink an enlarged spleen, and ease symptoms. The trial includes 15 adults w…

This study planned to test the drug hetrombopag in people with non-severe aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. The goal was to see if the drug could safely raise platelet counts. However, the trial was withdrawn before enrolling any …

This study was designed to test whether combining cyclosporine with drugs that boost platelet production (eltrombopag, hetrombopag, or romiplostim) could help people with newly diagnosed severe aplastic anemia who need blood transfusions. The trial was withdrawn before enrolling …

This trial aimed to compare the safety and effectiveness of adding luspatercept to cyclosporine versus cyclosporine alone in adults newly diagnosed with non-severe aplastic anemia (NSAA). The goal was to see if the combination could improve blood cell counts better than the stand…

This study was designed to test whether romiplostim N01 could help people with aplastic anemia caused by chemotherapy or radiation therapy. The trial was withdrawn before enrolling any participants, so no results are available. It was planned as a small, early-stage study at a si…

This study was designed to test whether adding enarodustat to cyclosporine helps people with a type of aplastic anemia who need regular blood transfusions. The trial was withdrawn before enrolling any participants, so no results are available. It would have compared the combinati…

This study tests a pill called ruxolitinib for people whose immune system attacks their bone marrow, causing conditions like severe anemia. The drug aims to stop this attack and help the bone marrow recover. Participants take the pill twice daily for up to 6 months and are follow…

This study tests whether adding the drug itacitinib to the usual two-drug regimen (cyclophosphamide and tacrolimus) can better prevent graft-versus-host disease (GVHD) in people receiving stem cell transplants for blood cancers. GVHD is a serious complication where donor cells at…

This study tests a stem cell transplant from a matched unrelated donor for people with advanced blood cancers or blood disorders. Patients get chemotherapy and radiation before the transplant to prepare their body, then take drugs to prevent the donor cells from attacking their o…

This phase 2 trial compares a drug combo (ASTX727 plus iadademstat) against ASTX727 alone for people with advanced myeloproliferative neoplasms (MPNs), a group of rare blood cancers. The study aims to see if adding iadademstat improves complete response rates. However, the trial …

This pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…

This phase 2 trial tests whether adding the drug ruxolitinib to standard care can prevent graft-versus-host disease (GVHD) in older adults with acquired aplastic anemia who receive a bone marrow transplant. GVHD is a common and serious complication where donor immune cells attack…

This phase 3 trial compares two standard treatments for severe aplastic anemia (SAA) in patients up to age 25 who lack a matched sibling donor: unrelated donor bone marrow transplant (URD BMT) versus immune suppressive therapy (IST). The study measures which approach better preve…

This study tests a new medicine, crovalimab, for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease that destroys red blood cells. It involves healthy volunteers and people with PNH to check safety and how well the drug works. The goal is to control the disease, not …

This phase 3 trial tests crovalimab in about 50 people with PNH who have not used similar drugs before. PNH causes red blood cells to break apart, leading to fatigue, pain, and need for transfusions. Crovalimab is given as an IV infusion and then as injections under the skin to b…

This phase 2 trial tests canakinumab, an anti-inflammatory drug, in 14 to 26 people with myelofibrosis, a rare bone marrow cancer that causes scarring, enlarged spleen, and fatigue. Participants receive injections every three weeks for about six months. The study checks if the dr…

This study tests a new drug called BMS-986158, either alone or combined with standard treatments (ruxolitinib or fedratinib), in about 216 people with high-risk myelofibrosis, a type of blood cancer. The goal is to see if the drug is safe and can shrink an enlarged spleen, a comm…

This early-stage trial tests a new drug called VSA012 in about 50 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study includes people who have not used complement inhibitors before or who still have low hemoglobin …

This study tests a combination of two experimental drugs, pozelimab and cemdisiran, for people with PNH, a rare blood disease that destroys red blood cells. The goal is to see if this combo works better and is safer than current standard treatments (ravulizumab and eculizumab). A…

This phase 3 study tests a drug called pacritinib against standard treatments chosen by the doctor in people with myelofibrosis who have very low platelet counts (below 50,000). The goal is to see if pacritinib can shrink the spleen and reduce symptoms like tiredness, pain, and i…

This phase 3 trial compares crovalimab, a new injectable drug, to eculizumab in about 190 people with paroxysmal nocturnal hemoglobinuria (PNH) who are already on complement inhibitors. The study aims to see if crovalimab is as safe and effective, with a focus on side effects and…

This study tests whether crovalimab works as well as eculizumab for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 210 adults who have not had prior complement inhibitor therapy will receive either drug. The main goals are to see if crovalimab…

This phase II trial tests the drug decitabine in 21 patients with advanced myelofibrosis, a bone marrow cancer that causes scarring and anemia. The goal is to see if decitabine can improve blood counts and reduce the need for transfusions. Researchers are also monitoring side eff…

This study follows 14 people with Fanconi Anemia who previously received RP-L102 gene therapy. Researchers will monitor their health for years to see if the treatment safely improves blood counts and reduces the need for a bone marrow transplant. The goal is to understand long-te…

This phase 2 trial tests whether the drug itacitinib, given with standard care, can prevent graft-versus-host disease (GVHD) in people who receive a donor stem cell transplant for blood cancers. GVHD happens when the donor cells attack the patient's body. The study includes 31 ad…

This study tests a stem cell transplant method for people with blood cancers like leukemia or lymphoma. The approach uses chemotherapy, total body radiation, and a drug called cyclophosphamide after the transplant to prevent graft-versus-host disease, where donor cells attack the…

This study tests whether giving the drug ruxolitinib before, during, and after a donor stem cell transplant can prevent graft-versus-host disease (GVHD) in people with myelofibrosis. GVHD is a serious complication where donor cells attack the patient's body. The trial enrolls 53 …

This study follows 9 people with Fanconi Anemia who already received a gene therapy that adds a working FANCA gene to their blood stem cells. Researchers will check their health and blood counts for 15 years to see if the treatment remains safe and keeps working. No new treatment…

This study tests whether combining ruxolitinib and thalidomide can shrink the cancer and improve symptoms in 30 people with myelofibrosis, a rare bone marrow disease. Participants include those who have not responded to prior treatment or are newly diagnosed with higher-risk dise…

This study tests whether adding eltrombopag to standard immunosuppressive therapy can improve blood cell counts in people with untreated severe aplastic anemia. Participants receive horse-ATG and cyclosporine plus eltrombopag for up to 6 months. The goal is to see if this combina…

This study tests whether adding the drug selinexor to the standard treatment ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a type of bone marrow cancer. About 353 adults who have not yet been treated for their condition will take either the combin…

This early-stage trial tests an experimental drug called INCB057643, given alone or with the standard drug ruxolitinib, in about 140 adults with myelofibrosis or other advanced blood cancers. The main goal is to check safety and side effects, while also measuring if the drug can …

This early-stage study tests a new drug called IOA-244 in about 210 adults with advanced or metastatic cancers like lung cancer, melanoma, and lymphoma. The main goal is to see if the drug is safe and tolerable, both alone and combined with other treatments. Researchers also chec…

This study tests a new drug (WJ01024) combined with an existing drug (ruxolitinib) in 33 adults with intermediate- or high-risk myelofibrosis whose spleen is enlarged and who didn't respond well to or couldn't tolerate previous JAK inhibitor treatments. The goal is to see if the …

This study tests a bone marrow transplant method that avoids harsh chemotherapy and radiation for people with dyskeratosis congenita, a genetic disorder causing bone marrow failure. The goal is to fix the blood system without worsening lung or liver disease or raising cancer risk…

This study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…

This early-phase trial tests a combination of three drugs—azacitidine, venetoclax, and pevonedistat—in adults newly diagnosed with a challenging form of acute myeloid leukemia (AML). The goal is to find the best dose and see if the combo can improve remission rates. About 40 part…

This study tests whether adding the drug abatacept to standard care can prevent graft-versus-host disease (GVHD) in children receiving stem cell transplants from unrelated donors. GVHD occurs when donor cells attack the patient's body, causing serious illness. The trial will enro…

This study follows 50 adults with PNH who take danicopan along with their usual medication (eculizumab or ravulizumab). Researchers track serious side effects and infections over time to see if the combination is safe for long-term use. Participants are already in a PNH registry …

This Phase 3 trial tested a stem cell transplant using blood stem cells from a half-matched (haploidentical) donor in 21 people with various blood cancers. The goal was to see if this approach could help patients who don't have a fully matched donor. Participants received chemoth…

This study looks at the long-term safety of danicopan when added to standard treatments (Soliris or Ultomiris) for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers will analyze data from 50 adult patients in an international registry, trac…

This study tests whether a lower dose of the drug cyclophosphamide, given after a stem cell transplant from a partially matched donor, can reduce serious infections in the first 100 days while still preventing graft-versus-host disease (a common complication where donor cells att…

This early-phase study tests the safety of combining three drugs—navitoclax, venetoclax, and decitabine—for people with advanced myeloid blood cancers like leukemia and myelofibrosis. The goal is to find a safe dose that can control the disease. Sixteen adults aged 18 and older a…

This study tests a modified plan to prevent and treat cytomegalovirus (CMV) infection in people with blood cancers who have had a donor stem cell transplant. CMV is a common virus that can cause serious problems after transplant. The study involves 153 participants and looks at w…

This study looks at two treatment options for people with a high-risk form of myelofibrosis, a rare bone marrow cancer. Participants choose whether they want a stem cell transplant or the best non-transplant treatments available. Researchers then track survival and quality of lif…

This study tests whether the drug sirolimus can prevent severe aplastic anemia from coming back after patients stop taking cyclosporine. About 84 people aged 2 and older who have responded to initial treatment will be randomly assigned to either take sirolimus for 3 months or jus…

This study tests an experimental drug called LP-005 in 30 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The goal is to see if LP-005 can lower a key disease marker (LDH) and raise hemoglobin levels without needing bloo…

This study tests whether adding the experimental drug navitoclax to the standard drug ruxolitinib can shrink the spleen and reduce symptoms better than other available treatments in adults with myelofibrosis that has returned or not improved. About 330 participants will be random…

This early-stage trial tests a new antibody drug called Hu8F4 in 72 people with advanced blood cancers like leukemia and myelodysplastic syndrome. The goal is to find the safest dose and see if it can slow or stop cancer growth. Researchers will also track how long patients live …

This phase 2 study tests the drug olaparib in 114 people with metastatic breast cancer whose tumors have mutations in certain DNA repair genes (like BRCA1, BRCA2, ATM, PALB2, and others). Olaparib is a PARP inhibitor that may kill cancer cells by blocking their ability to fix dam…

This early-phase trial tests a combination of two treatments for people whose myeloid blood cancer (like AML or MDS) has returned after a matched stem cell transplant. The treatments are a specially processed donor immune cell infusion and an immunotherapy drug called ipilimumab.…

This study looks at the long-term safety of a drug called HRS-5965 for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It includes 132 patients who previously took the drug and benefited from it. Researchers will track side effects and measure how we…

This phase 2 trial tests a gene therapy called RP-L102 for children with Fanconi anemia subtype A, a rare genetic disorder that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and infuse the corrected cells back. The goal…

This Phase 3 trial tests whether adding pelabresib to the standard drug ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a rare bone marrow disorder. About 430 adults who have not had prior JAK inhibitor treatment will take either the combination or …

This study is testing the safety of a drug called ATGAM in one patient with moderate to severe aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. The patient receives ATGAM by IV for 4 days and is monitored for 24 weeks for side effects. The goal …

This study tests a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…

This study tests a drug called fedratinib in Japanese adults with a serious bone marrow disease called myelofibrosis. The goal is to see if the drug can shrink an enlarged spleen and control the disease. About 31 people will take part, and the study will check safety and the best…

This program offers continued access to a combination of two drugs, pozelimab and cemdisiran, for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare condition where red blood cells break apart. It is for patients who have already completed a related study and aims to m…

This Phase 2 trial tests NTQ5082, an oral drug, in 24 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder where red blood cells break apart. The drug blocks a part of the immune system to prevent this destruction. The main goal is to see if it can raise h…

This study tests a new approach to stem cell transplants for people with blood cancers like leukemia and lymphoma. Patients receive a combination of umbilical cord blood and stem cells from a family member, with the cord blood chosen based on specific genetic markers rather than …

This study tests whether special lights and cameras can help find early signs of mouth cancer in people at high risk. About 338 participants with precancerous spots or conditions like Fanconi anemia will have their mouths examined with fluorescence imaging, which makes abnormal c…

This early-phase study tests whether vaccinating stem cell donors against cytomegalovirus (CMV) can prevent the virus from causing problems in blood cancer patients after a transplant. About 34 donor-recipient pairs are taking part. The vaccine is given to the donor before stem c…

This pilot study tests whether a daily mindful music listening program is practical and acceptable for patients hospitalized after an allogeneic stem cell transplant. About 35 adults will listen to music for one hour each day using a web-based platform. The study measures how fea…

This study looks at whether adding art therapy to physical therapy can help children aged 5 to 18 recover emotionally and physically after a stem cell transplant. For two weeks, some children receive both art and physical therapy daily, while others get only physical therapy. Res…

This study tests whether adding methadone to usual morphine-based pain medicine reduces severe mouth pain in children aged 6–18 who are getting a stem cell transplant for leukemia, aplastic anemia, or similar conditions. The mouth pain is a common side effect of the chemotherapy …

This study tests whether offering online educational videos and mailed saliva genetic testing kits helps more first-degree relatives of people with BRCA mutations get tested, compared to standard care with a family letter. About 820 participants will be enrolled across several me…

This study looks at how patients with advanced blood cancers (like acute myeloid leukemia) who are not eligible for a cure, along with their nurses and doctors, feel about the benefits of blood transfusions. Researchers will give questionnaires before each transfusion to compare …

This study follows 50 young men who had cancer or blood disorders as children and received treatments that can harm fertility. Some stored testicular tissue before treatment; others did not. Researchers will check their fertility through exams, ultrasounds, blood tests, and semen…

This study follows about 40 people with intermediate or high-risk myelofibrosis who are already taking gecacitinib. Researchers will collect bone marrow and blood samples each year to see how the disease and gene mutations change over time. No new drugs are given—this is an obser…

This study was designed to observe how patients with hereditary tyrosinemia type 1 (HT-1) in China respond to nitisinone treatment in everyday medical practice. It planned to track serious health events like liver problems or death. However, the study was withdrawn before enrolli…

This study aims to understand the genetic and molecular changes in people with bone marrow failure disorders. Researchers will analyze blood and bone marrow samples from up to 1,400 participants to track how these changes evolve over time. The goal is to better predict disease ou…

This study examines whether people with certain blood cancers (myeloproliferative neoplasms) have autoantibodies that block a key immune protein called type I interferon. These autoantibodies may increase the risk of severe COVID-19. Researchers will test blood samples from 219 p…

This study is collecting information on quality of life and symptoms from 572 Italian patients with certain types of blood cancers called myeloproliferative neoplasms (MPNs). Participants fill out questionnaires about how their disease and treatments affect their daily functionin…

This study was designed to see if a new monitoring algorithm could detect early signs of ovarian damage in women and girls who had cancer treatment. Researchers planned to compare a group using the new algorithm with a group that received standard care. However, the study was wit…

This study follows 36 patients with aplastic anemia who previously received romiplostim in earlier trials. Researchers will monitor blood cell counts and safety for up to 5 years to see if benefits last and to check for any new health issues. The goal is to better understand the …