Hematologic disorder
MONDO:0005570A disease involving the hematopoietic system.
Also known as: blood disease, blood disorder, disease of hematopoietic system, disease of the blood and blood-forming organs, disease or disorder of haematopoietic system, disease or disorder of hematopoietic system, disorder of haematopoietic system, disorder of hematopoietic system
8525 clinical trials for this condition and its sub-types, 176 tagged with Hematologic disorder itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Hematologic disorder
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Hematopoietic and lymphoid system neoplasm 692 trials · 6,335 incl. sub-types
9 sub-types
- Hematopoietic and lymphoid cell neoplasm 486 trials · 5,943 incl. sub-types Sub-types →
- Lymphoid hemopathy 0 trials · 4,285 incl. sub-types Sub-types →
- Myeloid hemopathy 0 trials · 1,765 incl. sub-types Sub-types →
- Bone marrow neoplasm 3 trials · 1,633 incl. sub-types Sub-types →
- Thymus neoplasm 5 trials · 240 incl. sub-types Sub-types →
- Spleen neoplasm 0 trials · 232 incl. sub-types Sub-types →
- Lymph node neoplasm 5 trials · 60 incl. sub-types Sub-types →
- STAT3-related early-onset multisystem autoimmune disease 2 trials
- Cavernous hemangioma 1 trial Sub-types →
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Leukocyte disorder 6 trials · 3,581 incl. sub-types
23 sub-types
- B-cell neoplasm 305 trials · 3,174 incl. sub-types Sub-types →
- T-cell leukemia 9 trials · 137 incl. sub-types Sub-types →
- B cell deficiency 5 trials · 86 incl. sub-types Sub-types →
- Leukopenia 6 trials · 78 incl. sub-types Sub-types →
- Dendritic cell sarcoma 0 trials · 53 incl. sub-types Sub-types →
- Eosinophil disorder 2 trials · 51 incl. sub-types Sub-types →
- Langerhans cell histiocytosis 40 trials · 41 incl. sub-types Sub-types →
- Phagocyte bactericidal dysfunction 0 trials · 37 incl. sub-types Sub-types →
- Mastocytosis 15 trials · 34 incl. sub-types Sub-types →
- Functional neutrophil defect 0 trials · 13 incl. sub-types Sub-types →
- Mast cell activation syndrome 8 trials · 9 incl. sub-types Sub-types →
- POEMS syndrome 8 trials
- Subcutaneous panniculitis-like T-cell lymphoma 8 trials
- EBV-positive T-cell lymphoproliferative disorder of childhood 3 trials · 7 incl. sub-types Sub-types →
- Thymoma type B 0 trials · 4 incl. sub-types Sub-types →
- Human granulocytic anaplasmosis 1 trial
- Pelger-Huet anomaly 0 trials
- Eosinophil peroxidase deficiency 0 trials
- Hereditary neutrophilia 0 trials
- Human monocytic ehrlichiosis 0 trials
- Leukostasis 0 trials
- Pituitary gland basophil adenoma 0 trials
- Small intestinal enteropathy-associated T-cell lymphoma 0 trials
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Bone marrow disorder 18 trials · 1,763 incl. sub-types
7 sub-types
- Bone marrow neoplasm 3 trials · 1,633 incl. sub-types Sub-types →
- Polycythemia 9 trials · 99 incl. sub-types Sub-types →
- Bone marrow failure syndrome 48 trials Sub-types →
- Osteomyelitis 43 trials · 46 incl. sub-types Sub-types →
- Fanconi anemia 29 trials · 42 incl. sub-types Sub-types →
- Drachtman Weinblatt Sitarz syndrome 0 trials
- Premature ovarian failure 15 0 trials
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Anemia 258 trials · 975 incl. sub-types
19 sub-types
- Aplastic anemia 110 trials · 411 incl. sub-types Sub-types →
- Normocytic anemia 0 trials · 180 incl. sub-types Sub-types →
- Microcytic anemia 0 trials · 130 incl. sub-types Sub-types →
- Hypochromic anemia 0 trials · 123 incl. sub-types Sub-types →
- Beta-thalassemia and related diseases 0 trials · 99 incl. sub-types Sub-types →
- Congenital anemia 0 trials · 58 incl. sub-types Sub-types →
- Anemia due to chronic disorder 34 trials
- Pure red-cell aplasia 5 trials · 24 incl. sub-types Sub-types →
- Neonatal anemia 7 trials · 23 incl. sub-types Sub-types →
- Deficiency anemia 3 trials · 16 incl. sub-types Sub-types →
- Pancytopenia 15 trials
- Anemia due to enzyme disorder 1 trial · 14 incl. sub-types Sub-types →
- Sideroblastic anemia 0 trials · 14 incl. sub-types Sub-types →
- Macrocytic anemia 0 trials · 11 incl. sub-types Sub-types →
- Hemoglobin C disease 2 trials
- Hereditary methemoglobinemia 0 trials · 1 incl. sub-types Sub-types →
- Hemoglobin D disease 0 trials
- Hemoglobin E disease 0 trials
- Hemoglobinopathy Toms River 0 trials
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Erythrocyte disorder 7 trials · 618 incl. sub-types
2 sub-types
- Hemoglobinopathy 34 trials · 446 incl. sub-types Sub-types →
- Malaria 135 trials · 170 incl. sub-types Sub-types →
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Blood coagulation disease 54 trials · 541 incl. sub-types
8 sub-types
- Inherited blood coagulation disorder 8 trials · 323 incl. sub-types Sub-types →
- Coagulation protein disease 13 trials · 274 incl. sub-types Sub-types →
- Thrombotic microangiopathy 26 trials · 151 incl. sub-types Sub-types →
- Thrombophilia 12 trials · 75 incl. sub-types Sub-types →
- Hemolytic-uremic syndrome 7 trials · 24 incl. sub-types Sub-types →
- Hemorrhagic disease of newborn 1 trial
- Marantic endocarditis 0 trials
- Prekallikrein deficiency 0 trials Sub-types →
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Hemorrhagic disease 108 trials · 510 incl. sub-types
28 sub-types
- Hemophilia A 180 trials Sub-types →
- Purpura 4 trials · 117 incl. sub-types Sub-types →
- Hemophilia B 84 trials · 91 incl. sub-types Sub-types →
- Vascular hemostatic disease 62 trials · 63 incl. sub-types Sub-types →
- Hereditary von Willebrand disease 18 trials · 29 incl. sub-types Sub-types →
- Inherited bleeding disorder, platelet-type 6 trials · 26 incl. sub-types Sub-types →
- Acquired hemophilia 2 trials · 8 incl. sub-types Sub-types →
- Congenital fibrinogen deficiency 2 trials · 6 incl. sub-types Sub-types →
- Factor VII deficiency 6 trials Sub-types →
- Congenital vitamin K-dependent coagulation factors deficiency 0 trials · 4 incl. sub-types Sub-types →
- Fetal and neonatal alloimmune thrombocytopenia 4 trials Sub-types →
- Acquired von willebrand syndrome 3 trials
- Factor X deficiency 2 trials Sub-types →
- Congenital factor XII deficiency 1 trial
- Congenital plasminogen activator inhibitor type 1 deficiency 1 trial
- East Texas bleeding disorder 0 trials
- Alpha-2-plasmin inhibitor deficiency 0 trials
- Combined deficiency of factor V and factor VIII 0 trials Sub-types →
- Congenital factor V deficiency 0 trials
- Congenital factor XI deficiency 0 trials
- Congenital factor XIII deficiency 0 trials Sub-types →
- Congenital high-molecular-weight kininogen deficiency 0 trials
- Hemophilia B leyden 0 trials
- Hemorrhagic disease due to alpha-1-antitrypsin Pittsburgh mutation 0 trials
- Inherited prekallikrein deficiency 0 trials
- Multiple sclerosis-ichthyosis-factor VIII deficiency syndrome 0 trials
- Prothrombin deficiency 0 trials Sub-types →
- Thrombomodulin-related bleeding disorder 0 trials
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Blood platelet disease 18 trials · 370 incl. sub-types
5 sub-types
- Thrombocytopenia 75 trials · 267 incl. sub-types Sub-types →
- Thrombocytosis disease 7 trials · 71 incl. sub-types Sub-types →
- Inherited bleeding disorder, platelet-type 6 trials · 26 incl. sub-types Sub-types →
- Qualitative platelet defect 6 trials
- TPM4-related platelet disorder 0 trials
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Splenic disorder 3 trials · 248 incl. sub-types
8 sub-types
- Spleen neoplasm 0 trials · 232 incl. sub-types Sub-types →
- Hypersplenism 12 trials
- Splenic sequestration 1 trial
- Congestive splenomegaly 0 trials Sub-types →
- Splenic abscess 0 trials
- Splenic infarction 0 trials
- Splenic tuberculosis 0 trials
- Wandering spleen 0 trials
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Thymus gland disorder 1 trial · 240 incl. sub-types
3 sub-types
- Thymus neoplasm 5 trials · 240 incl. sub-types Sub-types →
- Ectopic thymus 0 trials
- Thymic dysplasia 0 trials
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Autoimmune disorder of blood 0 trials · 152 incl. sub-types
2 sub-types
- Primary thrombocytopenia 27 trials · 110 incl. sub-types Sub-types →
- Autoimmune hemolytic anemia 49 trials · 72 incl. sub-types Sub-types →
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Congenital hematological disorder 1 trial · 101 incl. sub-types
21 sub-types
- Congenital anemia 0 trials · 58 incl. sub-types Sub-types →
- Constitutional neutropenia 2 trials · 28 incl. sub-types Sub-types →
- Congenital thrombotic thrombocytopenic purpura 5 trials
- Congenital vitamin K-dependent coagulation factors deficiency 0 trials · 4 incl. sub-types Sub-types →
- GNE myopathy 3 trials
- Congenital amegakaryocytic thrombocytopenia 1 3 trials
- Leukocyte adhesion deficiency type II 2 trials
- Congenital factor XII deficiency 1 trial
- Congenital plasminogen activator inhibitor type 1 deficiency 1 trial
- Thrombocytopenia-absent radius syndrome 1 trial
- Congenital agammaglobulinemia 0 trials Sub-types →
- Congenital analbuminemia 0 trials
- Congenital factor XI deficiency 0 trials
- Congenital factor XIII deficiency 0 trials Sub-types →
- Congenital progressive bone marrow failure-B-cell immunodeficiency-skeletal dysplasia syndrome 0 trials
- Congenital secondary polycythemia 0 trials Sub-types →
- Hereditary thrombocytosis with transverse limb defect 0 trials
- Hypercoagulability syndrome due to glycosylphosphatidylinositol deficiency 0 trials
- Macrothrombocytopenia-lymphedema-developmental delay-facial dysmorphism-camptodactyly syndrome 0 trials
- Radio-ulnar synostosis-amegakaryocytic thrombocytopenia syndrome 0 trials Sub-types →
- Sulfhemoglobinemia, congenital 0 trials
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Monoclonal gammopathy 24 trials · 68 incl. sub-types
3 sub-types
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Premalignant hematological system disease 3 trials · 25 incl. sub-types
2 sub-types
- Clonal hematopoiesis 10 trials · 22 incl. sub-types Sub-types →
- Atypical lymphoproliferative disorder 0 trials
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Paraneoplastic hematological syndrome 0 trials · 9 incl. sub-types
2 sub-types
- POEMS syndrome 8 trials
- Humoral hypercalcemia of malignancy 1 trial
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Parasitemia 5 trials
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Blood group incompatibility 2 trials
1 sub-type
- Rh isoimmunization 0 trials
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L-ferritin deficiency 1 trial
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Hyperamylasemia 1 trial
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Bloom syndrome 0 trials
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GATA1-Related X-Linked Cytopenia 0 trials
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Polyclonal hyperviscosity syndrome 0 trials
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Septicemic plague 0 trials
Most studied deeper sub-types
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New drug aims to cut sickle cell pain crises in half
Disease control OngoingThis phase 3 study tests two doses of crizanlizumab against a placebo in 255 adolescents and adults with sickle cell disease who have frequent pain crises. The goal is to see if the drug can reduce the number of severe pain episodes that require a hospital or clinic visit. Partic…
Phase 3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Sep 16, 2026 00:00 UTC
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Could dead donors provide Life-Saving bone marrow?
Disease control OngoingThis early study is testing whether bone marrow from deceased donors can be safely used for transplants in people with blood cancers like leukemia and lymphoma. Twelve patients will receive the transplant along with chemotherapy or radiation. The main goal is to see if the new ma…
Phase 1/2 • Sponsor: Ossium Health, Inc. • Aim: Disease control
Last updated Sep 12, 2026 00:00 UTC
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New hope for anemia patients: drug may cut need for blood transfusions
Disease control OngoingThis study tests a drug called luspatercept in people with anemia caused by myelodysplastic syndromes (MDS) or beta-thalassemia who need regular blood transfusions. The goal is to see if the drug is safe and can reduce the number of transfusions needed. About 85 participants in I…
Phase 4 • Sponsor: Bristol-Myers Squibb • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
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Could a daily pill help kids with rare blood disorder?
Disease control OngoingThis study tests a drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The trial compares mitapivat to a placebo to see if it can raise hemoglobin levels…
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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Could a pill replace blood transfusions for kids with rare anemia?
Disease control OngoingThis phase 3 trial tests the drug mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly. These children need regular blood transfusions. The study compares mitapivat to a placebo to see if…
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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Lower drug dose after transplant may cut infections without losing protection
Disease control OngoingThis 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…
Phase 2 • Sponsor: Center for International Blood and Marrow Transplant Research • Aim: Disease control
Last updated Aug 19, 2026 00:00 UTC
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New combo therapy targets high-risk MDS in early trial
Disease control CancelledThis study tests a new two-drug combination (pacritinib plus azacitidine) for people with moderate to very high risk myelodysplastic syndrome (MDS), a bone marrow disorder. The first part finds the safest dose of pacritinib, and the second part checks how well the combination wor…
Phase 1/2 • Sponsor: Thomas Jefferson University • Aim: Disease control
Last updated Aug 19, 2026 00:00 UTC
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CRISPR gene therapy: a Long-Term safety check for blood disease patients
Disease control By invitation onlyThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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Cow-Hemoglobin infusion could save lives when blood is not an option
Disease control Expanded access (ended)This study tests HBOC-201, an oxygen-carrying fluid made from cow hemoglobin, in adults with life-threatening anemia who cannot receive red blood cell transfusions. Participants have dangerously low hemoglobin levels and signs of oxygen deprivation. The goal is to see if HBOC-201…
Sponsor: Michael Boisen • Aim: Disease control
Last updated Jul 22, 2026 00:00 UTC
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New drug shows promise for controlling rare skin cancers
Disease control OngoingThis study tests a drug called brentuximab vedotin in 79 people with rare skin lymphomas (ALCL, mycosis fungoides, or lymphomatoid papulosis) that have a marker called CD30. The goal is to see if the drug can shrink or control the cancer. Participants receive the drug every 3 wee…
Phase 2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC
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Can a drug combo stop AML from coming back?
Disease control OngoingThis phase II trial tests whether a combination of two drugs, azacitidine and venetoclax, can help keep acute myeloid leukemia (AML) in remission after initial treatment. The study enrolls 50 adults whose AML is in first remission but who are not ready for a stem cell transplant.…
Phase 2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC
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CRISPR stem cell therapy shows promise for kids with sickle cell
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive the…
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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CRISPR gene therapy aims to free kids from lifelong blood transfusions
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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New hope for rare bleeding disorder: coagadex trial targets AL amyloidosis
Disease control CancelledThis phase 3 study tests Coagadex, a human clotting factor X, in 15 adults with acquired factor X deficiency caused by AL amyloidosis. The goal is to see if it safely stops active bleeding and helps manage bleeding during surgery. Participants receive the drug on demand for bleed…
Phase 3 • Sponsor: Kedrion S.p.A. • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC
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One-Time gene therapy could slash bleeding in hemophilia b
Disease control OngoingThis study tests a one-time gene therapy for adult men with moderate to severe hemophilia B, a bleeding disorder. The therapy delivers a working gene to help the body produce a clotting protein called Factor IX. Researchers will track how many bleeding episodes participants have …
Phase 3 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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New hope for blood cancer patients: experimental drug takes on standard therapy
Disease control OngoingThis study tests a new drug called pirtobrutinib against other approved drugs for mantle cell lymphoma, a type of blood cancer. About 500 people who have already had at least one prior treatment will take part. The goal is to see if the new drug works better at controlling the di…
Phase 3 • Sponsor: Loxo Oncology, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:34 UTC
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Shorter treatment course may ease side effects for AML patients
Disease control OngoingThis study tests a new way of giving two drugs—venetoclax and azacitidine—to adults with acute myeloid leukemia (AML) who are not healthy enough for standard chemotherapy. Instead of the usual 7 days of azacitidine, this approach uses only 5 days per cycle to try to reduce infect…
Phase 2 • Sponsor: University of Leipzig • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC
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New hope for myelofibrosis patients with dangerous low platelet counts
Disease control OngoingThis 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…
Phase 3 • Sponsor: Swedish Orphan Biovitrum • Aim: Disease control
Last updated Jun 27, 2026 12:26 UTC
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New hope for Hard-to-Treat blood cancers: pirtobrutinib takes on standard therapy
Disease control OngoingThis phase 3 study compares a new oral drug called pirtobrutinib (LOXO-305) against two standard treatment combinations (idelalisib plus rituximab or bendamustine plus rituximab) in 238 adults with chronic lymphocytic leukemia or small lymphocytic lymphoma who have already been t…
Phase 3 • Sponsor: Loxo Oncology, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:23 UTC
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New drug aims to stop transplant attack on the body
Disease control OngoingThis 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…
Phase 2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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New Immune-Boosting drug aims to outperform standard therapy in myeloma
Disease control OngoingThis Phase 3 trial tests whether elranatamab, an immune-boosting drug, works better than lenalidomide at preventing multiple myeloma from returning in patients who have just had a stem cell transplant. About 854 participants will receive either elranatamab injections or lenalidom…
Phase 3 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC
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Sickle cell drug pociredir tested for Long-Term safety in 50 patients
Disease control By invitation onlyThis study looks at the long-term safety of the drug pociredir in 50 adults with sickle cell disease who have already taken it and seen benefits in an earlier study. Participants will take pociredir once daily for up to 48 months. The main goal is to track side effects and other …
Phase 2 • Sponsor: Fulcrum Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC
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New pill could slash sickle cell pain crises
Disease control OngoingThis large Phase 3 trial is testing an oral drug called etavopivat in 450 people with sickle cell disease. The goal is to see if taking a pill once daily can raise hemoglobin levels and reduce the number of painful vaso-occlusive crises compared to a placebo. Participants must ha…
Phase 3 • Sponsor: Forma Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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One-Time gene therapy could free hemophilia patients from frequent infusions
Disease control OngoingThis study tests a one-time gene therapy (valoctocogene roxaparvovec) in 6 Japanese men with severe hemophilia A. The goal is to help their bodies produce their own clotting factor, reducing or stopping the need for regular factor VIII infusions. Participants will be monitored fo…
Phase 3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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New hope for blood cancer patients: using mismatched donors for transplants
Disease control OngoingThis study tests a stem cell transplant using donors who are not a perfect match for patients with blood cancers like leukemia and lymphoma. Doctors give special drugs after the transplant to help prevent the donor cells from attacking the patient's body. The goal is to see if th…
Phase 2 • Sponsor: Center for International Blood and Marrow Transplant Research • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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Targeted radiation shows promise for Hard-to-Treat blood cancers
Disease control OngoingThis study tests an experimental drug called iopofosine I 131 in people with certain blood cancers (like Waldenstrom macroglobulinemia, multiple myeloma, and lymphomas) that have come back or not responded to standard treatments. The drug delivers radiation directly to cancer cel…
Phase 2 • Sponsor: Cellectar Biosciences, Inc. • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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Fitness test could guide safer stem cell transplants for seniors
Disease control OngoingThis study tests whether a geriatric assessment—looking at thinking, mobility, mood, nutrition, and medications—can help doctors choose the right transplant intensity for patients aged 60 and older with myeloid blood cancers. The goal is to reduce deaths from transplant complicat…
Phase 2 • Sponsor: Memorial Sloan Kettering Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 09:06 UTC
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Could a cancer drug shield young transplant patients from a deadly complication?
Disease control OngoingThis study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children, adolescents, and young adults receiving a bone marrow or blood stem cell transplant for blood cancers. Researchers are first finding the best dose of vor…
Phase 1/2 • Sponsor: University of Michigan Rogel Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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New hope for blood cancer patients: triple therapy aims to keep disease in check longer
Disease control OngoingThis study tests whether adding a new targeted drug (pirtobrutinib) to a standard two-drug combination (venetoclax and rituximab) can help people with chronic lymphocytic leukemia or small lymphocytic lymphoma live longer without their cancer getting worse. About 600 adults who h…
Phase 3 • Sponsor: Loxo Oncology, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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Frozen testicle tissue may help infertile men have kids
Disease control OngoingThis experimental study tests whether frozen-thawed testis tissue, when grafted under the skin, can produce sperm in men with infertility. Five men will receive their own stored tissue. The goal is to see if viable sperm can be retrieved, potentially offering a future fertility o…
Sponsor: Herlev and Gentofte Hospital • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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New pill could outshine chemo for common blood cancer
Disease control OngoingThis phase 3 trial compares a new oral targeted drug, pirtobrutinib, against a standard chemo-immunotherapy combination (bendamustine plus rituximab) in people with untreated chronic lymphocytic leukemia or small lymphocytic lymphoma. About 309 participants will be randomly assig…
Phase 3 • Sponsor: Loxo Oncology, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Gene therapy for hemophilia a: how long does it last?
Disease control By invitation onlyThis study checks the long-term safety and effectiveness of a one-time gene therapy (BMN 270) for people with severe hemophilia A. Participants from earlier BioMarin trials will be followed for many years to see if the treatment continues to work and remains safe. The goal is to …
Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Can a psoriasis drug stop transplant rejection?
Disease control OngoingThis phase II trial tests whether the drug ustekinumab (Stelara) can prevent acute graft-versus-host disease (GVHD) in 116 adults receiving stem cell transplants for blood cancers. GVHD occurs when donor immune cells attack the patient's body. Ustekinumab aims to calm this immune…
Phase 2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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Engineered immune cells aim to tame chronic GVHD
Disease control PausedThis early-phase study tests a new treatment called CD6-CAR Tregs for people with chronic graft-versus-host disease (cGVHD), a complication after a stem cell transplant. The treatment uses specially engineered immune cells from a donor to help control the immune attack on the pat…
Phase 1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 07:57 UTC
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Gene therapy patients monitored for years to ensure lasting safety
Disease control OngoingThis study follows 8 people who received a one-time gene therapy (BIVV003 for sickle cell disease or ST-400 for beta-thalassemia) to see if it remains safe and effective over many years. Researchers will track serious side effects, survival, and blood markers like hemoglobin leve…
Sponsor: Sangamo Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:57 UTC
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Last-Resort treatment opens for rare swallowing disorder
Disease control Expanded access (ended)This program offers APT-1011, a fluticasone tablet that dissolves in the mouth, to patients aged 12 and older with eosinophilic esophagitis who cannot join other studies or have not responded to available treatments. The goal is to provide access until the drug is commercially av…
Sponsor: Ellodi Pharmaceuticals, LP • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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Promising ITP drug trial pulled before it even started
Disease control CancelledThis study was designed to test the safety and tolerability of a new drug called HMPL-523 (sovleplenib) in adults with immune thrombocytopenia (ITP), a condition where the immune system destroys platelets, causing bleeding problems. The trial was withdrawn before enrolling any pa…
Phase 1 • Sponsor: Hutchmed • Aim: Disease control
Last updated Jun 26, 2026 18:23 UTC
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Virtual reality could replace painkillers for bone marrow tests
Symptom relief By invitation onlyThis trial tests whether using virtual reality (VR) headsets to distract patients can reduce pain and anxiety during bone marrow examinations, which are often uncomfortable and stressful. Participants will either use VR headsets to watch or listen to calming content, or follow th…
Sponsor: Region Skane • Aim: Symptom relief
Last updated Aug 21, 2026 00:00 UTC
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New drug aims to ease daily misery of rare mast cell disease
Symptom relief OngoingThis phase 2 study tests whether the experimental drug bezuclastinib can reduce symptoms like itching, flushing, and fatigue in people with nonadvanced systemic mastocytosis, a rare condition where too many mast cells cause chronic discomfort. About 237 adults whose symptoms are …
Phase 2 • Sponsor: Cogent Biosciences, Inc. • Aim: Symptom relief
Last updated Jun 27, 2026 12:37 UTC
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Could listening to music ease the stress of stem cell transplants?
Symptom relief OngoingThis 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…
Sponsor: University of Colorado, Denver • Aim: Symptom relief
Last updated Jun 27, 2026 12:03 UTC
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Art and exercise may boost recovery for kids after transplant
Symptom relief OngoingThis 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…
Sponsor: Medical University of South Carolina • Aim: Symptom relief
Last updated Jun 27, 2026 08:11 UTC
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Massive data collection aims to spark breakthroughs in blood disorders
Knowledge-focused By invitation onlyThis study gathers medical information from up to 10,000 people with blood or heart conditions, as well as stem cell donors, treated at the National Institutes of Health. Researchers will review participants' medical records, test results, and treatments to build a database for g…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Sep 20, 2026 00:00 UTC
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NIH opens screening study to find candidates for blood disorder research
Knowledge-focused By invitation onlyThis study screens up to 10,000 people with blood disorders or healthy volunteers to see if they can safely join other active research studies at the National Heart, Lung, and Blood Institute. Participants undergo clinical, imaging, and lab tests. If no suitable study is availabl…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Sep 20, 2026 00:00 UTC
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NIH launches massive sample collection to unlock disease secrets
Knowledge-focused By invitation onlyThis 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…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC
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Could muscle loss predict blood cancer outcomes?
Knowledge-focused OngoingThis study is investigating how common muscle loss, known as sarcopenia, is in people with blood cancers like leukemia, lymphoma, and myeloma. Researchers will measure body composition using DXA scans and track hospital stays, disease progression, and survival. The goal is to see…
Sponsor: Rigshospitalet, Denmark • Aim: Knowledge-focused
Last updated Aug 16, 2026 00:00 UTC
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Massive study tracks 32,000 oil spill cleanup workers for hidden health risks
Knowledge-focused OngoingThis study follows over 32,000 people who helped clean up the 2010 Deepwater Horizon oil spill in the Gulf of Mexico. Researchers want to see if exposure to crude oil and chemical dispersants leads to long-term health problems like heart disease, cancer, or lung issues. Participa…
Sponsor: National Institute of Environmental Health Sciences (NIEHS) • Aim: Knowledge-focused
Last updated Jul 04, 2026 00:00 UTC
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Can a simple card keep cancer patients safer?
Knowledge-focused OngoingThis study tests whether giving cancer patients a drug vigilance card helps them avoid medication errors and interactions when seeing other doctors. One hundred patients on oral chemotherapy or targeted therapies at Limoges University Hospital will use the card. Researchers will …
Sponsor: University Hospital, Limoges • Aim: Knowledge-focused
Last updated Jun 28, 2026 00:00 UTC
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Frailty check could predict chemo danger for seniors
Knowledge-focused By invitation onlyThis study looks at how frailty—a state of weakness and reduced physical function—affects the risk of severe side effects from chemotherapy in people aged 70 and older with cancer. Researchers will assess 160 patients using various tests for frailty, muscle loss, and overall heal…
Sponsor: Azienda Socio Sanitaria Territoriale Ovest Milanese, Ospedale di Legnano • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:04 UTC
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Did storing testicular tissue as a child protect fertility? new study aims to find out
Knowledge-focused By invitation onlyThis 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…
Sponsor: Universitair Ziekenhuis Brussel • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:29 UTC
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New digital tool aims to cut medical errors during hospital transfers
Knowledge-focused By invitation onlyThis study tests a new digital platform that helps doctors and nurses share important patient information when a person is moved from one hospital to another. About 1,000 adults being transferred to a major hospital will take part. The goal is to see if better information sharing…
Sponsor: Brigham and Women's Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:13 UTC
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Scientists seek tissue samples to unlock cancer mysteries
Knowledge-focused By invitation onlyThis study collects blood and tissue samples from people receiving or donating bone marrow transplants for leukemia, lymphoma, or other blood diseases. Researchers will analyze these samples to find genes and proteins linked to cancer development and transplant complications. The…
Sponsor: Stanford University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:05 UTC
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Muscle weakness may predict transplant success in blood cancer patients
Knowledge-focused OngoingThis study follows 144 blood cancer patients undergoing stem cell transplants to see how muscle function changes and whether it predicts complications like infections, relapse, or survival. Researchers will measure muscle mass and track medical events over time. The goal is to un…
Sponsor: Rigshospitalet, Denmark • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:03 UTC