Primary myelofibrosis
MONDO:0009692Myelofibrosis with myeloid metaplasia is a myeloproliferative disease with annual incidence of approximately 1 case per 100,000 individuals and age at diagnosis around 60 (an increased prevalence is noted in Ashkenazi Jews). Clinical manifestations depend on the type of blood cell affected and may include anemia, pallor, splenomegaly, hypermetabolic state, petechiae, ecchymosis, bleeding, lymphadenopathy, hepatomegaly, portal hypertension.
Also known as: AMM, Agnogenic myeloid metaplasia, CIMF, chronic idiopathic myelofibrosis, idiopathic bone marrow fibrosis, idiopathic myelofibrosis, myelofibrosis with myeloid metaplasia, somatic, myelofibrosis, somatic
3252 clinical trials for this condition and its sub-types, 191 tagged with Primary myelofibrosis itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Primary myelofibrosis
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Familial myelofibrosis 0 trials
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Panostotic fibrous dysplasia 0 trials
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Can a new pill tame myelofibrosis?
Disease control CompletedThis trial is testing an experimental oral drug called PXS-5505 (amsulostat) in people with myelofibrosis, a type of bone marrow cancer. The study focuses on patients who are not eligible for a stem cell transplant. The main goal is to see if the drug is safe and tolerable, and t…
Phase 1/2 • Sponsor: Syntara • Aim: Disease control
Last updated Aug 06, 2026 00:00 UTC
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New stem cell transplant approach shows promise for blood cancer patients without full matches
Disease control CompletedThis 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…
Phase 3 • Sponsor: Dartmouth-Hitchcock Medical Center • Aim: Disease control
Last updated Jul 19, 2026 00:00 UTC
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Could a JAK inhibitor improve stem cell transplant success in myelofibrosis?
Disease control CompletedThis phase 2 trial tests whether giving ruxolitinib (Jakafi) before, during, and after a stem cell transplant can improve outcomes for people with myelofibrosis. The study includes patients with intermediate-2 or high-risk disease, or intermediate-1 with additional risk factors. …
Phase 2 • Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated Jul 11, 2026 00:00 UTC
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Experimental drug RAD001 tested for tough leukemias and lymphomas
Disease control CompletedThis study tested the drug RAD001 (everolimus) in 29 patients with relapsed or hard-to-treat blood cancers like leukemia, lymphoma, and myelofibrosis. The goal was to find the safest highest dose and see if it helps control the disease. It was a small early-phase trial at a singl…
Phase 1/2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New drug combo shrinks spleens in bone marrow cancer patients
Disease control CompletedThis study tested a new drug called pelabresib in people with certain blood cancers, including myelofibrosis and leukemia. In the first part, the drug was given alone to find the safest dose. In the second part, it was given with or without another drug (ruxolitinib) to see if it…
Phase 1/2 • Sponsor: Constellation Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 13:02 UTC
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New pill for rare blood cancers shows promise in early trial
Disease control CompletedThis early-phase study tested a new drug called TQ05105 in 79 adults with certain blood cancers (myeloproliferative neoplasms). The main goals were to find the safest dose and understand side effects. The drug works by blocking a protein (JAK2) that can cause abnormal blood cell …
Phase 1 • Sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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New pill shows promise in shrinking spleens for myelofibrosis patients
Disease control CompletedThis study tested a new drug called TQ05105 in 107 people with moderate to high-risk myelofibrosis, a rare bone marrow disorder that often causes an enlarged spleen. The goal was to see if TQ05105 could shrink the spleen better than the standard drug hydroxyurea. Participants too…
Phase 2 • Sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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Experimental combo targets Hard-to-Treat blood cancers
Disease control CompletedThis early-phase study tested a new drug called TGR-1202 combined with the standard drug ruxolitinib in 60 people with myelofibrosis or polycythemia vera. The goal was to see if the combination is safe and tolerable. Participants either had not responded well to ruxolitinib alone…
Phase 1 • Sponsor: Vanderbilt-Ingram Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 12:35 UTC
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New Two-Step transplant shows promise for blood cancer patients
Disease control CompletedThis study tested a two-step stem cell transplant for people with blood cancers like leukemia and lymphoma. First, patients received low-dose chemotherapy and radiation to prepare their bodies. Then, they got donor stem cells followed by drugs to prevent the donor cells from atta…
Phase 2 • Sponsor: Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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New drug combo offers hope for myelofibrosis patients who failed standard therapy
Disease control CompletedThis study tested a combination of two drugs, fedratinib and nivolumab, in 30 adults with myelofibrosis whose disease no longer responded well to standard JAK-inhibitor treatment. The goal was to see if the combination could shrink the spleen, improve symptoms, or reduce the need…
Phase 2 • Sponsor: Institut für Klinische Krebsforschung IKF GmbH at Krankenhaus Nordwest • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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Personalized plans boost survival odds for older stem cell patients
Disease control CompletedThis study tested a personalized geriatric optimization plan (GO!) in 30 adults aged 60 and older who were getting a donor stem cell transplant for blood cancers or bone marrow failure. The plan included tailored advice on diet, sleep, activity, and medications to improve physica…
Sponsor: Ohio State University Comprehensive Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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New drug combo aims to tame deadly transplant complication
Disease control CompletedThis phase II trial tested whether adding the drug itacitinib to standard medications (tacrolimus and sirolimus) can better prevent graft-versus-host disease (GVHD) in 59 patients with blood cancers receiving donor stem cell transplants. GVHD occurs when donor immune cells attack…
Phase 2 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 12:02 UTC
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Could a Pre-Transplant drug boost survival in myelofibrosis?
Disease control CompletedThis phase 2 trial looked at whether giving a JAK inhibitor (ruxolitinib) before a donor stem cell transplant helps people with myelofibrosis. The study enrolled 61 patients with primary or secondary myelofibrosis. The goal was to see if this approach improves survival and reduce…
Phase 2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC
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Drug cocktail shows promise against rare bone marrow cancers
Disease control CompletedThis study tested a combination of two drugs, ruxolitinib and azacytidine, in 121 people with myelofibrosis or related blood cancers. The goal was to see if the combo could shrink tumors or improve blood counts. While the treatment aims to control the disease, it is not a cure, a…
Phase 2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC
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New combo therapy aims to shrink spleens in bone marrow cancer patients
Disease control CompletedThis study tested whether adding the experimental drug navitoclax to the standard drug ruxolitinib works better than ruxolitinib alone for people with myelofibrosis, a type of bone marrow cancer. The main goal was to see if the combination could shrink an enlarged spleen by at le…
Phase 3 • Sponsor: AbbVie • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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One donor, two transplants: could this end lifelong Anti-Rejection drugs?
Disease control CompletedThis pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…
Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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New hope for myelofibrosis patients: drug combo shows promise in shrinking spleens
Disease control CompletedThis study tested a drug called navitoclax, either alone or added to the standard drug ruxolitinib, in 191 people with myelofibrosis. The main goal was to see if the treatment could shrink the spleen by at least 35% after 24 weeks. The trial also looked at whether it could reduce…
Phase 2 • Sponsor: AbbVie • Aim: Disease control
Last updated Jun 27, 2026 08:12 UTC
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Drug dasatinib tested for rare blood cancers
Disease control CompletedThis study tested the drug dasatinib in 68 adults with several types of blood and bone marrow disorders, including certain leukemias, myelofibrosis, and mastocytosis. The goal was to see if dasatinib could help control the disease and to check its safety. Participants took dasati…
Phase 2 • Sponsor: M.D. Anderson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 08:07 UTC
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New Two-Step transplant shows promise for tough blood cancers
Disease control CompletedThis study tested a two-step stem cell transplant for people with high-risk blood cancers like leukemia and lymphoma. First, patients got low-dose chemotherapy and radiation to prepare the body. Then, they received donor stem cells and immune cells to help destroy remaining cance…
Phase 2 • Sponsor: Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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New pill shows promise for rare blood cancer in early trial
Disease control CompletedThis early-phase trial tested a new drug called flonoltinib maleate in 31 people with myeloproliferative neoplasms (MPNs), a group of bone marrow cancers. The main goals were to check safety, find the right dose, and see how the body processes the drug. Researchers hope this drug…
Phase 1/2 • Sponsor: Chengdu Zenitar Biomedical Technology Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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Bomedemstat extension trial tests Long-Term safety for blood cancer patients
Disease control CompletedThis study looks at the long-term safety of bomedemstat, an oral drug, in 81 people with myeloproliferative neoplasms (MPNs) like essential thrombocythemia and primary myelofibrosis. Participants had already taken bomedemstat in a prior study. Researchers tracked side effects, sp…
Phase 2 • Sponsor: Imago BioSciences, Inc., a subsidiary of Merck & Co., Inc., (Rahway, New Jersey USA) • Aim: Disease control
Last updated Jun 27, 2026 07:51 UTC
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New drug itacitinib tested for Hard-to-Treat myelofibrosis
Disease control CompletedThis phase 2 study tested the drug itacitinib in 4 people with myelofibrosis (a bone marrow disorder) who had already tried other treatments like ruxolitinib or fedratinib. The main goals were to check safety and find the right dose, and to see if it could shrink an enlarged sple…
Phase 2 • Sponsor: Incyte Corporation • Aim: Disease control
Last updated Jun 26, 2026 13:11 UTC
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Pens and vouchers: can small rewards boost participation in blood cancer research?
Knowledge-focused CompletedResearchers want to learn what causes myeloproliferative neoplasms, a group of rare blood cancers. They are testing whether small incentives like pens, trolley tokens, or a £10 reimbursement encourage more people to join a case-control study. The trial includes about 100 patients…
Sponsor: Queen's University, Belfast • Aim: Knowledge-focused
Last updated Sep 13, 2026 00:00 UTC
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Could a platelet-boosting drug shorten recovery after stem cell transplants?
Knowledge-focused CompletedThis study looks back at medical records of people who had an allogeneic stem cell transplant to see whether romiplostim helped their platelets recover faster. Platelets are blood cells that help clotting, and low counts are common after transplant. The researchers will compare p…
Sponsor: Tongji Hospital • Aim: Knowledge-focused
Last updated Aug 07, 2026 00:00 UTC
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Scientists probe the root cause of a rare bone marrow disease
Knowledge-focused CompletedThis completed study looked at blood vessel cells from 30 people with primary myelofibrosis, a rare bone marrow disorder. Researchers wanted to see if these cells act like stem cells that could be the origin of the disease. By growing the cells in the lab, they aimed to understan…
Sponsor: Fondazione IRCCS Policlinico San Matteo di Pavia • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:37 UTC
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Healthy volunteers needed to test new myelofibrosis drug's absorption
Knowledge-focused CompletedThis study looked at how well two different capsule forms of TP-3654 are absorbed by the body and whether taking them with food changes absorption. It involved 24 healthy adults and was completed. The results will help design future studies for people with myelofibrosis, a bone m…
Phase 1 • Sponsor: Sumitomo Pharma America, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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Turkish myelofibrosis Patients' treatment patterns revealed in new study
Knowledge-focused CompletedThis study reviewed medical records of 355 people in Turkey with myelofibrosis (a type of bone marrow cancer) who were treated with the drug ruxolitinib for at least 3 months. Researchers collected information on patient demographics, disease characteristics, and how the drug was…
Sponsor: Novartis Pharmaceuticals • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:12 UTC
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Scientists uncover clues to inflammation in rare bone marrow disease
Knowledge-focused CompletedThis study looked at 70 people—some with primary myelofibrosis and some healthy—to understand how certain immune cells called MDSCs fuel inflammation in this rare bone marrow disease. Researchers also checked how these cells change with treatment and whether they help form new bl…
Sponsor: Fondazione IRCCS Policlinico San Matteo di Pavia • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC
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New drug interaction study for myelofibrosis treatment begins
Knowledge-focused CompletedThis phase 1 trial in 40 healthy adults tests how TQ05105, a drug for myelofibrosis, interacts with other medications that affect liver enzymes. Participants take TQ05105 alone and then with either itraconazole or rifampicin to see how these drugs change TQ05105 levels in the bod…
Phase 1 • Sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd. • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:03 UTC