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Home › Conditions › Myelofibrosis

Myelofibrosis

7 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Myelofibrosis

73 trials tracked for Myelofibrosis: 8 in phase 3 or 4 and 4 with published results. The most-cited published study has 159 citations.

73Trials tracked
8Phase 3 & 4
0Recruiting
4With published results
Phase distribution
Phase 3 8 Phase 2 55 Phase 1 3 Other / NA 7
  1. Phase 3 INC424 for Patients With Primary Myelofibrosis, Post Polycythemia Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis. Completed · 159 cited
  2. Phase 3 Momelotinib Versus Ruxolitinib in Subjects With Myelofibrosis Completed · 49 cited
  3. Phase 3 A Study of Momelotinib Versus Danazol in Symptomatic and Anemic Myelofibrosis Participants (MOMENTUM) Completed · 41 cited
  4. Phase 3 An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib Completed · 33 cited
  5. Phase 3 Eltrombopag Olamine in Treating Thrombocytopenia in Patients With Chronic Myeloid Leukemia or Myelofibrosis Receiving Tyrosine Kinase Therapy Completed
  6. Phase 3 Controlled Myelofibrosis Study With Oral Janus-associated Kinase (JAK) Inhibitor Treatment-II: The COMFORT-II Trial Completed
Show 44 more trials
  1. Phase 3 Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload Completed
  2. Phase 3 Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence Completed
  3. Phase 2 A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies Completed
  4. Phase 2 Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome Completed
  5. Phase 2 CC-4047 in Treating Patients With Myelofibrosis Completed
  6. Phase 2 Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients Undergoing a Donor Stem Cell Transplant For Hematological Cancer Completed
  7. Phase 2 Tacrolimus and Mycophenolate Mofetil in Preventing Graft-Versus-Host Disease in Patients Who Have Undergone Total-Body Irradiation With or Without Fludarabine Phosphate Followed by Donor Peripheral Blood Stem Cell Transplant for Hematologic Cancer Completed
  8. Phase 2 Ruxolitinib Phosphate and Danazol in Treating Anemia in Patients With Myelofibrosis Completed
  9. Phase 2 Alternative Dosing Strategy of Ruxolitinib in Patients With Myelofibrosis Completed
  10. Phase 2 Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies Completed
  11. Phase 2 Myeloablative Umbilical Cord Blood Transplantation in Hematological Diseases Completed
  12. Phase 2 Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis Completed
  13. Phase 2 Study of Ruxolitinib (INCB018424) Sustained Release Formulation in Myelofibrosis Patients Completed
  14. Phase 2 Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis Completed
  15. Phase 2 An Extension Study of Bomedemstat (IMG-7289/MK-3543) in Participants With Myeloproliferative Neoplasms (IMG-7289-CTP-202/MK-3543-005) Completed
  16. Phase 2 A Phase Ib/II Dose-finding Study to Assess the Safety and Efficacy of LDE225 + INC424 in Patients With MF Completed
  17. Phase 2 Lenalidomide for Patients With Myelofibrosis (MF) Completed
  18. Phase 2 Reduced Intensity Conditioning Transplant Using Haploidentical Donors Completed
  19. Phase 2 CEP-701 for PH-negative Myelofibrosis Completed
  20. Phase 2 Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis Completed
  21. Phase 2 Long-term Safety and Efficacy of Momelotinib in Subjects With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Polycythemia Vera or Essential Thrombocythemia Completed
  22. Phase 2 Open Label Ruxolitinib (INCB018424) in Patients With Myelofibrosis and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis Completed
  23. Phase 2 Ondansetron in Preventing Nausea and Vomiting in Patients Undergoing Stem Cell Transplant Completed
  24. Phase 2 Vaccine Therapy in Reducing the Frequency of Cytomegalovirus Events in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant Completed
  25. Phase 2 Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis Completed
  26. Phase 2 Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloablative Allotransplantation for the Treatment of Blood Cancer Completed
  27. Phase 2 A Safety and Efficacy Study to Evaluate Luspatercept in Subjects With Myeloproliferative Neoplasm-associated Myelofibrosis Who Have Anemia With and Without Red Blood Cell-transfusion Dependence Completed
  28. Phase 2 Study to Evaluate Activity of 2 Dose Levels of Imetelstat in Participants With Intermediate-2 or High-Risk Myelofibrosis (MF) Previously Treated With Janus Kinase (JAK) Inhibitor Completed
  29. Phase 2 PD-1 Inhibition in Advanced Myeloproliferative Neoplasms Completed
  30. Phase 2 Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies Completed
  31. Phase 2 Efficacy and Safety of Ruxolitinib in the Treatment of Anemic Myelofibrosis Patients. Completed
  32. Phase 2 3-AP and Fludarabine in Treating Patients With Myeloproliferative Disorders, Chronic Myelomonocytic Leukemia, or Accelerated Phase or Blastic Phase Chronic Myelogenous Leukemia Completed
  33. Phase 2 Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies Completed
  34. Phase 2 Safety and Tolerability Study of Oral NS-018 in Patients With Primary Myelofibrosis (MF), Post-polycythemia Vera MF or Post-essential Thrombocythemia MF Completed
  35. Phase 2 Dasatinib as Therapy for Myeloproliferative Disorders (MPDs) Completed
  36. Phase 2 Reduced-Intensity Conditioning Before Donor Stem Cell Transplant in With High-Risk Hematologic Malignancies Completed
  37. Phase 2 Efficacy and Safety of Simtuzumab in Adults With Primary, Post Polycythemia Vera or Post Essential Thrombocythemia Myelofibrosis Completed
  38. Phase 2 Lenalidomide and Prednisone in Treating Patients With Myelofibrosis Completed
  39. Phase 2 Phase 2 Study: An Open-Label, Randomized, Phase 2 Dose-Finding Study of Pacritinib in Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post- Essential Thrombocythemia Myelofibrosis Previously Treated With Ruxolitinib Completed
  40. Phase 2 JAK Inhibitor Before Donor Stem Cell Transplant in Treating Patients With Primary or Secondary Myelofibrosis Completed
  41. Phase 2 A Phase II Study of CC-5013 in Myelofibrosis Completed
  42. Phase 2 Itacitinib, Tacrolimus, and Sirolimus for the Prevention of GVHD in Patients With Acute Leukemia, Myelodysplastic Syndrome, or Myelofibrosis Undergoing Reduced Intensity Conditioning Donor Stem Cell Transplantation Completed
  43. Phase 2 A Phase 2 Study of RO7490677 In Participants With Myelofibrosis Completed
  44. Phase 2 To Assess the Safety, Tolerability and Efficacy of Itacitinib Immediate Release Tablets in Participants With Primary or Secondary Myelofibrosis Who Have Received Prior Ruxolitinib and/or Fedratinib Monotherapy (LIMBER-213) Completed

Showing the 50 most-cited and recently-updated of 73 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found Updated — new results For clinicians

Myelofibrosis: what the trials found

Fludarabine is established in the treatment of myelofibrosis [1, 19]. INC424 demonstrated a 71.7% rate of participants achieving at least a 50% reduction in spleen length over 5 years 2. Ruxolitinib showed significant efficacy in reducing spleen volume, with 31.9% of patients achieving a $\geq$35% reduction by week 24 and 28.5% by week 48 8; it was also evaluated in smaller cohorts 12.

Momelotinib demonstrated superior outcomes compared to placebo across multiple metrics: a significantly higher splenic response rate (SRR) of $\geq$25% at week 24 (39.23% vs. 6.15%, p<0.0001) 4, a significant reduction in the number of red blood cell (RBC) transfusions per month (p<0.001) 3, and a significantly higher rate of transfusion independence at week 24 (30.0% vs. 20.00%, p=0.0116) 4. Additionally, Momelotinib showed a significant improvement in total symptom score (TSS) response rates compared to placebo at week 24 (24.62% vs. 9.23%, p=0.0095) 4, though it did not show a statistically significant difference in TSS response rate compared to another comparator group (p=0.98) 3.

Fedratinib showed superior results over placebo in spleen volume reduction (RR $\geq$25% at 47.0% vs. 13.4%, p<0.0001) and symptom response rates (34.1% vs. 16.9%, p=0.0033) 5. Bomedemstat was evaluated for its impact on spleen volume and size, showing varied results across different study arms 17. Eltrombopag Olamine is also established in the treatment landscape 6.

Pomalidomide 0.5 mg showed no significant difference in the percentage of participants achieving RBC-transfusion independence compared to the control group (p=1.000) 7. Deferasirox is also an established intervention for this condition 9.

Recent results — preliminary, needs further review

  • Fludarabine phosphate was evaluated in a Phase 2 trial 10.

For the clinician treating this condition

  • Momelotinib demonstrates significant superiority over placebo in splenic response, transfusion independence, and symptom reduction at week 24 [3, 4].
  • Fedratinib is associated with significantly higher spleen volume response rates and symptom response rates compared to placebo 5.
  • Ruxolitinib effectively reduces spleen volume by $\geq$35% in a significant portion of the patient population 8.

AI synthesis of 11 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Biallelic SLCO2A1 variants may be associated with myelofibrosis in patients with primary hypertrophic osteoarthropathy
Pulmonology & Critical Care Sys. Review
Biallelic SLCO2A1 variants may be associated with myelofibrosis in patients with primary hypertrophic osteoarthropathy Rare gene variants linked to bone disease and blood cancer
This case report and review of literature describes a patient with primary hypertrophic osteoarthropathy (PHO) and concurrent myelofibrosis.…
A man with a rare bone disorder also had a blood cancer, and genetic testing revealed the link.
Frontiers Jul 4, 2026
Narrative review proposes phenotype-driven framework for managing myelofibrosis splenomegaly
Cardiology Sys. Review
Narrative review proposes phenotype-driven framework for managing myelofibrosis splenomegaly New combo strategies shrink spleens better than older treatments for myelofibrosis
This narrative review addresses the management of splenomegaly in patients with myelofibrosis. The authors synthesize evidence regarding eme…
Emerging combination strategies like pelabresib plus ruxolitinib or navitoclax plus ruxolitinib show superior spleen shrinkage for myelofibr…
Frontiers May 26, 2026
Case report and review: azacytidine plus ruxolitinib in connective tissue disease with CMML and myelofibrosis
Allergy & Immunology Sys. Review
Case report and review: azacytidine plus ruxolitinib in connective tissue disease with CMML and myelofibrosis A single patient found relief from blood cancer with a new drug combo
This case report and literature review describes a 52-year-old male with connective tissue disease, chronic myelomonocytic leukemia, and mye…
One man with blood cancer saw his fever and anemia improve after two years on a specific drug mix, offering a glimpse into potential new tre…
Frontiers May 14, 2026
VEXAS Syndrome Clinical Features and Treatment Outcomes in a Chinese Cohort of 16 Patients
Allergy & Immunology Cohort
VEXAS Syndrome Clinical Features and Treatment Outcomes in a Chinese Cohort of 16 Patients Small study links UBA1 gene variants to blood and inflammatory issues in China
This retrospective cohort study of 16 Chinese patients describes main clinical features and treatment responses in VEXAS syndrome.
A specific genetic change called a UBA1 variant was found in 81 percent of patients in China with blood disorders and inflammation affecting…
Frontiers Apr 16, 2026
Itacitinib plus standard therapy in acute leukemia or myelofibrosis patients undergoing reduced-intensity conditioning donor stem cell transplantation
Oncology Phase II
Itacitinib plus standard therapy in acute leukemia or myelofibrosis patients undergoing reduced-intensity conditioning donor stem cell transplantation Early trial explores itacitinib for stem cell transplant patients with blood disorders
This Phase IIa trial evaluated itacitinib combined with tacrolimus and sirolimus in patients with acute leukemia, myelodysplastic syndrome, …
A new trial adds itacitinib to standard care for stem cell transplant patients with blood disorders to help prevent graft-versus-host diseas…
CT.gov Apr 13, 2026
Case report: Low-intensity chemotherapy achieves remission in PMF transformed to MPAL
Oncology Cohort
Case report: Low-intensity chemotherapy achieves remission in PMF transformed to MPAL Single case report describes treatment for rare leukemia transformation in elderly patient
A case report describes a 75-year-old female with JAK2V617F-positive primary myelofibrosis that transformed to mixed phenotype acute leukemi…
A 75-year-old woman with rare leukemia transformation from blood disorder achieved remission after two cycles of low-intensity chemotherapy.
Frontiers Apr 3, 2026
Phase 3 trial of navitoclax plus ruxolitinib versus ruxolitinib alone in myelofibrosis posts results
Oncology Phase III
Phase 3 trial of navitoclax plus ruxolitinib versus ruxolitinib alone in myelofibrosis posts results Phase 3 trial tests navitoclax with ruxolitinib for myelofibrosis spleen volume
A phase 3 randomized controlled trial in 252 adults with myelofibrosis compared navitoclax plus ruxolitinib to ruxolitinib plus placebo.
A new drug combination aims to shrink painful enlarged spleens in myelofibrosis patients, but results from this large global trial are not y…
CT.gov Apr 2, 2026