Disease · MONDO_0044903
myelofibrosis
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. myelofibrosis. Diseases, record MONDO_0044903. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0044903
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Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
Drugs125
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| RUXOLITINIB | CHEMBL1789941 | Small molecule | Approval | TTD | 77 | 76 | 76 | Drugs and targets |
| PACRITINIB | CHEMBL2035187 | Small molecule | Approval | TTD | 12 | 11 | 11 | Drugs and targets |
| MOMELOTINIB | CHEMBL1078178 | Small molecule | Approval | TTD | 10 | 9 | 9 | Drugs and targets |
| FEDRATINIB | CHEMBL1287853 | Small molecule | Approval | TTD | 9 | 8 | 8 | Drugs and targets |
| RUXOLITINIB PHOSPHATE | CHEMBL1795071 | Small molecule | Approval | DailyMed and PMDA | 6 | 4 | 4 | Drugs and targets |
| FEDRATINIB HYDROCHLORIDE | CHEMBL4297216 | Small molecule | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| MOMELOTINIB DIHYDROCHLORIDE MONOHYDRATE | CHEMBL6068336 | Small molecule | Approval | PMDA | 1 | 0 | 0 | Drugs and targets |
| NAVITOCLAX | CHEMBL443684 | Small molecule | Phase 3 | ClinicalTrials.gov and TTD | 7 | 6 | 6 | Drugs and targets |
| POMALIDOMIDE | CHEMBL43452 | Small molecule | Phase 3 | ClinicalTrials.gov | 6 | 6 | 6 | Drugs and targets |
| SELINEXOR | CHEMBL3545185 | Small molecule | Phase 3 | ClinicalTrials.gov | 5 | 5 | 5 | Drugs and targets |
1 to 10 of 125
See all 125 drugsTargets164
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| JAK2 | Janus Kinase (JAK) | 13 | Janus Kinase (JAK) inhibitor; Tyrosine-protein kinase JAK2 inhibitor | Approval | https://conductscience.org/library/genes/3717 | Drugs and targets |
| JAK1 | Janus Kinase (JAK) | 6 | Janus Kinase (JAK) inhibitor; Tyrosine-protein kinase JAK1 inhibitor | Approval | https://conductscience.org/library/genes/3716 | Drugs and targets |
| FKBP1A | Peptidyl-prolyl cis-trans isomerase FKBP1A | 4 | FK506-binding protein 1A inhibitor | Phase 2 | https://conductscience.org/library/genes/2280 | Drugs and targets |
| FLT3 | Receptor-type tyrosine-protein kinase FLT3 | 4 | Tyrosine-protein kinase receptor FLT3 inhibitor | Approval | https://conductscience.org/library/genes/2322 | Drugs and targets |
| SMO | Protein smoothened | 4 | Smoothened homolog antagonist; Smoothened homolog inhibitor | Phase 2 | https://conductscience.org/library/genes/6608 | Drugs and targets |
| ACVR1 | Activin receptor type-1 | 3 | Activin receptor type-1 inhibitor | Approval | https://conductscience.org/library/genes/90 | Drugs and targets |
| BCL2 | Apoptosis regulator Bcl-2 | 3 | Apoptosis regulator Bcl-2 inhibitor | Phase 3 | https://conductscience.org/library/genes/596 | Drugs and targets |
| CRBN | CRL4(CRBN) E3 ubiquitin ligase | 3 | CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator | Phase 3 | https://conductscience.org/library/genes/51185 | Drugs and targets |
| CUL4A | CRL4(CRBN) E3 ubiquitin ligase | 3 | CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator | Phase 3 | https://conductscience.org/library/genes/8451 | Drugs and targets |
| DDB1 | CRL4(CRBN) E3 ubiquitin ligase | 3 | CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator | Phase 3 | https://conductscience.org/library/genes/1642 | Drugs and targets |
1 to 10 of 164
See all 164 targetsTrials210
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 4 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01493414 | INC424 for Patients With Primary Myelofibrosis, Post Polycythemia Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis. | Phase 3 | 2,233 | 2019-04-26 | Myelofibrosis | RUXOLITINIB | Its record names the disease | ClinicalTrials.gov |
| NCT01969838 | Momelotinib Versus Ruxolitinib in Subjects With Myelofibrosis | Phase 3 | 432 | 2023-05-12 | Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis | MOMELOTINIB, RUXOLITINIB | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT04603495 | Phase 3 Study of Pelabresib (CPI-0610) in Myelofibrosis (MF) (MANIFEST-2) | Phase 3 | 430 | 2024-10-28 | Myelofibrosis, Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis | PELABRESIB, RUXOLITINIB | Its record names the disease | ClinicalTrials.gov |
| NCT01773187 | Pacritinib Versus Best Available Therapy to Treat Myelofibrosis | Phase 3 | 327 | 2020-09-29 | Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis | PACRITINIB | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT02055781 | Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia | Phase 3 | 311 | 2021-11-18 | Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis | PACRITINIB | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT01178281 | Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence | Phase 3 | 267 | 2014-03-14 | Primary Myelofibrosis, MPN-associated Myelofibrosis | POMALIDOMIDE | Its record names the disease | ClinicalTrials.gov |
| NCT04551066 | To Evaluate the Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis (LIMBER-313) | Phase 3 | 252 | 2024-10-04 | Myelofibrosis, Primary Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis, Post Polycythemia Vera Myelofibrosis | PARSACLISIB, RUXOLITINIB | Its record names the disease | ClinicalTrials.gov |
| NCT04173494 | A Study of Momelotinib Versus Danazol in Symptomatic and Anemic Myelofibrosis Participants (MOMENTUM) | Phase 3 | 195 | 2022-12-22 | Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis | DANAZOL, MOMELOTINIB | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT03222609 | A Study Evaluating Tolerability and Efficacy of Navitoclax Alone or in Combination With Ruxolitinib in Participants With Myelofibrosis | Phase 2 | 191 | 2025-03-12 | Myelofibrosis (MF) | NAVITOCLAX, RUXOLITINIB | Its record names the disease | ClinicalTrials.gov |
| NCT04551053 | To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib (LIMBER-304) | Phase 3 | 177 | 2024-08-23 | Myelofibrosis, Primary Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis, Post Polycythemia Vera Myelofibrosis | PARSACLISIB, RUXOLITINIB | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 210
See all 210 trialsComparisons in its trials38
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Pelabresib + Ruxolitinib (Experimental Arm) vs Placebo + Ruxolitinib (Control Arm): Key Secondary: Number of Participants With TSS50 Response at Week 24 | 112 of 214 | 100 of 216 | 430 | NCT04603495 | Trial comparisons |
| Pelabresib + Ruxolitinib (Experimental Arm) vs Placebo + Ruxolitinib (Control Arm): Number of Participants With Splenic Response by Central Radiology Reads at Week 24 | 141 of 214 | 76 of 216 | 430 | NCT04603495 | Trial comparisons |
| Pelabresib + Ruxolitinib (Experimental Arm) vs Placebo + Ruxolitinib (Control Arm): Number of Participants With Modified Total Symptom Score (mTSS) Response at Week 24 | 120 of 214 | 104 of 216 | 430 | NCT04603495 | Trial comparisons |
| Pelabresib + Ruxolitinib (Experimental Arm) vs Placebo + Ruxolitinib (Control Arm): Number of Participants Who Transitioned From Red Blood Cell (RBC) Transfusion Dependence to Transfusion Independence | 0 of 214 | 0 of 216 | 430 | NCT04603495 | Trial comparisons |
| Pelabresib + Ruxolitinib (Experimental Arm) vs Placebo + Ruxolitinib (Control Arm): deaths from any cause | 11 of 212 | 10 of 214 | 426 | NCT04603495 | Trial comparisons |
| Pelabresib + Ruxolitinib (Experimental Arm) vs Placebo + Ruxolitinib (Control Arm): serious adverse events | 63 of 212 | 63 of 214 | 426 | NCT04603495 | Trial comparisons |
| Pelabresib + Ruxolitinib (Experimental Arm) vs Placebo + Ruxolitinib (Control Arm): Number of Participants With Improvement From Baseline in Bone Marrow Fibrosis of at Least 1 Grade at Week 24 | 36 of 192 | 21 of 188 | 380 | NCT04603495 | Trial comparisons |
| Pacritinib vs Best Available Therapy: Spleen Volume Reduction | 42 of 220 | 5 of 107 | 327 | NCT01773187 | Trial comparisons |
| Pacritinib vs Best Available Therapy: deaths from any cause | 27 of 220 | 3 of 106 | 326 | NCT01773187 | Trial comparisons |
| Pacritinib vs Best Available Therapy: serious adverse events | 117 of 220 | 33 of 106 | 326 | NCT01773187 | Trial comparisons |
1 to 10 of 38
See all 38 comparisons in its trialsPapers from its trials106
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0044903
GET https://conductscience.org/api/diseases/v1/MONDO_0044903/drugs?page=2