[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"primary-myelofibrosis-pmf\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:primary-myelofibrosis-pmf":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,48,74,109,135,232,258],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":30,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100620446","phase-3-a-phase-3-study-of-pelabresib-dak539-and-ruxolitinib-in-myelofibrosis-mf-100620446",false,"NCT07357727","A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)","A Phase 3, Randomized, Double-blind, Active-control Study of Pelabresib (DAK539) and Ruxolitinib vs. Placebo and Ruxolitinib in Adult Patients With Myelofibrosis Who Are JAK Inhibitor Naive","MANIFEST-3","Key Inclusion Criteria:\n\n* Participants have diagnosis of primary myelofibrosis (PMF) or post-polycythemia vera myelofibrosis (post-PV MF) or post-essential thrombocythemia myelofibrosis (post-ET MF) according to the International Consensus Classification (ICC) of Myeloid Neoplasms and Acute Leukemias 2022\n* DIPSS risk category of intermediate-1, intermediate-2 or high-risk\n* Spleen volume ≥ 450 cm3 by CT or MRI scan (local read sufficient if no central read available)\n* Have an average TSS of ≥15 within 7 days prior to randomization, using MFSAF v. 4.0 (at least 4 out of 7 TSS assessments required for average calculation)\n* Participants with an Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2\n* Blasts \\\u003C5% in peripheral blood. Assessment of blasts in peripheral blood is mandatory at screening\n* Platelet count ≥ 100 x 10\\^9\u002FL in the absence of growth factors or transfusions for the previous 4 weeks\n\nKey Exclusion Criteria:\n\n* Prior splenectomy at any time or splenic irradiation in the previous 6 months\n* Prior hematopoietic cell transplant or participant anticipated to receive a hematopoietic cell transplant within 24 weeks from the date of randomization\n* Blasts ≥ 5% in bone marrow if results available at screening or history of accelerated phase (AP) or leukemic transformation\n* History of a malignancy (other than MF, PPV-MF or PET-MF) in the past 3 years in need of systemic treatment\n* Received any approved or investigational agent other than hydroxyurea or anagrelide for the treatment of MF within 14 days of first dose of study treatment or within 5 half-lives of the approved or investigational agent, whichever is longer\n* Prior treatment with any JAK inhibitor or Bromodomain and extraterminal domain (BET) inhibitor\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.","ALL","18 Years",{"count":20,"type":21},460,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","The purpose of this trial is to evaluate whether treatment with pelabresib in combination with ruxolitinib leads to improved clinical outcomes compared to ruxolitinib alone in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not previously received Janus kinase (JAK) inhibitor therapy.",[27,28,29],"Primary Myelofibrosis (PMF)","Post-polycythemia Vera Myelofibrosis (PPV-MF)","Post-essential Thrombocythemia Myelofibrosis (PET-MF)",[31,32,33,34],"Pelabresib (DAK539)","Ruxolitinib","Adult participants","Myelofibrosis (MF)","RECRUITING","2026-07-01",{"date":38,"type":39},"2026-07-02","ACTUAL",{"date":41,"type":39},"2026-05-27",{"date":43,"type":21},"2030-12-27",{"name":45,"class":46},"Novartis Pharmaceuticals","INDUSTRY",20,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":4,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":55,"targetDuration":4,"studyType":22,"phases":57,"briefSummary":59,"conditions":60,"keywords":63,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":5},"100619093","phase-1-study-of-pelabresib-add-on-to-ruxolitinib-in-japanese-adult-patients-with-myelofibrosis-100619093","NCT07340138","Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients With Myelofibrosis","A Phase 1b Study of Pelabresib (DAK539) add-on to Stable Dose of Ruxolitinib in Japanese Adult Patients With Myelofibrosis","Key Inclusion Criteria:\n\n* Participants have diagnosis of primary myelofibrosis (PMF), post-polycythemia vera MF (Post-PV MF) or post-essential thrombocythemia MF (Post-ET MF) according to the International Consensus Classification (ICC) for Myeloid Neoplasms and Acute Leukemias 2022.\n* DIPSS risk category intermediate-1, intermediate-2 or high-risk at screening.\n* Participants currently treated with ruxolitinib monotherapy AND who are likely to benefit from the addition of pelabresib to ruxolitinib in the opinion of the investigator.\n* Receiving ruxolitinib at a stable dose (5 to 25 mg BID) for at least 8 weeks prior to the first dose of pelabresib.\n* Palpable spleen (spleen length below left costal margin \\[LCM\\] must be recorded) or documented splenomegaly by MRI or CT (image report must be recorded) at screening.\n* Platelet count ≥ 100 × 10\\^9\u002FL in the absence of growth factor support (including thrombopoietin mimetics\u002Fagonists) or platelet transfusions 4 weeks prior to the first dose of pelabresib.\n* Blasts \\\u003C 5% in peripheral blood. Assessment of blasts in peripheral blood is mandatory at screening.\n\nKey Exclusion Criteria:\n\n* Prior splenectomy at any time or splenic irradiation in the previous 6 months\n* Prior hematopoietic cell transplant or participants anticipated to receive a hematopoietic cell transplant within 24 weeks from the first dose of pelabresib.\n* Blasts ≥ 5% in bone marrow if results available at screening or history of accelerated phase or leukemic transformation.\n* History of a malignancy (other than MF, PV or ET) except for adequately treated local basal cell or squamous cell carcinoma of the skin, cervical carcinoma in situ, superficial bladder cancer, asymptomatic prostate cancer without known metastatic disease and with no requirement for therapy or requiring only hormonal therapy and with normal prostate-specific antigen for ≥ 1 year prior to start of pelabresib, adequately treated Stage 1 or 2 cancer currently in complete remission, or any other cancer that has been in complete remission for ≥ 3 years\n* Received any approved or investigational agent for the treatment of MF except ruxolitinib within 14 days of first dose of pelabresib or within 5 half-lives of the approved or investigational agent, whichever is longer.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.",{"count":56,"type":21},6,[58],"PHASE1","This Phase 1b, multicenter, open-label study aims to evaluate the safety, pharmacokinetics (PK), and preliminary efficacy of pelabresib as add-on to ruxolitinib in Japanese patients with myelofibrosis (MF).",[27,61,62],"Post-polycythemia Vera Myelofibrosis (Post-PV MF)","Post-essential Thrombocythemia Myelofibrosis (Post-ET MF)",[31,64,65,34],"Ruxolitinib (INC424)","Japanese adult participants","2026-06-15",{"date":68,"type":39},"2026-06-16",{"date":70,"type":39},"2026-04-15",{"date":72,"type":21},"2030-12-18",{"name":45,"class":46},{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":4,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":22,"phases":83,"briefSummary":84,"conditions":85,"keywords":90,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":108},"100629072","phase-1-a-phase-1-study-of-prt12396-in-participants-with-select-myeloproliferative-neoplasms-100629072","NCT07469891","A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms","A Phase 1, Open-Label, Multi-Center, Safety and Efficacy Study of PRT12396 in Participants With Polycythemia Vera and Myelofibrosis","Inclusion Criteria:\n\n* Willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations (including contraception requirements), and other study procedures.\n* Confirmed diagnosis of PV or MF according to WHO 2016 or revised ICC\u002FWHO 2022 criteria\n* Documented presence of a JAK2 V617 mutation\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0-2\n* Estimate life expectancy of ≥12 weeks per investigator assessment.\n* Negative serum or urine pregnancy test and agree to use contraception or maintain true abstinence.\n* Adequate organ function and bone marrow reserves (hematology, renal, and hepatic)\n\nExclusion Criteria:\n\n* History of another malignancy within 3 years prior to enrollment, except for malignancy considered cured with low risk of recurrence.\n* Clinically significant anemia due to nutritional deficiency or hemolytic disorders.\n* Active or uncontrolled infection requiring systemic therapy or hospitalization.\n* Any other medical or psychiatric conditions that, in the Investigator's judgment, would increase risk or interfere with study participation or interpretation of results.\n* Clinically significant or uncontrolled medical conditions, including active infection or cardiovascular disease, that would increase risk or interfere with study participation.\n* Unresolved toxicity \\> Grade 1 from prior anticancer therapy, except for alopecia or peripheral neuropathy ≤ Grade 2.\n* Pregnancy or breastfeeding\n* Known sensitivity or contraindication to any component of study, or the excipients of study treatment.\n* Prior systemic therapy for PV or MF, prior or planned allogeneic hematopoietic stem-cell transplantation, recent major surgery, prior splenectomy or prior splenic irradiation, or use of hematopoietic growth factors within protocol-defined washout periods.\n* Use of strong or moderate cytochrome P450 (CYP) 3A4 inhibitor or inducer, sensitive CYP3A substrates with narrow therapeutic range, or acid-reducing agents that cannot be discontinued prior to study treatment.\n* Participation in another interventional clinical study.",{"count":82,"type":21},100,[58],"This is a first-in-human, open-label, multi-center Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in participants with high-risk polycythemia vera (PV) and myelofibrosis (MF), and to determine the maximum tolerated dose (MTD) and recommended dose(s) for expansion (RDE\\[s\\]). The study consists of a dose-escalation phase followed by a dose-expansion phase to further evaluate selected dose level(s).",[86,34,87,88,89,27],"Polycythemia Vera (PV)","Myeloproliferative Neoplasms (MPNs)","Post-Polycythemia Vera Myelofibrosis","Post-Essential Thrombocythemia Myelofibrosis",[91,92,93,94,95,96,97,98],"High-Risk Polycythemia Vera (PV)","Intermediate-1 or Intermediate-2 Risk Primary MF","High-Risk Primary MF","Post-Polycythemia Vera MF","Post-Essential Thrombocythemia MF","PRT12396","JAK Inhibitor","Myeloproliferative Neoplasms (MPN)","2026-06-05",{"date":101,"type":39},"2026-06-09",{"date":103,"type":39},"2026-04-29",{"date":105,"type":21},"2028-04",{"name":107,"class":46},"Prelude Therapeutics",5,{"id":110,"slug":111,"hasResults":11,"nctId":112,"briefTitle":113,"officialTitle":113,"acronym":4,"eligibilityCriteria":114,"healthyVolunteers":115,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":116,"targetDuration":4,"studyType":118,"phases":4,"briefSummary":119,"conditions":120,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":124,"lastUpdatePostDateStruct":125,"startDateStruct":127,"completionDateStruct":129,"leadSponsor":131,"locationsCount":134},"100640417","identification-of-mpn-specific-antigens-and-anti-mpn-tcrs-in-myeloproliferative-neoplasm-100640417","NCT07590986","Identification of MPN-specific Antigens and Anti-MPN TCRs in Myeloproliferative Neoplasm","Inclusion Criteria:\n\n* Patients previously diagnosed or suspected of having PV, ET, pre-PMF, overt PMF, or an unclassifiable MPN.\n* Patients not previously diagnosed with a haematological disease as controls\n\nExclusion Criteria:\n\n* Patients with any other hematological malignancy.",true,{"count":117,"type":21},150,"OBSERVATIONAL","The goal of this observational study is to learn how the immune system responds to myeloproliferative neoplasms (MPN) in adults with known or suspected MPN. Researchers also want to identify immune cells that could help develop future immune-based treatments for MPN.\n\nThe main questions it aims to answer are:\n\n* Are there T cells in the bone marrow that can recognize MPN cells?\n* Which targets (antigens) on MPN cells are recognized by these immune cells?\n* Can researchers identify T cell receptors (TCRs) that may be used in future TCR-based therapies?\n\nResearchers will study samples from adults with polycythemia vera (PV), essential thrombocythemia (ET), primary myelofibrosis (PMF), post-PMF, or unclassifiable MPN. Samples from healthy donors without blood disorders will also be included for comparison.\n\nBy providing blood and bone marrow samples, participants will:\n\n* Allow researchers to study immune cells and genetic material from these samples\n* Allow researchers to perform laboratory tests to study how immune cells recognize MPN cells\n\nResearchers will use laboratory methods such as genetic sequencing, cell analysis, and cell culture experiments to better understand immune responses in MPN.\n\nParticipants will not receive direct medical benefit from this study. The results may help researchers better understand MPN and support the future development of immune-based therapies.",[121,122,123,27],"Myeloproliferative Neoplasm (MPN)","Polycytemia Vera","Essential Thrombocythemia (ET)","2026-05-08",{"date":126,"type":39},"2026-05-15",{"date":128,"type":21},"2026-05",{"date":130,"type":21},"2037-01",{"name":132,"class":133},"Université Catholique de Louvain","OTHER",1,{"id":136,"slug":137,"hasResults":11,"nctId":138,"briefTitle":139,"officialTitle":140,"acronym":4,"eligibilityCriteria":141,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":142,"targetDuration":4,"studyType":118,"phases":4,"briefSummary":144,"conditions":145,"keywords":190,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":222,"lastUpdatePostDateStruct":223,"startDateStruct":225,"completionDateStruct":227,"leadSponsor":229,"locationsCount":231},"100620792","mpn-progression-registry-observational-study-tracking-symptoms-treatments-and-disease-progression-in-people-with-myeloproliferative-neoplasms-mpns-100620792","NCT07362225","MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)","The MPN PROGRESSion Registry: A Retrospective and Prospective Observational Study Collecting Patient-Reported Outcomes, Electronic Health Records, and Claims Data to Track Symptoms, Treatments, and Disease Progression in Individuals Diagnosed With Myeloproliferative Neoplasms (MPNs).","Inclusion Criteria:\n\n* Adults aged 18 years or older at the time of enrollment.\n* Confirmed diagnosis of a myeloproliferative neoplasm (MPN), including one or more of the following subtypes, according to WHO 2022 criteria, including patients originally diagnosed with one of these conditions but who have received one or more stem cell transplants (SCTs) or bone marrow transplants (BMTs):\n* Polycythemia vera (PV)\n* Essential thrombocythemia (ET)\n* Primary myelofibrosis (PMF)\n* Secondary myelofibrosis (post-ET or post-PV MF)\n* Pre-fibrotic primary myelofibrosis (pre-PMF)\n* Myeloproliferative neoplasm-unclassifiable (MPN-U)\n* Myeloproliferative neoplasm, accelerated phase (MPN-AP)\n* Myeloproliferative neoplasm, blast phase (MPN-BP)\n* Post-MPN acute myeloid leukemia (AML)\n* Myelodysplastic syndrome (MDS)\u002FMPN overlap syndrome\n* Myeloproliferative neoplasm, blast phase (MPN-BP)\n* Ability to provide informed consent electronically or through a legally authorized representative.\n* Willingness to share health information, including electronic health records (EHR), laboratory values, and survey responses.\n* Willingness to complete periodic patient-reported outcome (PRO) surveys and symptom tracking assessments.\n\nExclusion Criteria:\n\n* Individuals under 18 years of age.\n* Inability to provide informed consent, either directly or through a legally authorized representative.\n* Currently enrolled in an interventional clinical trial where participation would interfere with the ability to participate in this observational registry (based on investigator or sponsor judgment).\n* Any condition that, in the judgment of the study team, would make participation in the registry infeasible or unsafe.",{"count":143,"type":21},5000,"The MPN PROGRESSion Registry is a multi-year, observational research study designed to improve understanding of myeloproliferative neoplasms (MPNs)-a group of rare, chronic blood cancers that include polycythemia vera (PV), essential thrombocythemia (ET), primary myelofibrosis (MF), pre-fibrotic primary myelofibrosis (pre-PMF), secondary myelofibrosis, myeloproliferative neoplasm-unclassifiable (MPN-U), MPN in accelerated phase (MPN-AP), and MPN in blast phase (MPN-BP), post-MPN Acute Myeloid Leukemia (AML), and MDS\u002FMPN overlap syndrome as defined above per WHO 2022 criteria, including patients originally diagnosed with one of these conditions but who have received one or more SCTs and\u002For BMTs . These conditions are characterized by abnormal blood cell production in the bone marrow and may lead to complications such as blood clots, bleeding, bone marrow fibrosis, and, in some cases, progression to acute leukemia.\n\nThe central hypothesis of the registry is that collecting and analyzing real-world, longitudinal data-including electronic health records (EHRs), laboratory values, treatments, and patient-reported outcomes (PROs)-from a diverse population of people living with MPNs will help identify patterns and predictors of disease progression, treatment response, quality of life, and long-term outcomes. These insights are intended to guide future research, inform clinical guidelines, and support improvements in patient care.\n\nThe registry is non-interventional and observational; participants do not receive investigational treatments, and all medical care continues under the supervision of their own physicians. Data collection includes EHRs, PRO surveys, patient-reported symptom and lab tracking, insurance claims, and, in the future, may include linkages with other relevant disease registries and datasets. Potential collaborations under consideration include those with the European LeukemiaNet (ELN) MPN Registry, the Mayo Clinic MPN Database, the Center for International Blood and Marrow Transplant Research (CIBMTR), the SEER Program, Harmony Alliance Foundation, and the National Cancer Database (NCDB).\n\nThe registry emphasizes the patient voice, incorporating lived experiences related to hallmark MPN symptoms such as fatigue, pruritus (itching), bone pain, night sweats, and social and emotional impacts. Participants will be followed for at least five years, with many enrolled for ten years or longer, to capture the natural history of disease and long-term outcomes. PRO surveys will be completed approximately every six months, and EHR data will be regularly reviewed to track changes in clinical status, treatment, and disease evolution.\n\nStatistical analyses will use descriptive and inferential methods to examine clinical characteristics, symptom burden, disease trajectories, and patient-centered outcomes. Planned subgroup analyses may compare differences across diagnoses, treatment approaches, demographics, or genomic factors. Analytic plans will be finalized during the course of the study and may evolve in response to emerging scientific questions.\n\nThe registry is open to adults (18 years or older) living in the United States who have been diagnosed with any of the included MPN subtypes and are willing to share health information and complete study surveys. Individuals currently enrolled in interventional clinical trials or unable to provide informed consent may be excluded. Participation is voluntary, and participants may withdraw from the study at any time without affecting their medical care.\n\nPrivacy and data security are core priorities. Participant data will be securely stored and managed in accordance with all applicable privacy laws and research regulations. No identifiable information will be shared with external parties without appropriate authorization. Oversight is provided by a Steering Committee and a Patient Engagement Advisory Committee (PEAC), ensuring rigorous scientific, ethical, and patient-centered governance.\n\nThe registry is sponsored by the MPN Research Foundation, a nonprofit organization advancing research and patient advocacy in myeloproliferative neoplasms (MPNs). Participants can contact the registry team at any time with questions and will receive periodic updates on study findings.\n\nThis study aims to address critical gaps in understanding the real-world experiences of people with MPNs-such as symptom burden over time, risk factors for progression, and how different treatments impact patient outcomes. Findings may inform clinical trial design, support biomarker discovery, and contribute to the development of updated treatment recommendations. The registry is committed to including participants from diverse backgrounds and clinical settings to ensure findings are broadly applicable across the MPN community. Summary results will be shared through scientific publications, presentations, and other dissemination efforts to advance MPN research and care globally.",[146,147,86,123,148,27,149,34,150,151,152,153,154,155,156,157,158,159,160,161,162,88,28,61,163,164,89,29,165,62,166,167,168,169,170,87,171,172,173,174,175,176,177,178,179,180,181,182,183,184,185,186,187,188,189],"Polycythemia Vera","ET (Essential Thrombocythemia)","Primary Myelofibrosis (MF)","Myelofibrosis","Myelofibrosis, Primary","Myelofibrosis, Post ET","Myelofibrosis, Post PV","Myelofibrosis (PMF)","Myelofibrosis，MF","Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis","Myelofibrosis Due to and Following Polycythemia Vera","Myelofibrosis Transformation in Essential Thrombocythemia","Myelofibrosis With High Molecular Risk Mutations","MF","Secondary Myelofibrosis","Secondary Myelofibrosis in Myeloproliferative Disease","Secondary Myelofibrosis (Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis)","Post-polycythemia Vera Myelofibrosis(Post-PV MF)","Post-PV MF","Post-essential Thrombocythemia Myelofibrosis(Post-ET MF)","Post-ET MF","Pre-fibrotic Myelofibrosis","Myeloproliferative Disorder","Myeloproliferative Disorders","Myeloproliferative Disorders (MPD)","Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis","Myeloproliferative Neoplasm With 10% Blasts or Higher","Myeloproliferative Neoplasms","MPN","MPN (Myeloproliferative Neoplasms)","MPN-associated Myelofibrosis","Myeloproliferative Neoplasm, Unclassifiable","Myeloproliferative Neoplasm, Not Otherwise Specified","Accelerated Phase MPN","Accelerated Phase Myeloproliferative Neoplasm","Blast Phase MPN","Blast Phase Myeloproliferative Neoplasm","Thrombocythemia Myelofibrosis (PET-MF)","Thrombocythemia, Essential","Thrombocythemia, Hemorrhagic","Agnogenic Myeloid Metaplasia","Chronic Idiopathic Myelofibrosis","Idiopathic Myelofibrosis","MDS\u002FMPN Crossover Syndromes",[173,146,191,160,192,168,177,193,194,195,196,197,198,199,200,201,202,203,204,205,206,207,208,209,210,211,212,213,214,215,216,217,218,219,220,221],"Essential Thrombocythemia","Pre-fibrotic Primary Myelofibrosis","Myeloproliferative Neoplasm, Accelerated Phase","Myeloproliferative Neoplasm, Blast Phase","Chronic Myeloproliferative Disease","Hematologic Neoplasms","Bone Marrow Neoplasms","Blood Cancer","Disease Progression","Biomarker Discovery","Patient-Reported Outcomes","Electronic Health Records","Real-World Evidence","Longitudinal Study","Observational Study","Registry Study","Natural History Study","Quality of Life","Rare Diseases","Chronic Hematologic Diseases","Risk Stratification","Symptom Burden","Medical Claims Data","Longitudinal Data Collection","Patient-Centered Research","Health Outcomes Research","Clinical Outcomes","Clinical Guidelines Development","Regulatory Science","Drug Development","Treatment Response","2026-01-15",{"date":224,"type":39},"2026-01-23",{"date":226,"type":39},"2025-09-26",{"date":228,"type":21},"2035-09-08",{"name":230,"class":133},"MPN Research Foundation",2,{"id":233,"slug":234,"hasResults":11,"nctId":235,"briefTitle":236,"officialTitle":236,"acronym":4,"eligibilityCriteria":237,"healthyVolunteers":11,"sex":17,"minAge":238,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":239,"phases":4,"briefSummary":240,"conditions":241,"keywords":246,"overallStatus":253,"whyStopped":4,"lastUpdateSubmitDate":254,"lastUpdatePostDateStruct":255,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":257,"locationsCount":4},"100419739","managed-access-programs-for-inc424-ruxolitinib-100419739","NCT04745637","Managed Access Programs for INC424, Ruxolitinib","Inclusion criteria\n\n1. An independent request was received from a licensed physician.\n2. The patient has a serious or life-threatening disease or condition and there is no comparable or satisfactory alternative therapy available for diagnosis, monitoring, or treatment.\n3. The patient is not eligible or able to enroll in a clinical trial or continue participation in such trial.\n4. There is a potential patient benefit to justify the potential risk of the treatment use, and the potential risk is not unreasonable in the context of the disease or condition to be treated.\n5. The patient must meet any other medical criteria established by the medical experts responsible for the product or by the health authority in the country of request (as applicable).\n6. Provision of the product will not interfere with the initiation, conduct, or completion of a Novartis clinical trial or overall development program.\n7. Managed Access provision is allowed per local laws\u002Fregulations.","2 Years","EXPANDED_ACCESS","The purpose of this registration is to list Managed Access Programs (MAPs) related to INC424, Ruxolitinib",[27,242,183,243,86,244,245],"Post Polycythemia Myelofibrosis (PPV MF)","Severe\u002FVery Severe COVID-19 Illness","Steroid Refractory Acute Graft Versus Host Disease (SR aGVHD)","Steroid Refractory Chronic Graft Versus Host Disease (SR cGVHD)",[247,248,27,242,183,249,86,250,251,252,32],"MAP","Manage Access Program","Severe\u002Fvery severe COVID-19 illness","Steroid refractory acute Graft versus Host Disease (SR aGVHD)","Steroid refractory chronic Graft versus Host Disease (SR cGVHD)","INC424","AVAILABLE","2025-11-14",{"date":256,"type":39},"2025-11-18",{"name":45,"class":46},{"id":259,"slug":260,"hasResults":11,"nctId":261,"briefTitle":262,"officialTitle":263,"acronym":4,"eligibilityCriteria":264,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":265,"targetDuration":4,"studyType":22,"phases":266,"briefSummary":268,"conditions":269,"keywords":271,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":274,"lastUpdatePostDateStruct":275,"startDateStruct":277,"completionDateStruct":279,"leadSponsor":281,"locationsCount":134},"100575327","phase-2-ropeginterferon-alfa-2b-plus-ruxolitinib-for-myelofibrosis-100575327","NCT06770842","Ropeginterferon Alfa 2b Plus Ruxolitinib for Myelofibrosis","Safety and Efficacy of Ropeginterferon Alfa-2b in Combination With Ruxolitinib in Patients With Myelofibrosis Demonstrating Suboptimal Response to Ruxolitinib Monotherapy","Inclusion Criteria:\n\n* Willing and able to provide informed consent\n* Age ≥18 years\n* Diagnosis of Overt Myelofibrosis (primary, post-ET, or post-PV) per World Health Organization (WHO) 2022 diagnostic criteria\n* Intermediate-1, Intermediate-2, or high-risk disease by Dynamic International Prognostic Scoring System (DIPSS)\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0-2\n* Platelet count ≥75 x 109\u002FL prior to dosing on Cycle 1 Day 1\n* Absolute neutrophil count ≥0.5 x 109\u002FL prior to dosing on Cycle 1 Day 1\n* Peripheral blast count ≤10% prior to dosing on Cycle 1 Day 1\n* Women of childbearing potential and fertile men must agree to use an approved method of contraception from screening until 30 days after the last dose of ropeginterferon and ruxolitinib.\n* Patients with suboptimal response to ruxolitinib as per one of the below:\n\n  i. Relapsed: Ruxolitinib treatment for ≥3 months with spleen regrowth, defined as \\\u003C10% SVR or \\\u003C30% decrease in spleen size from baseline, following an initial response\\* ii. Refractory: Ruxolitinib treatment for ≥3 months with \\\u003C10% SVR or \\\u003C30% decrease in spleen size from baseline.\n\n  \\* Response to ruxolitinib is defined as a ≥35% reduction in spleen volume from baseline, or a ≥50% reduction in spleen size for baseline spleen sizes \\>10 cm below left costal margin (LCM); a non-palpable spleen for baseline spleen sizes between 5-10 cm below LCM; or not eligible for spleen response for baseline spleen \\\u003C5 cm below LCM.\n\nExclusion Criteria:\n\nSubjects will not be eligible for participation if they meet any of the following exclusion criteria:\n\n* Prior or current use of interferon alfa (IFNα) preparations for MPN\n* Patients currently on other investigational therapy (ies)\n* Contraindications or hypersensitivity to IFNα preparations\n* History of organ and haematopoietic stem cell transplantation\n* History of splenectomy\n* Pregnant or lactating females, or females planning to become pregnant at any time during the study\n* Documented autoimmune disease at screening\n* Infection with human immunodeficiency virus (HIV)\n* Active and uncontrolled infections with hepatitis B virus (HBV) and hepatitis C virus (HCV). Please note that patients on antiviral therapy with undetectable HBV DNA and HCV RNA may be recruited.\n* Evidence of severe retinopathy including but not limited to macular degeneration, diabetic retinopathy and hypertensive retinopathy.\n* History of clinically significant neuropsychiatric conditions including but not limited to depression and epilepsy.\n* Clinically significant neuropsychiatric conditions including but not limited to depression and epilepsy.\n* Concurrent second active and non-stable malignancy (patients with a concurrent second active but stable malignancy, i.e., non-melanoma skin cancers, are eligible)\n* Evidence of alcohol or drug abuse within 6 months\n* Evidence at the time of Screening of significant renal or hepatic insufficiency (unless due to hemolysis) as defined by any of the following local lab parameters:\n\n  * Calculated glomerular filtration rate (GFR; using the Cockcroft-Gault equation) \\\u003C40 mL\u002Fmin or serum creatinine \\>1.5 x the local upper limit of normal\n  * Aspartate transaminase (AST) or alanine aminotransferase (ALT) ≥2.5 x the local upper limit of normal\n* Unwilling or unable to comply with the study protocol",{"count":47,"type":21},[267],"PHASE2","In this open-label single arm phase 2 study, approximately 20 patients with MF demonstrating suboptimal response to ruxolitinib monotherapy will be enrolled. Patients will continue to receive ruxolitinib at a stable dose and ropeginterferon alfa 2b will be added to the regimen.",[27,242,270],"Post Essential Thrombocythaemia Myelofibrosis (PET-MF)",[32,272,149,273],"Ropeginterferon alfa 2b","Ruxolitinib failiure","2025-06-09",{"date":276,"type":39},"2025-06-11",{"date":278,"type":39},"2025-03-01",{"date":280,"type":21},"2027-12",{"name":282,"class":133},"The University of Hong Kong"]