Clinical Research Directory

A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)

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.

Maintenance Treatment With Imetelstat in Patients With High-Risk Myeloid Neoplasms After Allo-SCT

The purpose of this clinical trial is to learn if imetelstat is safe to use in participants with high-risk myeloid malignancies who have received an allogenic hematologic stem cell transplantation (allo-SCT). Participants will receive imetelstat by intravenous infusion once every 28 days for up to one year.

Peri-Transplant Gecacitinib in Patients With Myelofibrosis Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

This study aims to evaluate the efficacy, safety, and feasibility of peri-transplant gecacitinib in patients with myelofibrosis undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Myelofibrosis is a bone marrow disorder that can cause reduced blood cell production, enlargement of the spleen, and other complications. Allogeneic stem cell transplantation offers a potential cure, but its benefits may be limited by graft-versus-host disease (GVHD), disease relapse, and transplant-related complications. This is a multicenter, prospective, open-label, single-arm clinical study. Participants will receive gecacitinib before, during, and after transplantation, together with standard transplantation care and GVHD prophylaxis. The primary objective is to evaluate GVHD-free, relapse-free survival (GRFS) at 1 year after transplantation. GRFS measures survival without severe acute GVHD, chronic GVHD requiring systemic immunosuppressive treatment, disease relapse or progression, or death. The study plans to enroll approximately 46 participants, with 43 expected to be evaluable for the primary endpoint. Participants will be followed for up to 2 years after transplantation. Secondary assessments include overall survival, progression-free survival, GVHD, disease relapse, non-relapse mortality, hematopoietic engraftment, treatment feasibility, and safety.

A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms

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).

Study of Pelabresib add-on to Ruxolitinib in Japanese Adult Patients With Myelofibrosis

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).

Study to Evaluate Safety, Pharmacokinetic and Pharmacodynamic Dose Escalation and Expansion Study of PXS-5505 in Patients With Primary, Post-polycythemia Vera or Post-essential Thrombocythemia Myelofibrosis

This study will be an open-label phase 1/2a study to evaluate the safety and tolerability of PXS-5505 in patients with primary, postpolycythemia vera (PV) or post-essential thrombocythemia (ET) myelofibrosis.

Identification of MPN-specific Antigens and Anti-MPN TCRs in Myeloproliferative Neoplasm

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. The main questions it aims to answer are: * Are there T cells in the bone marrow that can recognize MPN cells? * Which targets (antigens) on MPN cells are recognized by these immune cells? * Can researchers identify T cell receptors (TCRs) that may be used in future TCR-based therapies? Researchers 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. By providing blood and bone marrow samples, participants will: * Allow researchers to study immune cells and genetic material from these samples * Allow researchers to perform laboratory tests to study how immune cells recognize MPN cells Researchers will use laboratory methods such as genetic sequencing, cell analysis, and cell culture experiments to better understand immune responses in MPN. Participants 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.

MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)

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/MPN 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/or 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. The 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. The 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). The 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. Statistical 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. The 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. Privacy 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. The 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. This 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.

Ropeginterferon Alfa 2b Plus Ruxolitinib for Myelofibrosis

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.