Primary Myelofibrosis

Researchers are studying myelofibrosis (MF), a disease involving abnormal bone marrow fibrosis, stem cell growth, and inflammation, which is difficult to monitor due to invasive biopsy methods. This observational study aims to assess the usefulness and accuracy of two imaging techniques, 18F-FDG PET/CT and 18F-FAPI PET/MRI, in evaluating systemic fibrosis in MF patients. The study will also explore how these imaging results relate to patients' clinical outcomes. Participants diagnosed with primary or secondary myelofibrosis will undergo both 18F-FDG PET/CT and 18F-FAPI PET/MRI scans. The study design is prospective, observational, self-controlled, and conducted across multiple centers. Imaging, hematologic, cytogenetic, and pathological assessments will be performed within two weeks of each other. Patients must be stable and able to cooperate with the imaging procedures. During the study, participants will be monitored over an average of two years to evaluate the diagnostic performance of 18F-FAPI PET/MRI and compare clinical prognoses based on imaging and pathology results. The study will include assessments such as imaging scans and clinical follow-up. Safety and adherence to protocol will be tracked, with informed consent required. The goal is to improve noninvasive monitoring of myelofibrosis progression and treatment effects.

Researchers are studying the use of 3'-deoxy-3'-[18F] fluorothymidine (FLT) positron emission tomography (PET) imaging in patients with cancer. This phase I trial aims to evaluate how well FLT PET imaging measures tumor growth and the activity of the DNA synthetic pathway in various cancers, including solid tumors and blood cancers. The study also seeks to determine how effective this imaging method is at detecting lesions and assessing response to treatment. Participants receive up to four FLT PET imaging procedures. During each procedure, a small amount of the FLT tracer compound is injected into the vein, followed by PET scan data collection for two hours to measure tumor growth. Blood samples may be taken during the scans, and urine samples collected afterward to analyze breakdown products of the tracer. Throughout the study, patients undergo assessments including PET or CT PET scans to measure tracer uptake and retention in tumors and normal organs. Researchers also evaluate changes in key enzymes related to DNA synthesis before and after therapy. These evaluations help monitor tumor activity and treatment response. The total time participants spend in the scanner during imaging is up to two hours per session, with a focus on capturing detailed tumor growth information.

Researchers are evaluating the diagnostic effectiveness of 68Ga FAPI PET/CT imaging for patients with myelofibrosis, a condition affecting the bone marrow. The study aims to compare this imaging technique with conventional CT scans and to identify different fibrosis grades using bone marrow biopsy as the reference standard. This observational study seeks to determine how well 68Ga FAPI PET/CT detects myelofibrosis by measuring sensitivity, specificity, and prediction rates. Participants include those with suspected or confirmed myelofibrosis and patients with primary or secondary myelofibrosis who have not been treated with ruxolitinib. The main intervention is the 68Ga FAPI PET/CT scan, which involves lying on a scanning bed for about 20 minutes. The study does not involve treatment but focuses on diagnostic imaging to assess the disease. During the study, participants will undergo the 68Ga FAPI PET/CT scan and bone marrow biopsy. Researchers will evaluate diagnostic accuracy by tracking sensitivity, specificity, positive prediction rate, and negative prediction rate over up to 24 months. Participants will be monitored for their ability to complete the imaging and adherence to study protocols, with safety considerations including excluding those with allergies or intolerance to the imaging agent and those with certain health conditions.

Researchers are evaluating the safety and effectiveness of TQ05105 Tablets combined with TQB3617 Capsules in people with intermediate- and high-risk Myelofibrosis, a type of bone marrow cancer. This open, single-arm, multi-center clinical trial is sponsored by Chia Tai Tianqing Pharmaceutical Group Co., Ltd. and aims to find the best dose and measure improvements in spleen size and symptoms over time. Participants will take TQ05105 Tablets, which target Janus kinase 1 and 2 enzymes, together with TQB3617 Capsules, which inhibit bromodomain and extra-terminal proteins. Both medications are taken orally in 21-day treatment cycles. The study includes an initial phase to determine the maximum tolerated dose and recommended dose, followed by longer evaluation periods lasting up to 120 weeks. During the study, participants will undergo regular assessments including measurement of spleen volume, symptom questionnaires, gene mutation analysis, and monitoring of side effects. The main outcomes focus on spleen volume reduction and symptom improvement, as well as survival and safety over the study period. Total participation may last up to two years, with ongoing monitoring to evaluate treatment response and tolerability.

Researchers are evaluating the safety, tolerability, and effectiveness of TQB3909 tablets combined with azacitidine in adults with myeloid malignancies, including acute myeloid leukemia and myelodysplastic syndromes. This open, multi-center clinical trial is designed as a Phase Ib/II study to better understand how this combination treatment works in these blood cancers. Participants receive TQB3909 tablets once daily in 28-day treatment cycles along with azacitidine. The study focuses on monitoring how well patients tolerate the treatment and its effects on their disease. The trial includes assessment of various response rates and survival outcomes over several weeks. Throughout the trial, participants undergo regular evaluations including monitoring for adverse events and laboratory tests for up to 24 weeks. Researchers measure remission rates, duration of remission, and survival outcomes up to 60 weeks. Participants' safety and response to treatment are closely tracked during the study.

Researchers are evaluating AJ1-11095, an oral type II JAK2 inhibitor, in adults with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not responded to or relapsed after prior treatment with at least one type I JAK2 inhibitor. This phase 1, non-randomized, open-label trial aims to assess the safety, tolerability, pharmacokinetics, clinical activity, and biomarker changes of AJ1-11095 in this patient group. The study includes a dose escalation phase followed by an expansion phase to identify the best dose for future studies.

Researchers are studying the safety, tolerability, how the body processes it, and early signs of effectiveness of an investigational drug called PRT12396 in people with high-risk polycythemia vera (PV) and myelofibrosis (MF). These conditions are types of myeloproliferative neoplasms that affect blood cell production. The study aims to find the highest dose that can be tolerated and to recommend doses for further testing. It is a first-in-human, open-label Phase 1 trial conducted at multiple centers. The trial has two parts: first, a dose-escalation phase where participants receive increasing oral doses of PRT12396 twice daily to assess safety and determine the recommended dose. Second, a dose-expansion phase enrolls more participants to further evaluate the selected dose's safety, tolerability, and preliminary effects. PRT12396 capsules are taken by mouth with water, either an hour before or two hours after meals. Participants will attend scheduled visits and undergo laboratory tests to monitor their health and response to treatment. The research team will assess dose-limiting toxicities, adverse events, and various blood-related measures such as hematologic response and symptom scores over about two years. The study plans to enroll up to 100 participants and includes careful monitoring of safety and drug levels in the body throughout the trial period.

Researchers are evaluating cytokine induced memory-like natural killer (CIML NK) cells combined with IL-2 in adults aged 18 and older with Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and Myeloproliferative Neoplasms (MPN) who have relapsed after haploidentical or HLA matched stem cell transplantation. The study also includes pediatric patients aged 12 and older with AML, MDS, and Juvenile Myelomonocytic Leukemia (JMML) who relapse after stem cell transplantation. This is a Phase I clinical trial focused on testing the safety and determining the appropriate dose of these investigational CIML NK cells, which have not yet been approved by the FDA for relapsed disease treatment. The treatment involves intravenous infusion of CIML NK cells on day 0. Prior to this, patients receive chemotherapy with fludarabine administered once daily for three doses starting on day -5, and cyclophosphamide given on days -5 and -4. This regimen is designed to prepare the body for CIML NK cell infusion. Both adult and pediatric patients undergo this treatment schedule. Participants will be closely monitored for safety over 6 weeks, with additional evaluations including objective response rate at 28 days, and assessments for leukemia-free survival and overall survival at 100 days and one year. Researchers will also track the incidence and severity of acute and chronic graft-versus-host disease over time. The study involves various tests such as bone marrow examinations, blood tests, and pregnancy tests when applicable. Participants must meet eligibility criteria and provide informed consent to join this study, which continues until the end date in December 2026.

Researchers are studying a new treatment for patients with relapsed or refractory myeloid malignancies, including acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and chronic myelomonocytic leukemia (CMML). This phase I/II open-label study aims to find the safest and most effective dose of CAR.70-engineered IL15-transduced cord blood-derived natural killer (NK) cells with TGF-beta receptor 2 knockout (TGFBR2 KO) combined with chemotherapy. The trial's goal is to assess safety, response rates, and several key outcomes related to disease control and patient survival. The treatment involves lymphodepleting and priming chemotherapy with dexamethasone, decitabine, fludarabine, and cyclophosphamide, followed by a one-time infusion of the modified NK cells. The study begins with a dose-escalation phase to determine the maximum tolerated dose (MTD) of these cells. After establishing the recommended phase 2 dose, two groups of patients will receive the treatment: one group with relapsed or refractory AML, and another with MDS or CMML after hypomethylating agent failure. Participants will undergo regular monitoring for safety and effectiveness, including assessments of response rates 30 days after infusion and safety follow-up for about one year. Researchers will also track remission rates, measurable residual disease, duration of response, relapse-free survival, overall survival, and both hematologic and non-hematologic side effects. Immune system recovery and the persistence of the infused cells will also be studied to better understand the treatment’s impact over time.

Researchers are evaluating the safety and tolerability of olutasidenib as a maintenance therapy for patients with IDH1-mutated myeloid malignancies after they have received a stem cell transplant. This phase II open-label study aims to learn how patients respond to olutasidenib in the post-transplant setting and assess various outcomes including progression-free survival, overall survival, relapse rates, and graft versus host disease. Starting 30 to 120 days after stem cell infusion, participants take olutasidenib by mouth twice daily for 28 days per cycle. These cycles repeat every 28 days for up to two years unless the disease progresses or unacceptable side effects occur. During the study, patients may have blood samples taken regularly and may undergo bone marrow biopsies, heart scans (ECHO/MUGA), and chest x-rays at screening. Participants will be closely monitored through clinical evaluations and laboratory tests throughout the study period, which can last up to two years. Researchers will measure safety outcomes, adverse events, and disease progression. The study includes follow-up to observe relapse and survival rates, along with assessments for graft versus host disease. The total participation time averages about one year for safety monitoring, with ongoing evaluations during treatment cycles.