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 PETCT and 18F-FAPI PETMRI, 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 PETCT and 18F-FAPI PETMRI 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 PETMRI 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 evaluating the diagnostic effectiveness of 68Ga FAPI PETCT 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 PETCT 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 PETCT 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 PETCT 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 IbII 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.

This trial investigates TQ05105 tablets in adults with intermediate or high-risk myelofibrosis, a type of bone marrow cancer. It includes two groups one studying how the drug behaves in the body in participants with different levels of kidney function, and another assessing the drugs safety and effect in those who have not responded well or cannot tolerate previous Janus kinase JAK inhibitor treatments. The study is a phase II, open-label, single-arm, multicenter trial. Participants receive TQ05105 tablets in 28-day treatment cycles. The first group focuses on pharmacokinetics to guide dosing for normal to moderate kidney impairment. The second group evaluates the drugs safety and potential benefits in participants with spleen enlargement and prior JAK inhibitor therapy that was ineffective or intolerable. Treatment continues with regular dosing and monitoring. During the study, participants undergo assessments including measurements of spleen volume reduction and various pharmacokinetic parameters such as drug concentration over time. Safety is monitored through recording adverse events during treatment and up to 4 weeks after the last dose. Additional outcomes include symptom assessments, survival rates, and blood transfusion needs. The study lasts up to several years, with primary measurements at 24 weeks and extended follow-up averaging three years.

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 evaluating PRT12396, an investigational oral drug, in participants with high-risk polycythemia vera PV and myelofibrosis MF through a first-in-human, open-label, multi-center Phase 1 study. The study aims to assess the safety, tolerability, pharmacokinetics, and early effects of PRT12396 while determining the maximum tolerated dose and recommended dose for further study. Participants include those diagnosed with PV or MF, including specific subtypes with evidence of disease burden such as splenomegaly. The study is divided into two parts a dose-escalation phase where increasing oral doses of PRT12396 are given twice daily to evaluate safety and identify the best dose levels, followed by a dose-expansion phase where additional participants receive the recommended dose to further assess safety and early efficacy. Capsules are taken twice daily, swallowed whole with water, and may be taken one hour before or two hours after meals. Participants will be involved for an average of about two years, during which researchers will monitor dose-limiting toxicities, adverse events, dose modifications, and pharmacokinetic measures such as blood drug concentration. Other assessments include blood counts, spleen size, symptom scores, and patient global impressions of change. Safety and response outcomes will be tracked throughout the study to guide future research and treatment options.

Researchers are evaluating cytokine induced memory-like natural killer CIML NK cells combined with IL-2 in patients aged 12 years and older who have Acute Myeloid Leukemia AML, Myelodysplastic Syndromes MDS, Myeloproliferative Neoplasms MPN, or Juvenile Myelomonocytic Leukemia JMML that returned after stem cell transplantation. This Phase I clinical trial aims to test the safety and find the appropriate dose for this investigational treatment, which has not been approved by the FDA for relapsed disease. Participants receive intravenous infusions of CIML NK cells on day 0. Before this, patients are given chemotherapy drugs fludarabine daily for three doses starting on day -5 and cyclophosphamide on days -5 and -4 to prepare the body. The study includes both adult and pediatric patients who have relapsed after haploidentical or HLA-matched stem cell transplants. The treatment is followed for safety and response over several weeks. During the study, participants will be monitored closely for safety for six weeks and assessed for treatment response after 28 days. Further follow-ups include checking for leukemia-free survival and overall survival at 100 days and one year, as well as monitoring for acute and chronic graft-versus-host disease over several months to one year. The trial involves regular evaluations including bone marrow tests, blood tests, and clinical assessments to track disease status and side effects throughout participation.

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 III 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 trials 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 treatments 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 ECHOMUGA, 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.