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 Flonoltinib Maleate Tablets in adults with intermediate- or high-risk myelofibrosis, a type of bone marrow disorder. This Phase III clinical trial compares Flonoltinib with the active control drug Ruxolitinib Phosphate. Participants are assigned to one of the two groups based on their risk level using the Dynamic International Prognostic Scoring System DIPSS. The study aims to measure how well these treatments reduce spleen size and improve symptoms over time. Participants take either Flonoltinib 75mg once daily or Ruxolitinib according to prescribed doses twice daily, both taken on an empty stomach. The trial is open-label and conducted at multiple centers, with approximately 105 participants planned for enrollment. Treatment continues until withdrawal criteria are met. The study includes evaluations at various time points up to 24 weeks, focusing on spleen volume reduction and symptom improvement. During the trial, participants undergo regular assessments including physical exams, spleen size measurements using MRI or CT scans, and symptom scoring using the MPN-SAF Total Symptom Score. Blood tests monitor organ function and safety. The primary outcome is the percentage of subjects achieving at least a 35% reduction in spleen volume at week 24. Secondary outcomes include time to spleen response, symptom score changes, and overall survival. Safety and laboratory tests are conducted throughout the study, which lasts several months from enrollment to final assessments.

Researchers are evaluating flonoltinib maleate tablets in adult patients with intermediate to high-risk myelofibrosis MF who are refractory, relapsed, or intolerant to JAK inhibitors. This single-arm, open-label, multicenter phase IIb clinical trial aims to assess the safety and efficacy of this treatment in patients who have limited options after prior JAK inhibitor therapy. Participants will receive flonoltinib maleate tablets orally once daily on an empty stomach. The dose, either 50mg or 75mg, is assigned based on platelet counts measured during the screening period. Treatment continues until participants meet withdrawal criteria. The study includes approximately 64 participants and focuses on those with splenomegaly and specific blood count and organ function criteria. During the study, participants will be monitored regularly for spleen volume reduction, symptom improvement using the MPN-SAF TSS score, and overall survival over 24 weeks. Safety and organ function tests, including blood counts and liver and kidney function, will be performed. Researchers will track treatment effects and side effects throughout the trial duration, with all assessments planned at various time points including weeks 2, 4, 8, 12, and 24.

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 JAB-8263, a small-molecule inhibitor targeting BET proteins, in adult patients with advanced malignant solid and hematologic tumors in this Phase 12a open-label study. The study aims to find the highest safe dose, assess dose-limiting toxicities, evaluate safety and tolerability, and gather early evidence of antitumor activity in patients whose tumors have progressed despite standard treatments or lack standard options. The study has two parts dose escalation and dose expansion, each enrolling about 30 subjects. Participants receive JAB-8263 orally every two days in 28-day cycles, with dose adjustments to determine the recommended Phase 2 dose. The study monitors how the drug behaves in the body and its preliminary effects on tumors. Participants will undergo evaluations over approximately 18 months, including safety assessments, pharmacokinetic measurements, and tumor response evaluations using standardized criteria. Researchers will record adverse events, response rates, and survival outcomes while monitoring dose-related toxicities. The study does not use randomization or masking, and participants are closely followed to assess treatment effects and safety.

Researchers are studying the use of unlicensed cryopreserved cord blood units CBUs for transplantation in both children and adults with blood cancers and other related disorders. This observational study involves patients with hematologic malignancies and various inherited and acquired disorders affecting the blood and immune system. The main goal is to monitor how well neutrophil recovery occurs after transplantation using these unlicensed CBUs in multiple institutions. Participants receive unlicensed cryopreserved CBUs as part of their transplant treatment. The study includes patients of any age receiving these CBUs for approved indications. The protocol focuses on the access and distribution of these unlicensed units rather than a specific treatment intervention. The study gathers data from recipients who receive these CBUs, tracking outcomes after transplantation. Participants are monitored for neutrophil recovery at 60 and 100 days after transplant, defined by a neutrophil count of at least 500mm3. Researchers also collect information on infection transmission, infusion reactions, survival rates at one year, and incidence of acute and chronic graft versus host disease. Platelet recovery is also evaluated. Safety and efficacy outcomes are followed over time to better understand the effects of unlicensed CBUs in this patient population.

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.