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31 clinical trials in this condition
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ItalyIn short
This article provides detailed information about 30 ongoing clinical trials for myelofibrosis, a rare bone marrow disorder. These trials are testing various medications and treatment combinations across multiple countries in Europe, offering new hope for patients who have not responded well to current therapies or are seeking alternative treatment options.
This study provides continued access to itacitinib for patients who have previously participated in trials involving this medication and are benefiting from treatment. The trial includes patients with myelofibrosis, post-lung transplant bronchiolitis obliterans syndrome, or chronic graft-versus-host disease.
Main inclusion criteria: Patients must be currently enrolled in an Incyte-sponsored itacitinib-based study and receiving clinical benefit from treatment. They should be tolerating the medication well according to the study protocol and be compliant with study requirements.
Main exclusion criteria: Patients who have not been receiving clinical benefit from itacitinib-based therapy in an Incyte-sponsored clinical study are excluded.
Focus: The study evaluates the long-term safety of itacitinib for participants continuing treatment. Regular assessments monitor for adverse events and determine ongoing clinical benefits.
Investigational drug: Itacitinib is a JAK inhibitor administered orally in tablet form. It works by targeting Janus kinases involved in inflammation and immune responses.
This trial allows patients who previously participated in ruxolitinib studies to continue treatment. It evaluates the safety of ruxolitinib alone or in combination with other cancer therapies for myelofibrosis.
Main inclusion criteria: Patients must be enrolled in an Incyte-sponsored clinical study of ruxolitinib that has completed or been terminated. They should be tolerating treatment well and benefiting from it, with at least stable disease.
Main exclusion criteria: Patients with conditions other than myelofibrosis or outside the specified age range are excluded.
Focus: The study monitors patients receiving continued treatment with ruxolitinib to evaluate safety and effectiveness. Regular assessments track response to treatment and any adverse events.
Investigational drug: Ruxolitinib is a JAK inhibitor administered orally. It works by blocking enzymes that contribute to myelofibrosis symptoms, helping manage the condition.
This study compares imetelstat with best available therapy in patients with intermediate-2 or high-risk myelofibrosis who have not responded to JAK inhibitor treatment.
Main inclusion criteria: Patients must have a confirmed diagnosis of myelofibrosis, be classified as intermediate-2 or high-risk, and have tried JAK-inhibitor treatment for 3 to 6 months without success. They must have a measurable enlarged spleen and active symptoms.
Main exclusion criteria: Patients who experienced a return or worsening of their condition after JAK-inhibitor treatment or those outside the specified risk categories are excluded.
Focus: The trial evaluates whether imetelstat can improve overall survival compared to standard therapies. Secondary goals include assessing symptom response and spleen size reduction at 24 weeks.
Investigational drug: Imetelstat is a telomerase inhibitor administered intravenously. It targets enzymes that help cancer cells maintain their ability to divide and grow.
This trial tests pacritinib in patients with myelofibrosis who have severe thrombocytopenia. The study compares pacritinib with physician's choice therapies.
Main inclusion criteria: Patients must have primary myelofibrosis, post-essential thrombocythemia myelofibrosis, or post-polycythemia vera myelofibrosis with a platelet count below 50,000 per microliter. They must have an enlarged spleen and a symptom score of 10 or more.
Main exclusion criteria: Patients with serious medical conditions, uncontrolled high blood pressure, severe liver or kidney disease, or those who are pregnant or breastfeeding are excluded.
Focus: The study evaluates whether pacritinib can achieve at least a 35% reduction in spleen volume at 24 weeks. It also assesses symptom improvement and overall safety.
Investigational drug: Pacritinib is a kinase inhibitor taken orally as a capsule. It targets specific pathways involved in myelofibrosis, particularly in patients with low platelet counts.
This study evaluates RVU120 alone and in combination with ruxolitinib for intermediate or high-risk myelofibrosis.
Main inclusion criteria: Patients must be at least 18 years old with confirmed myelofibrosis, intermediate or high-risk disease, and adequate liver and kidney function. They must have a measurable enlarged spleen and active symptoms.
Main exclusion criteria: Patients with conditions other than myelofibrosis or outside the specified age range are excluded.
Focus: The trial assesses the effectiveness of RVU120 in treating myelofibrosis, either alone or with ruxolitinib. Regular monitoring tracks spleen size, symptom improvement, and safety.
Investigational drug: RVU120 is a targeted cancer therapy administered orally. It works by targeting specific molecular pathways crucial for cancer cell survival.
This trial tests TL-895 in patients with relapsed or refractory myelofibrosis or indolent systemic mastocytosis.
Main inclusion criteria: Patients must be at least 18 years old with a confirmed diagnosis of myelofibrosis or indolent systemic mastocytosis. They should have adequate blood, liver, and kidney function and active symptoms.
Main exclusion criteria: Patients with serious health conditions, those who are pregnant or breastfeeding, or those unable to follow study procedures are excluded.
Focus: The study determines the recommended dose and schedule of TL-895 and evaluates its effectiveness in improving symptoms. Regular assessments monitor safety and treatment response.
Investigational drug: TL-895 is a kinase inhibitor administered orally as a film-coated tablet. It targets specific signaling pathways involved in blood disorders.
This study investigates TP-3654 alone and in combination with momelotinib for intermediate or high-risk myelofibrosis.
Main inclusion criteria: Patients must be at least 18 years old with confirmed myelofibrosis, intermediate or high-risk disease according to DIPSS, and previous treatment with or failure of JAK inhibitors. They must have a measurable enlarged spleen.
Main exclusion criteria: Patients with conditions other than intermediate or high-risk myelofibrosis or vulnerable populations are excluded.
Focus: The trial determines the appropriate dosage of TP-3654 and evaluates its safety and preliminary effectiveness. The study tracks symptom changes and spleen size reduction.
Investigational drug: TP-3654 is an investigational drug administered orally. It targets specific pathways involved in myelofibrosis progression.
This study evaluates the long-term safety of luspatercept in patients who previously participated in luspatercept trials.
Main inclusion criteria: Patients must be at least 18 years old, enrolled in a previous luspatercept study, and continue to benefit from treatment. They must follow study visit schedules and agree to use effective birth control.
Main exclusion criteria: Patients with a history of progression to acute myeloid leukemia or other cancers, or those not meeting age or participation criteria, are excluded.
Focus: The trial monitors long-term safety, including progression to acute myeloid leukemia and development of other cancers. Regular assessments ensure ongoing safety and treatment efficacy.
Investigational drug: Luspatercept is an erythroid maturation agent administered via subcutaneous injection. It helps regulate red blood cell production.
This extension study evaluates the long-term safety of pelabresib in patients with myeloproliferative neoplasms, specifically myelofibrosis and essential thrombocythemia.
Main inclusion criteria: Patients must be at least 18 years old, currently receiving pelabresib in a previous study, and experiencing clinical benefits. They must agree to follow study procedures and use effective birth control.
Main exclusion criteria: Patients with conditions other than myeloproliferative neoplasms or outside the specified age range are excluded.
Focus: The study assesses long-term safety, overall survival, and leukemia-free survival. Regular monitoring tracks adverse events and treatment response.
Investigational drug: Pelabresib is a BET inhibitor administered orally in tablet form. It targets proteins involved in gene expression regulation, helping control abnormal cell growth.
This overview highlights 30 ongoing clinical trials for myelofibrosis across multiple European countries. Several notable patterns emerge from the data. Italy, Germany, France, and Spain feature prominently as trial locations, with Italy hosting at least 10 trials. Poland, Belgium, and Romania also have significant trial activity.
Many trials focus on patients who have not responded adequately to JAK inhibitor therapy, particularly ruxolitinib, which is a current standard treatment. Several studies investigate combination therapies, such as navtemadlin with ruxolitinib, selinexor with ruxolitinib, and navitoclax with ruxolitinib. There is also interest in developing treatments for patients with severe thrombocytopenia and anemia associated with myelofibrosis.
The trials employ various approaches, including telomerase inhibitors, kinase inhibitors, and targeted therapies. Several extension studies provide continued access to investigational medications for patients benefiting from previous trials. The research aims to improve outcomes for patients with intermediate-2 or high-risk myelofibrosis, particularly those with limited treatment options.
This guide is here to help you understand the condition. It does not replace a conversation with your doctor, who knows your situation best.
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