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Primary myelofibrosis – Trials in Disease

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Ongoing Clinical Trials for Primary Myelofibrosis

Primary myelofibrosis is a rare bone marrow disorder being studied in 8 clinical trials across Europe. These trials are testing various investigational medications, including JAK inhibitors, combination therapies, and novel treatments, with the goal of reducing spleen size, improving symptoms, and decreasing the need for blood transfusions in patients with this condition.

Clinical trial locations

Study of Pacritinib for Patients with Severe Thrombocytopenia in Myelofibrosis Conditions

This trial is testing pacritinib, a medication taken as a capsule, for patients with severe thrombocytopenia, a condition where platelet counts are dangerously low. The study compares pacritinib with other treatments chosen by doctors, such as hydroxycarbamide, ruxolitinib, or various corticosteroids.

Main inclusion criteria: Patients must have one of three types of the condition—primary, post-essential thrombocythemia, or post-polycythemia vera forms. They need to have very low platelet counts (less than 50,000 per microliter), a palpable spleen enlargement of at least 5 cm, and significant symptoms with scores reflecting pain, itching, or night sweats. Participants must be at least 18 years old and have adequate organ function.

Main exclusion criteria: The trial excludes patients with other serious medical conditions, recent heart attacks or strokes, uncontrolled blood pressure, severe liver or kidney disease, pregnancy or breastfeeding, active participation in other trials, allergic reactions to similar medications, certain cancer histories, and active infections requiring treatment.

Main focus: The study aims to evaluate whether pacritinib can significantly reduce spleen size and improve symptom scores compared to physician-selected treatments over 24 weeks. Regular imaging tests like MRI or CT scans will measure these outcomes.

Investigational drug: Pacritinib is a kinase inhibitor that targets specific pathways involved in the disease, aiming to reduce spleen size and improve symptoms in patients with severe thrombocytopenia due to this bone marrow disorder.

Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

This study evaluates the long-term safety of luspatercept, administered by subcutaneous injection, in patients who previously participated in other luspatercept trials. It focuses on monitoring for progression to acute myeloid leukemia or development of other cancers.

Main inclusion criteria: Participants must be at least 18 years old, willing to follow study schedules, currently enrolled in a previous luspatercept study while benefiting from treatment, or ready to start treatment after previously receiving placebo. Women of childbearing potential need negative pregnancy tests and must agree to use effective birth control.

Main exclusion criteria: Patients who have not participated in previous luspatercept trials, those with a history of progression to acute myeloid leukemia, those who have developed other cancers or pre-cancerous conditions, and those falling outside specified criteria are excluded.

Main focus: The study monitors long-term safety, specifically looking for progression to serious blood cancers and other malignancies. Participants receive luspatercept injections with regular safety assessments over an extended period up to 360 days.

Investigational drug: Luspatercept is an erythroid maturation agent that helps the body produce more red blood cells by binding to certain proteins, potentially reducing the need for transfusions.

Study of Momelotinib and Luspatercept for Patients with Transfusion-Dependent Myelofibrosis

This trial studies a combination treatment using momelotinib (oral tablet) and luspatercept (subcutaneous injection) for patients dependent on blood transfusions. The study aims to assess if this combination can help patients achieve transfusion independence.

Main inclusion criteria: Participants must be at least 18 years old with confirmed diagnoses of primary or post-polycythemia vera/essential thrombocythemia forms. They must require regular transfusions or have low hemoglobin levels, be classified as high or intermediate risk, have no planned stem cell transplants, and possess a life expectancy greater than 24 weeks. Women of childbearing potential must have negative pregnancy tests and use effective birth control.

Main exclusion criteria: While detailed exclusion criteria are limited in the provided data, patients with the condition itself cannot participate, which appears to be a data inconsistency.

Main focus: The study evaluates whether the combination therapy can achieve transfusion independence by week 24, meaning patients would not need blood transfusions for at least 12 consecutive weeks (except in cases of significant bleeding).

Investigational drugs: Momelotinib is a JAK inhibitor targeting pathways involved in blood cell production, while luspatercept helps increase red blood cell production, together aiming to reduce transfusion needs and improve quality of life.

Study on Tasquinimod for Patients with Myelofibrosis Who Are Refractory or Intolerant to JAK2 Inhibitors

This trial tests tasquinimod, taken as a capsule in varying doses (0.25 mg, 0.5 mg, and 1 mg), for patients who have not responded to or cannot tolerate JAK inhibitor treatments.

Main inclusion criteria: Patients must have a confirmed diagnosis based on a bone marrow test not older than six months, have tried JAK inhibitors without success or found them intolerable, be classified as intermediate-1 with symptoms or higher risk, have spleen enlargement of at least 5 cm, be at least 18 years old, have peripheral blood blast counts under 10%, and maintain adequate performance status. They must be able to swallow oral medication and provide informed consent.

Main exclusion criteria: Patients unable to stop interfering treatments, those with severe uncontrolled heart problems, severe liver or kidney disease, recent cancer history (within 5 years except non-melanoma skin cancer), pregnancy or breastfeeding, active infections requiring antibiotics, known allergies to the study drug, recent participation in other trials (within 30 days), or history of substance abuse (within 2 years) are excluded.

Main focus: The two-phase study first determines the safe dosage of tasquinimod, then evaluates its effectiveness in reducing spleen size after 24 weeks using imaging techniques like MRI or CT scans.

Investigational drug: Tasquinimod is an anti-cancer agent taken orally once daily that targets proteins contributing to cancer cell growth and survival, specifically being tested for patients resistant to or intolerant of JAK2 inhibitors.

Study of Navtemadlin for Patients with Myelofibrosis Resistant to JAK Inhibitors

This study investigates navtemadlin (KRT-232), an oral medication, for patients with primary or secondary forms who have not responded well to JAK inhibitor treatments. Participants are randomly assigned to receive either navtemadlin or the best available therapy.

Main inclusion criteria: Adults aged 18 or older with confirmed diagnoses of primary, post-polycythemia vera, or post-essential thrombocythemia forms must have high, intermediate-2, or intermediate-1 risk levels according to the Dynamic International Prognostic System. They must have previously tried JAK inhibitors without success, have an ECOG performance status of 2 or less, and have TP53 wild-type disease confirmed by central laboratory testing.

Main exclusion criteria: Specific exclusion criteria are not detailed in the provided data beyond general clinical trial standards.

Main focus: The trial aims to determine if navtemadlin can achieve significant spleen volume reduction by week 24, along with symptom score improvements, transfusion independence, and overall survival rates.

Investigational drug: KRT-232 is an anti-cancer agent that promotes cancer cell death by inhibiting a protein that helps cancer cells survive, specifically being tested for patients who haven’t responded to JAK inhibitor treatments.

Study of Selinexor for Patients with Previously Treated Myelofibrosis

This trial evaluates selinexor, a film-coated tablet, compared to physician-chosen treatments for patients who have already received treatment for the condition.

Main inclusion criteria: Patients must have a confirmed diagnosis with recent pathology reports, adequate kidney function (creatinine clearance greater than 15 mL/min), be at least 18 years old, have ECOG performance status of 2 or less, have received JAK inhibitor treatment for at least 6 months, show measurable spleen enlargement (450 cm³ or more), and have disease that has returned, not responded, or cannot tolerate JAK inhibitors. Platelet counts must be 75 x 10⁹/L or higher, with adequate neutrophil counts and liver function tests.

Main exclusion criteria: Patients without previous treatment, those outside specified age ranges, and vulnerable populations are excluded.

Main focus: The study compares the effectiveness and safety of selinexor versus physician-selected treatments, monitoring spleen size changes, symptom improvements, and overall health over the treatment period.

Investigational drug: Selinexor is a selective inhibitor of nuclear export that blocks a protein helping cancer cells grow and spread, aiming to slow or stop disease progression as a targeted cancer therapy.

Study on Luspatercept for Treating Anemia in Adults with Myelofibrosis on JAK2 Inhibitor Therapy Requiring Red Blood Cell Transfusions

This clinical trial tests luspatercept (ACE-536) administered by subcutaneous injection for treating anemia in patients already receiving JAK2 inhibitor therapy and requiring regular blood transfusions. The study compares luspatercept with placebo.

Main inclusion criteria: Participants must be at least 18 years old with confirmed primary or post-condition diagnoses. They need to require regular red blood cell transfusions (4 to 12 units over 12 weeks with no transfusion-free gap longer than 6 weeks). Patients must be on continuous JAK2 inhibitor therapy for at least 32 weeks with a stable dose for 16 weeks before enrollment, expected to continue for 24 weeks after. ECOG performance scores must be 2 or less. Women of childbearing potential require negative pregnancy tests and effective birth control.

Main exclusion criteria: Patients without anemia related to myeloproliferative neoplasm-associated disease, those not receiving JAK2 inhibitor treatment, and those not requiring regular transfusions are excluded.

Main focus: The study aims to determine if luspatercept can reduce the need for blood transfusions over 24 weeks, with participants monitored for achieving transfusion-free periods of at least 12 consecutive weeks.

Investigational drug: Luspatercept is an erythroid maturation agent that binds to proteins regulating red blood cell production, helping the body generate more red blood cells and potentially reducing transfusion needs.

Study on the Safety and Effectiveness of Navtemadlin and Ruxolitinib for Patients with Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib

This study examines a combination treatment of navtemadlin (KRT-232) and ruxolitinib, both taken orally as tablets, for patients who haven’t responded adequately to ruxolitinib alone.

Main inclusion criteria: Adults over 18 years old must have confirmed diagnoses of primary, post-polycythemia vera, or post-essential thrombocythemia forms. They must have been treated with ruxolitinib for at least 18 weeks with a stable dose for 8 weeks before enrollment. Spleen must be enlarged to at least 5 cm below the rib cage or have a volume of at least 450 cm³. Patients must have at least 2 disease-related symptoms with specific scores, undergo spleen imaging within 14 days before starting treatment, have ECOG performance status of 0 to 2, demonstrate adequate organ function, and agree to use effective birth control during the study.

Main exclusion criteria: Specific detailed exclusions are not provided in the source data beyond standard clinical trial criteria.

Main focus: The trial determines the optimal dosage of KRT-232 when combined with ruxolitinib and evaluates whether this combination can achieve at least a 35% reduction in spleen volume by week 24, along with symptom improvements and reduced transfusion needs.

Investigational drugs: KRT-232 is an MDM2 inhibitor that promotes cancer cell death by activating the p53 pathway, while ruxolitinib is an established JAK inhibitor that blocks enzymes involved in blood cell overproduction, together aiming to provide better results for patients.

Summary

Eight ongoing clinical trials are investigating various treatment approaches for primary myelofibrosis across multiple European countries. Italy and France appear most frequently as trial locations, followed by Spain, with representation across Eastern, Western, and Southern Europe.

Several investigational drugs are being tested: pacritinib for patients with severe thrombocytopenia, luspatercept in multiple studies focusing on transfusion dependence and anemia, tasquinimod and navtemadlin for JAK inhibitor-resistant cases, and selinexor for previously treated patients. Notably, some trials explore combination therapies, such as momelotinib with luspatercept, and navtemadlin with ruxolitinib.

The trials primarily target patients who have either failed previous JAK inhibitor therapy or require blood transfusions. Common outcome measures include spleen volume reduction, symptom improvement, transfusion independence, and overall safety monitoring. Most studies focus on patients classified as intermediate or high risk according to established prognostic scoring systems.

These trials represent important research efforts to address unmet medical needs in patients with this challenging bone marrow disorder, particularly for those who haven’t responded adequately to current standard treatments.

Ongoing Clinical Trials on Primary myelofibrosis

  • Study on Long-Term Safety of Luspatercept for Patients with Myelodysplastic Syndrome, Beta-Thalassemia, or Myelofibrosis Who Previously Participated in Luspatercept Trials

    Recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria France Germany Greece Italy The Netherlands +2

  • Study of Momelotinib and Luspatercept for Patients with Transfusion-Dependent Myelofibrosis

    Not yet recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Spain

  • Study on Apixaban, Rivaroxaban, and Aspirin for Preventing Blood Clots in Patients with JAK2V617F-Positive Myeloproliferative Neoplasms

    Not yet recruiting

    4 1 1 1
    Investigated diseases:
    Investigated drugs:
    France

  • Study on Tasquinimod for Patients with Myelofibrosis Who Are Refractory or Intolerant to JAK2 Inhibitors

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany The Netherlands

  • Study of Navtemadlin for Patients with Myelofibrosis Resistant to JAK Inhibitors

    Not recruiting

    4 1 1 1
    Investigated diseases:
    Investigated drugs:
    Bulgaria Croatia Czechia France Germany Greece +7

  • Study on Luspatercept for Treating Anemia in Adults with Myelofibrosis on JAK2 Inhibitor Therapy Requiring Red Blood Cell Transfusions

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia France Germany Greece +6

  • Study of Fedratinib and Nivolumab for Patients with Myelofibrosis Resistant to JAK-inhibitor Treatment

    Not recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

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