Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis
Condition: Myelofibrosis
MeSH terms: Primary Myelofibrosis
Intervention: APLIDIN (plitidepsin)
Intervention description: Aplidin® (plitidepsin) lyophilized powder and solvent for concentrate for solution for infusion. (2 mg plitidepsin vial and 4 ml ampoule).
Plitidepsin will be administered at 5 mg/m2 intravenously diluted to a total volume of 250 ml in 0.9% saline or 5% dextrose solution on Day 1 and 15 every four weeks for a maximum period of 6 cycles.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 12.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Aplidin; Plitidepsin; Myelofibrosis; Pharma Mar

Summary:

      This is an open-label, Phase II Clinical Trial of Aplidin® (plitidepsin) in Patients with
      Primary Myelofibrosis and post polycythemia vera/essential thrombocythemia (Post-PV/ET)
      Myelofibrosis.
    

Eligibility criteria:

        Inclusion Criteria:

          1. Diagnosis of Primary Myelofibrosis (PMF) or Post Polycythemia Vera/Essential
             Thrombocythemia Myelofibrosis(post-ET/PV MF) as per revised World Health Organization
             (WHO) criteria.

          2. High-risk or intermediate-2 risk Myelofibrosis (MF) as defined by the International
             Prognostic Scoring System (IPSS); or intermediate-I risk MF associated with
             symptomatic splenomegaly/hepatomegaly and/or unresponsive to available therapy.

          3. At least 18 years of age, with life expectancy of ≥12 weeks.

          4. Able to provide informed consent and being willing to sign an informed consent form
             (ICF).

          5. Eastern Cooperative Oncology Group (ECOG) performance status ≤2.

          6. Evidence of acceptable organ function within seven days of initiating study drug

        Exclusion Criteria:

          1. Previous treatment with plitidepsin.

          2. Any of the following therapies within two weeks prior to initiation of study drug:

               -  chemotherapy (e.g., hydroxyurea),

               -  immunomodulatory drug therapy (e.g., thalidomide),

               -  immunosuppressive therapy,

               -  corticosteroids >10 mg/day prednisone or equivalent, or

               -  erythropoietin.

          3. Incomplete recovery from major surgery within four weeks of study entry.

          4. Radiation therapy within four weeks of study entry.

          5. Women of childbearing potential

          6. Women who are pregnant or are currently breastfeeding.

          7. Myopathy grade > 2

          8. Known positive status for human immunodeficiency virus (HIV).

          9. Active hepatitis B or C virus (HBV or HCV) infection

         10. Diagnosis of another invasive malignancy

         11. Any acute active infection.

         12. Known hypersensitivity to the study drug or any of its formulation components (e.g.,
             Cremophor®).

         13. Treatment with any investigational product in the 30 days before inclusion in the
             study.