Clinical trial NCT01149681: Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis
Official title: Open-label, Phase II Clinical Trial of Aplidin® (Plitidepsin) in Patients With Primary Myelofibrosis (PMF) and Post Polycythemia Vera/Essential Thrombocythemia (Post-PV/ET) Myelofibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Myelofibrosis
Keywords: Aplidin
Plitidepsin
Myelofibrosis
Pharma Mar
Lead sponsor: PharmaMar
Sponsor class: INDUSTRY
First posted: 2010-06-23
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed description: This trial tries to assess response rate (ORR) of plitidepsin in patients with:

primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocythemia myelofibrosis (post-ET MF). Besides, the study results will allow to evaluate the effect of plitidepsin on bone marrow (BM) or peripheral blood histology and to determine the quality of life (QoL) and symptoms or participant patients.

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.

Primary outcomes:
measure: Objective Response Rate (ORR)
timeFrame: All patients were followed up to progressive disease, start of a new anti-cancer therapy, death or one year after the last treatment visit of the last patient, whichever occured first
Secondary outcomes:
measure: Quality of Life (QoL)
timeFrame: All patients were followed up to progressive disease, start of a new anti-cancer therapy, death or one year after the last treatment visit of the last patient, whichever occured first
Other outcomes:
measure: Progression-free Survival (PFS)
timeFrame: All patients were followed up to progressive disease or death, whichever occured first, up to 30 days after their last dose
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Careggi di Firenze
Study officials:
References: