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NCT Number: NCT01149681

Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis

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.

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Key information

Conditions

Myelofibrosis Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myeloproliferative Disorders Primary Myelofibrosis

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Azienda Ospedaliero Universitaria Careggi di Firenze, Florence, Italy

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of Primary Myelofibrosis (PMF) or Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis(post-ET/PV MF) as per revised World Health Organization (WHO) criteria.
  • 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.
  • At least 18 years of age, with life expectancy of ≥12 weeks.
  • Able to provide informed consent and being willing to sign an informed consent form (ICF).
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤2.
  • Evidence of acceptable organ function within seven days of initiating study drug

Exclusion criteria

  • Previous treatment with plitidepsin.
  • 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.
  • Incomplete recovery from major surgery within four weeks of study entry.
  • Radiation therapy within four weeks of study entry.
  • Women of childbearing potential
  • Women who are pregnant or are currently breastfeeding.
  • Myopathy grade > 2
  • Known positive status for human immunodeficiency virus (HIV).
  • Active hepatitis B or C virus (HBV or HCV) infection
  • Diagnosis of another invasive malignancy
  • Any acute active infection.
  • Known hypersensitivity to the study drug or any of its formulation components (e.g., Cremophor®).
  • Treatment with any investigational product in the 30 days before inclusion in the study.

Treatment and study plan

APLIDIN (plitidepsin)

Drug

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.

Primary outcomes

  1. Objective Response Rate (ORR)

    Time frame: 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

    Objective response rate (ORR) of plitidepsin in patients with: primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.

    ORR according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) response criteria (Tefferi et al., 2006) in the evaluable population: defined as a confirmed disease response, on two consecutive evaluations performed at least eight weeks apart. Overall response (OR) = Complete Response (CR) + Partial response (PR) + Clinical improvement (CI).

Secondary outcomes

  1. Quality of Life (QoL)

    Time frame: 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

    Quality of life (QoL) and symptoms assessment according to the Myelofibrosis Symptom Assessment Form (MFSAF), after treatment with plitidepsin. For full details please refer to Mesa RA, Schwager S, Radia D, Cheville A, Hussein K, Niblack J, et al. The Myelofibrosis Symptom Assessment Form (MFSAF): an evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis. Leuk Res 2009;33(9):1199-203.

    Scale measures: 0 to 10 (0 if absent) ranking being 1 the most favorable and 10 least favorable.

Other outcomes

  1. Progression-free Survival (PFS)

    Time frame: All patients were followed up to progressive disease or death, whichever occured first, up to 30 days after their last dose

    Progression free survival (PFS) is defined as the time from start of treatment to the date of documented progressive disease (PD) by IWG-MRT criteria or death (regardless of the cause of death), whichever comes first. Patients who progress or die will be considered to have had an event, except if this event occurs after the start of subsequent antitumor therapy, in which case the patient will be censored at the time of last disease assessment prior to or on the first day of the first subsequent antitumor therapy. If the patient is lost for the assessment of progression during the follow-up period, or has more than one missing follow-up between the date of last tumor assessment and the date of progression, death or further antitumor therapy, the PFS will be censored at the date of last valid disease assessment before the missing evaluations.

Sponsors and collaborators

Lead sponsor

PharmaMar

Industry

Registry information

Official study 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

Important dates

Study start
2010
Primary completion
2011
Study completion
2011
First posted
Jun 23, 2010
Registry last updated
Oct 12, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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