Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis

NCT01149681 | Completed Phase 2 Results

• 1 other identifier: APL-B-020-10
• Study Type: interventional
• Target: 12
• Locations: 2 countries
• Sites: 2

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.

Conditions

Myelofibrosis

Keywords

Aplidin; Plitidepsin; Myelofibrosis; Pharma Mar

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate (ORR)

  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).

  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 (1)

• Quality of Life (QoL)

  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 (1)

• Progression-free Survival (PFS)

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

Study Arms (1)

Arm one

EXPERIMENTAL

Drug: APLIDIN (plitidepsin)

Interventions

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.

Arm one

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• PharmaMar: lead

Study Sites (2)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Azienda Ospedaliero Universitaria Careggi di Firenze

Florence, 50134, Italy

Location

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

plitidepsin

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Clinical Development Department of PharmaMar´s Oncology,Business Unit.,
• Organization: Pharma Mar, S.A.

clinicaltrials@pharmamar.com

+34 918466000

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 22, 2010
• First Posted: June 23, 2010
• Study Start: July 1, 2010
• Primary Completion: February 1, 2011
• Study Completion: February 1, 2011
• Last Updated: October 12, 2020
• Results First Posted: October 12, 2020

Record last verified: 2020-09

Locations

IT (1); US (1)