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Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis

Primary Purpose

Myelofibrosis

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

APLIDIN (plitidepsin)

About this trial

This is an interventional treatment trial for Myelofibrosis focused on measuring Aplidin, Plitidepsin, Myelofibrosis, Pharma Mar

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Mayo Clinic
Azienda Ospedaliero Universitaria Careggi di Firenze

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm one

Arm Description

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

Quality of Life (QoL)

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.

Full Information

NCT ID

NCT01149681

First Posted

June 22, 2010

Last Updated

September 17, 2020

Sponsor

PharmaMar

1. Study Identification

Unique Protocol Identification Number

NCT01149681

Brief Title

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

2. Study Status

Record Verification Date

September 2020

Overall Recruitment Status

Completed

Study Start Date

July 2010 (undefined)

Primary Completion Date

February 2011 (Actual)

Study Completion Date

February 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

PharmaMar

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Myelofibrosis

Keywords

Aplidin, Plitidepsin, Myelofibrosis, Pharma Mar

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm one

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

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.

Primary Outcome Measure Information:

Title

Objective Response Rate (ORR)

Description

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

Secondary Outcome Measure Information:

Title

Quality of Life (QoL)

Description

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

Other Pre-specified Outcome Measures:

Title

Progression-free Survival (PFS)

Description

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.

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Facility Information:

Facility Name

Mayo Clinic

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

Facility Name

Azienda Ospedaliero Universitaria Careggi di Firenze

City

Firenze

ZIP/Postal Code

50134

Country

Italy

12. IPD Sharing Statement

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Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis

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