Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis

NCT ID: NCT01149681

Last Updated: 2020-10-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-07-31
• Study Completion Date: 2011-02-28

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.

Conditions

Myelofibrosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm one

Group Type: EXPERIMENTAL

APLIDIN (plitidepsin)

Intervention Type: 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.

Interventions

APLIDIN (plitidepsin)

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.

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

PharmaMar

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Mayo Clinic

Rochester, Minnesota, United States

Azienda Ospedaliero Universitaria Careggi di Firenze

Florence, , Italy

Countries

United States; Italy

Other Identifiers

APL-B-020-10

Identifier Type: -

Identifier Source: org_study_id