Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01149681
• Date of registration: 22/06/2010
• Prospective Registration: Yes
• Primary sponsor: PharmaMar
• Public title: Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis
• Scientific 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
• Date of first enrolment: July 2010
• Target sample size: 12
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01149681
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Italy, United States

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Myelofibrosis

Intervention(s)

Drug: APLIDIN (plitidepsin)

Primary Outcome(s)

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]

Secondary Outcome(s)

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]

Secondary ID(s)

APL-B-020-10

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 12/10/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01149681