Clinical Trial of Aplidin® in Patients With Primary Myelofibrosis

Pharma Mar

Status and phase: Completed; Phase 2

Conditions: Myelofibrosis

Treatments: Drug: APLIDIN (plitidepsin)

Study type: Interventional

Funder types: Industry

Identifiers

NCT01149681

APL-B-020-10

Details and patient eligibility

About

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.

Full 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.

Enrollment: 12 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

Arm one

Experimental group

Treatment:

Drug: APLIDIN (plitidepsin)