NCT01178281

Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence

A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 267 (actual)
• Sponsor: Celgene · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The objective of this study is to determine whether pomalidomide is safe and effective in reversing red blood cell (RBC)-transfusion-dependence in persons with myeloproliferative neoplasm (MPN)-associated myelofibrosis (global study) and in reversing anemia in Chinese with MPN-associated myelofibrosis and severe anemia not receiving RBC-transfusions (China extension study only)

Detailed description

The multicenter global study was conducted in 15 countries including Australia, Austria, Belgium, Canada, China, France, Germany, Italy, Japan, the Netherlands, Russia, Spain, Sweden, the United Kingdom, and the United States. The global study enrolled participants with myeloproliferative neoplasm (MPN)-associated myelofibrosis and RBC-transfusion-dependence. Participants were randomly assigned to receive pomalidomide or placebo in a blinded fashion. In most countries participating in the global study, RBC-transfusions are typically given for a hemoglobin level \<80-90 g/L. In China, RBC-transfusions are rarely given unless the hemoglobin level is \<60 g/L. Consequently, few Chinese with MPN-associated myelofibrosis meet RBC-transfusion-dependence criteria of the global study. A China-specific extension was developed to test the ability of pomalidomide to improve severe anemia (defined as a hemoglobin \< 80 g/L for ≥ 84 days in persons not receiving RBC-transfusions). The China-specific extension study consisted of a single-arm, open-label study in adults with MPN-associated myelofibrosis and severe anemia not receiving RBC transfusions with the objective of describing the frequency of anemia response. The Global (intent-to-treat \[ITT\] and safety) population in the main study and the China extension (ITT and safety) population are mutually exclusive.

Conditions

• Primary Myelofibrosis
• MPN-associated Myelofibrosis

Interventions

Type	Name	Description
DRUG	Pomalidomide 0.5 mg	Pomalidomide 0.5 mg capsule taken by mouth once daily. Immunomodulatory agent with demonstrated efficacy in the treatment of subjects with RBC-transfusion-dependence associated with MNP-associated myelofibrosis.
DRUG	Placebo	Placebo Comparator to active drug; Placebo capsule taken by mouth once daily
DRUG	Pomalidomide	Pomalidomide 0.5 mg capsule taken by mouth once daily.

Timeline

• Start date: 2010-09-08
• Primary completion: 2013-01-01
• Completion: 2018-05-15
• First posted: 2010-08-10
• Last updated: 2019-07-17
• Results posted: 2014-03-14

Locations

87 sites across 16 countries: United States, Australia, Austria, Belgium, Canada, China, France, Germany, Italy, Japan, Netherlands, Poland, Russia, Spain, Sweden, United Kingdom