Clinical trial NCT01178281
Conditions: Primary Myelofibrosis MPN-associated Myelofibrosis
Keywords: Myelofibrosis Post-polycythemia vera myelofibrosis Post-essential thrombocythemia myelofibrosis RBC-transfusion-dependence Primary Myelofibrosis
Sponsor: Celgene
Start date: 2010-09-08; Completion date: 2018-05-15
Enrollment: 267 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief 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.

Arms: arm 1: Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects or disease progression.

Participants who were RBC-transfusion independent or experienced clinical benefit (defined as a reduction from Baseline of ≥ 50% in RBC-transfusion frequency during the prior 84-day interval) could continue to receive pomalidomide until loss of RBC-transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied. arm 2: Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression.

Participants who were RBC-transfusion independent or experienced clinical benefit could continue to receive placebo until loss of RBC- transfusion independence response or clinical benefit, or other criteria for treatment discontinuation applied. arm 3: Participants received pomalidomide 0.5 mg/day by mouth for at least 168 days unless there were unacceptable side effects, disease progression, or they received a RBC-transfusion.

Participants who experienced anemia response could continue treatment until the response was lost or other criteria for treatment discontinuation applied.
Interventions: intervention 1: Pomalidomide 0.5 mg intervention 2: Placebo intervention 3: Pomalidomide
Intervention descriptions: intervention 1: 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. intervention 2: Placebo Comparator to active drug; Placebo capsule taken by mouth once daily intervention 3: Pomalidomide 0.5 mg capsule taken by mouth once daily.

Protocol documents: