Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01178281 | Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence | PHASE3 | COMPLETED | 267 | — | — | Sep 8, 2010 | May 15, 2018 | Jul 17, 2019 | 87 | United States, Australia +14 |
RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval.
A response in the China extension study was defined as an increase in hemoglobin ≥ 15 g/L above baseline value (in the absence of RBC transfusion) for ≥ 84 consecutive days.
| Arm | Type | Description |
|---|---|---|
| Pomalidomide 0.5 mg | EXPERIMENTAL | 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. |
| Placebo | PLACEBO_COMPARATOR | 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. |
| China Extension: Pomalidomide 0.5 mg | EXPERIMENTAL | 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. |
| Name | Type | Description |
|---|---|---|
| Pomalidomide 0.5 mg | DRUG | 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. |
| Placebo | DRUG | Placebo Comparator to active drug; Placebo capsule taken by mouth once daily |
| Pomalidomide | DRUG | Pomalidomide 0.5 mg capsule taken by mouth once daily. |
Inclusion Criteria: * Age ≥ 18 years * Myeloproliferative-neoplasm (MPN)-associated myelofibrosis * RBC-transfusion-dependence (global study): * Average RBC-transfusion frequency ≥ 2 units/28 days over at least the 84 days immediately prior to randomization. There must be no interval \> 42 days ...