Trial · NCT01178281
Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence
NCT01178281, Phase 3, Completed, Celgene; results first posted 2014-03-14
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence. Trials, record NCT01178281. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01178281
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence
- NCT number
- NCT01178281
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 267 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Primary Myelofibrosis, MPN-associated Myelofibrosis
- Interventions
- Pomalidomide 0.5 mg, Placebo, Pomalidomide
- First posted
- 2010-08-10
- Last updated
- 2019-07-17
- Other registries' IDs
- eudract:2010-018965-42
- Results first posted
- 2014-03-14
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Achieved RBC-Transfusion Independence | 168 days | RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval. |
| Primary | China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days | From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks. | 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. |
| Secondary | Overall Survival | From first dose of study drug up to end of study; median follow-up time was 19.1 months in the pomalidomide 0.5 mg arm and 17.6 months in the placebo arm. | The time from randomization to the death or to the latest date when participants are known to be alive. Overall survival was analyzed using Kaplan-Meier method; participants who were alive or lost to follow-up were censored at the latest date they were known to be alive. |
| Secondary | Duration of RBC-Transfusion Independence | From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm. | The duration of RBC-transfusion independence is the time from the date at which the first RBC-transfusion independence started to the date of another RBC-transfusion given at least 84 days after the time the transfusion independence started. The duration of the RBC-transfusion independence was analyzed using the Kaplan-Meier method. Data were censored at the end of the treatment phase for participants who had not received another RBC-transfusion after the start of transfusion independence by the end of treatment phase. |
| Secondary | Time to RBC-Transfusion Independence | 168 days | Time to response was measured from first dose of study drug to the start of the first response. The start date of the response was defined as one day after the last date of an RBC-transfusion for participants who received a RBC-transfusion after the first dose, and as the date of the first dose of study drug for participants who received no RBC-transfusions during the 84 days after the first dose of study drug. |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAE) | From the first dose of study drug until 28 days after last dose; median treatment duration was 23.7 weeks in the pomalidomide arm, 23.9 weeks in the placebo arm, and 24.0 weeks in the China extension pomalidomide arm. | A TEAE is an adverse event (AE) that starts on or after the first dose of study drug. The severity of each AE was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE),Version 4.0 and according to the following scale: Grade 1 = Mild (transient or mild discomfort; no limitation in activity; no medical intervention/therapy required); Grade 2 = Moderate (mild to moderate limitation in activity, some assistance may be needed; minimal medical intervention/therapy required); Grade 3 = Severe (marked limitation in activity, assistance usually required; medical intervention/therapy required, hospitalization possible); Grade 4 = Life-threatening (extreme limitation in activity, significant assistance or medical intervention/therapy required, hospitalization or hospice care probable); Grade 5 = Death Drug-related (related) AEs are those suspected by the Investigator as being related to administration of study drug |
| Secondary | Healthcare Resource Utilization | From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm. | |
| Secondary | Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score | Baseline and Days 85 and 169 | EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). For the health state profile participants rate their perceived health state today on 5 dimensions: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression on a Likert-type scale from 1 to 3, where 1 = "no problems," 2 = "some problems," and 3 = "extreme problems." The EQ-5D Health Utility Index (HUI) was generated from the five health state domain scores, and ranges from -0.594 (worst) and 1 (best) imaginable health state, with -0.594 representing an "unconscious" health state. |
| Secondary | Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale | Baseline and Days 85 and 169 | EQ-5D is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D includes 2 components: the EQ-5D health state profile (descriptive system) and the EQ-5D visual analog scale (VAS). On the VAS the participant rates his/her health state on a line from 0 (worst imaginable health) to 100 (best imaginable health). |
| Secondary | Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score | Baseline and Days 85 and 169 | The FACT-An is a 47-item, cancer-specific questionnaire consisting of a core 27-item general questionnaire measuring the four general domains of QoL (physical, social/family, emotional and functional well-being), and an additional 20-item anemia questionnaire (FACT-An Anemia subscale) that measures 13 fatigue-associated items (FACT-F Fatigue subscale) and seven non-fatigue-related items. Each item is scored using a 5-point Likert rating scale (0 = Not at all; 1 = A little bit; 2 = Somewhat; 3 = Quite a bit; and 4 = Very much). FACT-An total score is calculated by adding all the FACT-An subscales together. The total score ranges from 0-188 with higher scores representing better QOL. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Who Achieved RBC-Transfusion Independence | 168 days | Global study intent to treat population (ITT) which includes all participants randomized to either of the two study drugs, regardless of whether or not any study drug was actually taken. | percentage of participants | NUMBER |
| PRIMARY | China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days | From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks. | China extension intent-to-treat population, which includes all participants enrolled in the China extension study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Overall Survival | From first dose of study drug up to end of study; median follow-up time was 19.1 months in the pomalidomide 0.5 mg arm and 17.6 months in the placebo arm. | Global study intent-to-treat population | months | MEDIAN |
| SECONDARY | Duration of RBC-Transfusion Independence | From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm. | Global study intent-to-treat population with an 84-day RBC-transfusion independence response | months | MEDIAN |
| SECONDARY | Time to RBC-Transfusion Independence | 168 days | Global study intent-to-treat population with an 84-day RBC-transfusion independence response | weeks | MEDIAN |
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAE) | From the first dose of study drug until 28 days after last dose; median treatment duration was 23.7 weeks in the pomalidomide arm, 23.9 weeks in the placebo arm, and 24.0 weeks in the China extension pomalidomide arm. | Participants who received at least 1 dose of study drug | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Healthcare Resource Utilization | From first dose of study drug up to 28 days after last dose, as of the data cut-off date of 16 Jan 2013; median treatment duration was 23.6 weeks in the pomalidomide arm and 23.9 weeks in the placebo arm. | Analysis of healthcare resource utilization data were not collected during the study and no analysis were performed. | ||
| SECONDARY | Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score | Baseline and Days 85 and 169 | Global study intent-to-treat population with available data at baseline and each time point | score on a scale | MEAN |
| SECONDARY | Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale | Baseline and Days 85 and 169 | Global study intent-to-treat population with available data at baseline and each time point. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score | Baseline and Days 85 and 169 | Global study intent-to-treat population with available data at baseline and each time point. | units on a scale | MEAN |
Source ClinicalTrials.gov
Results by group69
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Who Achieved RBC-Transfusion Independence | Pomalidomide 0.5 mg | 17.3 | 95% Confidence Interval 11.88 to 23.84 | percentage of participants | |
| Percentage of Participants Who Achieved RBC-Transfusion Independence | Placebo | 16.7 | 95% Confidence Interval 9.42 to 26.38 | percentage of participants | |
| China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days | China Extension: Pomalidomide 0.5 mg | 1 | Participants | ||
| Overall Survival | Pomalidomide 0.5 mg | 24.2 | 95% Confidence Interval 19.5 to 33.5 | months | |
| Overall Survival | Placebo | 26.2 | 95% Confidence Interval 18.0 to 32.6 | months | |
| Duration of RBC-Transfusion Independence | Pomalidomide 0.5 mg | NA | 95% Confidence Interval 4.8 to NA | months | |
| Duration of RBC-Transfusion Independence | Placebo | 5.8 | 95% Confidence Interval 3.0 to NA | months | |
| Time to RBC-Transfusion Independence | Pomalidomide 0.5 mg | 6.9 | Full Range 0.1 to 20.1 | weeks | |
| Time to RBC-Transfusion Independence | Placebo | 2.4 | Full Range 0.1 to 15.4 | weeks | |
| Number of Participants With Treatment-emergent Adverse Events (TEAE) | Pomalidomide 0.5 mg | Any adverse event (AE) | 164 | Participants |
1 to 10 of 69Source ClinicalTrials.gov
See all 69 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pomalidomide 0.5 mg | 168 | |
| Overall Study | STARTED | Placebo | 84 | |
| Overall Study | STARTED | China Extension: Pomalidomide 0.5 mg | 15 | |
| Overall Study | Received Study Drug | Pomalidomide 0.5 mg | 167 | |
| Overall Study | Received Study Drug | Placebo | 83 | |
| Overall Study | Received Study Drug | China Extension: Pomalidomide 0.5 mg | 15 | |
| Overall Study | COMPLETED | Pomalidomide 0.5 mg | 168 | |
| Overall Study | COMPLETED | Placebo | 84 | |
| Overall Study | COMPLETED | China Extension: Pomalidomide 0.5 mg | 15 | |
| Overall Study | NOT COMPLETED | Pomalidomide 0.5 mg | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events498
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | ANAEMIA | Blood and lymphatic system disorders | Global Study: Pomalidomide 0.5 mg | 12 | 167 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Global Study: Placebo | 5 | 83 | |
| other | ANAEMIA | Blood and lymphatic system disorders | China Extension: Pomalidomide 0.5 mg | 2 | 15 | |
| other | NEUTROPENIA | Blood and lymphatic system disorders | Global Study: Pomalidomide 0.5 mg | 25 | 167 | |
| other | NEUTROPENIA | Blood and lymphatic system disorders | Global Study: Placebo | 4 | 83 | |
| other | NEUTROPENIA | Blood and lymphatic system disorders | China Extension: Pomalidomide 0.5 mg | 0 | 15 | |
| other | THROMBOCYTOPENIA | Blood and lymphatic system disorders | Global Study: Pomalidomide 0.5 mg | 21 | 167 | |
| other | THROMBOCYTOPENIA | Blood and lymphatic system disorders | Global Study: Placebo | 12 | 83 | |
| other | THROMBOCYTOPENIA | Blood and lymphatic system disorders | China Extension: Pomalidomide 0.5 mg | 1 | 15 | |
| other | ARRHYTHMIA | Cardiac disorders | Global Study: Pomalidomide 0.5 mg | 2 | 167 |
1 to 10 of 498Source ClinicalTrials.gov
See all 498 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Global Study: Pomalidomide 0.5 mg | 115 | 167 |
| Total, all-cause mortality | Global Study: Placebo | 54 | 83 |
| Total, all-cause mortality | China Extension: Pomalidomide 0.5 mg | 8 | 15 |
| Total, other adverse events | Global Study: Pomalidomide 0.5 mg | 155 | 167 |
| Total, other adverse events | Global Study: Placebo | 76 | 83 |
| Total, other adverse events | China Extension: Pomalidomide 0.5 mg | 12 | 15 |
| Total, serious adverse events | Global Study: Pomalidomide 0.5 mg | 76 | 167 |
| Total, serious adverse events | Global Study: Placebo | 29 | 83 |
| Total, serious adverse events | China Extension: Pomalidomide 0.5 mg | 0 | 15 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01178281
GET https://conductscience.org/api/trials/v1/NCT01178281/results?page=2