Trial · NCT00463385
A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia
NCT00463385, Phase 2, Completed, Celgene; results first posted 2013-10-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia. Trials, record NCT00463385. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00463385
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia
- NCT number
- NCT00463385
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 88 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Myelofibrosis With Myeloid Metaplasia, Myeloid Metaplasia, Myelofibrosis
- Interventions
- Pomalidomide, Prednisone, Placebo to pomalidomide, Placebo to prednisone
- First posted
- 2007-04-20
- Last updated
- 2019-11-20
- Results first posted
- 2013-10-28
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 With a Clinical Response Within the First 6 Cycles of Treatment | Up to 168 days | A clinical responder was defined as either: 1. A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or 2. A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or 3. A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at \> 5 cm and became not palpable. Participants who discontinued the study early without achieving clinical response were counted as non-responders. |
| Secondary | Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment | Up to 336 days | A clinical responder was defined as either: 1. A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or 2. A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or 3. A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at \> 5 cm and became not palpable. Participants who discontinued the study early without achieving clinical response were counted as non-responders. |
| Secondary | Time to the First Clinical Response | Up to 168 days | The time to the first clinical response achieved within 168 days after the first study drug dosing date was calculated for participants who achieved a clinical response as: Start date of the first clinical response - the first study drug date +1. A clinical responder was defined as either: 1. A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or 2. A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or 3. A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at \> 5 cm and became not palpable. |
| Secondary | Duration of First Clinical Response | Up to 40 months | For RBC-transfusion-dependent patients, duration of response was calculated as the last day of response - first day of response +1, where the last day of response was the date of the first RBC-transfusion administrated at or more than 56 days after the response started. For patients who did not receive a subsequent transfusion after the response started, the end date of response was censored at the day of last hemoglobin assessment. For RBC-transfusion-independent patients, the duration of response was calculated as the last day of response - first day of response +1, where the last day of response was the earlier of the date of a hemoglobin increase of \< 2.0 g/dL and the date of a RBC transfusion at ≥ 56 days after the response started. For patients whose hemoglobin measurements were always ≥ 2.0 g/dL and never received a RBC transfusion after response started, the end date of the response was censored at the date of last hemoglobin measurement. Kaplan-Meier methodology was used. |
| Secondary | Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Subscale and Total Scores | Baseline and Cycle 6 (168 days). | The FACT-An comprises the four subscales of the 27-item FACT-General Scale (FACT-G), Physical Well-being, Social/Family Well-being, Emotion Well-being, Functional Well-Being, and the Additional Concerns Anemia subscale. Questions are rated on a scale from 0 to 4, where higher scores indicate more impact on quality of life. * Physical Well-being consists of 7 questions, the subscale score ranges from 0-28; * Social/Family Well-being consists of 7 questions, the subscale score ranges from 0-28; * Emotion Well-being consists of 6 questions, the subscale score ranges from 0-24; * Functional Well-Being consists of 7 questions, the subscale score ranges from 0-28; * Anemia subscale consists of 20 questions, the subscale score ranges from 0-80; * Total FACT-An score ranges from 0-188. |
| Secondary | Change From Baseline in Hemoglobin Concentration for Responders | Baseline, Cycle 6 (168 days) | Change from Baseline in hemoglobin for participants with a clinical response within the first 6 cycles of treatment. |
| Secondary | Change From Baseline in Hemoglobin Concentration for Non-Responders | Baseline, Cycle 6 (168 days) | Change from Baseline in hemoglobin for participants without a clinical response within the first 6 cycles of treatment. |
| Secondary | Change From Baseline in Likert Abdominal Pain Scale | Baseline and Cycle 6 (168 days) | Participants rated abdominal discomfort or pain over the previous week on a scale from zero to ten, where zero is no discomfort or pain and ten is the worst pain imaginable. |
| Secondary | Percentage of Participants With Clinical Response by Baseline JAK2 Assessment | Up to 336 days | Percentage of participants who achieved a clinical response, presented by participants with positive and negative janus kinase 2 (JAK2) V617F mutation results at Baseline. |
| Secondary | Number of Participants With Adverse Events (AEs) | From date of the first dose of the study drug until discontinuation or the data cut-off date (up to approximately 45 months). | A serious AE (SAE) was defined as any AE which resulted in death or was life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or constituted an important medical event (events that may have jeopardized the patient or required intervention to prevent one of the outcomes listed above). The severity of AEs were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 3.0) or according to the following scale: Grade 1 = Mild; Grade 2 = Moderate; Grade 3 = Severe; Grade 4 = Life-threatening; Grade 5 = Death. The Investigator determined the relationship between study drug and the occurrence of an AE as "Not Related" or "Related" (since the study was double-blinded, a patient receiving only prednisone could have an AE that was judged as related to pomalidomide, and vice-versa). |
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 With a Clinical Response Within the First 6 Cycles of Treatment | Up to 168 days | Modified intent-to-treat (MITT), defined as the patients who had a confirmed diagnosis of Myelofibrosis with myeloid metaplasia (MMM), received at least one dose of study drug, and participated in the study for at least 56 days. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment | Up to 336 days | Intent-to-treat (ITT), defined as as all patients who were randomized, independent of whether they received study treatment or not. | percentage of participants | NUMBER |
| SECONDARY | Time to the First Clinical Response | Up to 168 days | Intent-to-treat population with a clinical response | weeks | MEDIAN |
| SECONDARY | Duration of First Clinical Response | Up to 40 months | Intent-to-treat population with a clinical response. | months | MEDIAN |
| SECONDARY | Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Subscale and Total Scores | Baseline and Cycle 6 (168 days). | Intent-to-treat patients with available data. | units on a scale | MEAN |
| SECONDARY | Change From Baseline in Hemoglobin Concentration for Responders | Baseline, Cycle 6 (168 days) | Intent-to-treat participants with a clinical response and available hemoglobin values at each time point. | g/dL | MEDIAN |
| SECONDARY | Change From Baseline in Hemoglobin Concentration for Non-Responders | Baseline, Cycle 6 (168 days) | Intent-to-treat participants with no clinical response and available hemoglobin values at each time point. | g/dL | MEDIAN |
| SECONDARY | Change From Baseline in Likert Abdominal Pain Scale | Baseline and Cycle 6 (168 days) | Intent-to-treat patients with available data. | units on a scale | MEAN |
| SECONDARY | Percentage of Participants With Clinical Response by Baseline JAK2 Assessment | Up to 336 days | Intent-to-treat population with non-missing JAK2 Baseline assessment results. The number of participants analyzed indicates the number of participants with a positive or negative JAK2 result for each treatment group respectively. | percentage of participants | NUMBER |
| SECONDARY | Number of Participants With Adverse Events (AEs) | From date of the first dose of the study drug until discontinuation or the data cut-off date (up to approximately 45 months). | Safety population (all treated patients). | participants | NUMBER |
Source ClinicalTrials.gov
Results by group115
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment | Prednisone | 55.0 | 95% Confidence Interval 31.53 to 76.94 | percentage of participants | |
| Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment | Pomalidomide 2 mg | 23.5 | 95% Confidence Interval 6.81 to 49.90 | percentage of participants | |
| Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment | Pomalidomide 2 mg + Prednisone | 21.1 | 95% Confidence Interval 6.05 to 45.57 | percentage of participants | |
| Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment | Pomalidomide 0.5 mg + Prednisone | 47.6 | 95% Confidence Interval 25.71 to 70.22 | percentage of participants | |
| Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment | Prednisone | 50.0 | 95% Confidence Interval 28.22 to 71.78 | percentage of participants | |
| Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment | Pomalidomide 2 mg | 18.2 | 95% Confidence Interval 5.19 to 40.28 | percentage of participants | |
| Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment | Pomalidomide 2 mg + Prednisone | 18.2 | 95% Confidence Interval 5.19 to 40.28 | percentage of participants | |
| Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment | Pomalidomide 0.5 mg + Prednisone | 45.5 | 95% Confidence Interval 24.39 to 67.79 | percentage of participants | |
| Time to the First Clinical Response | Prednisone | 0.3 | Full Range 0.1 to 15.6 | weeks | |
| Time to the First Clinical Response | Pomalidomide 2 mg | 8.0 | Full Range 2.6 to 17.3 | weeks |
1 to 10 of 115Source ClinicalTrials.gov
See all 115 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Prednisone | 22 | |
| Overall Study | STARTED | Pomalidomide 2 mg | 22 | |
| Overall Study | STARTED | Pomalidomide 2 mg + Prednisone | 22 | |
| Overall Study | STARTED | Pomalidomide 0.5 mg + Prednisone | 22 | |
| Overall Study | Treated | Prednisone | 22 | |
| Overall Study | Treated | Pomalidomide 2 mg | 22 | |
| Overall Study | Treated | Pomalidomide 2 mg + Prednisone | 19 | |
| Overall Study | Treated | Pomalidomide 0.5 mg + Prednisone | 22 | |
| Overall Study | COMPLETED | Prednisone | 0 | |
| Overall Study | COMPLETED | Pomalidomide 2 mg | 0 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events720
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 | Fatigue | General disorders | Prednisone | 6 | 22 | |
| other | Fatigue | General disorders | Pomalidomide 2 mg | 2 | 22 | |
| other | Fatigue | General disorders | Pomalidomide 2 mg + Prednisone | 6 | 19 | |
| other | Fatigue | General disorders | Pomalidomide 0.5 mg + Prednisone | 7 | 22 | |
| other | Oedema Peripheral | General disorders | Prednisone | 6 | 22 | |
| other | Oedema Peripheral | General disorders | Pomalidomide 2 mg | 8 | 22 | |
| other | Oedema Peripheral | General disorders | Pomalidomide 2 mg + Prednisone | 10 | 19 | |
| other | Oedema Peripheral | General disorders | Pomalidomide 0.5 mg + Prednisone | 10 | 22 | |
| other | Pyrexia | General disorders | Prednisone | 5 | 22 | |
| other | Pyrexia | General disorders | Pomalidomide 2 mg | 7 | 22 |
1 to 10 of 720Source ClinicalTrials.gov
See all 720 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Prednisone | ||
| Total, all-cause mortality | Pomalidomide 2 mg | ||
| Total, all-cause mortality | Pomalidomide 2 mg + Prednisone | ||
| Total, all-cause mortality | Pomalidomide 0.5 mg + Prednisone | ||
| Total, other adverse events | Prednisone | 20 | 22 |
| Total, other adverse events | Pomalidomide 2 mg | 20 | 22 |
| Total, other adverse events | Pomalidomide 2 mg + Prednisone | 17 | 19 |
| Total, other adverse events | Pomalidomide 0.5 mg + Prednisone | 21 | 22 |
| Total, serious adverse events | Prednisone | 6 | 22 |
| Total, serious adverse events | Pomalidomide 2 mg | 10 | 22 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT00463385
GET https://conductscience.org/api/trials/v1/NCT00463385/results?page=2