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| ID | Type | Description | Link |
|---|---|---|---|
| 2010-018965-42 | EudraCT Number |
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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)
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Who Achieved RBC-Transfusion Independence | RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval. | 168 days |
| China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days | 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. | From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival | 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. | 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. |
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Inclusion Criteria:
Age ≥ 18 years
Myeloproliferative-neoplasm (MPN)-associated myelofibrosis
RBC-transfusion-dependence (global study):
determining eligibility.
RBC-transfusions due to bleeding are not scored in determining eligibility.
RBC-transfusions due to chemotherapy-induced anemia are not scored in determining eligibility.
≥ 2 hemoglobin concentrations ≤ 80 g/L for ≥ 84 days immediately before the day of enrollment.
No RBC-transfusion within 6 months prior to enrollment.
Exclusion Criteria:
Prior blood cell or bone marrow allotransplant.
Use of drugs to treat MPN-associated myelofibrosis ≤ 30 days before starting study drug.
Treatment with erythropoietin or androgenic steroids ≤ 84 days before starting study drug.
Anemia due to reasons other than MPN-associated myelofibrosis.
Pregnant or lactating females.
More than 10% blasts by bone marrow examination or more than 10% blasts in blood in consecutive measurements spanning at least 8 weeks
Prior history of malignancies,other than the disease being studied, unless the subject has been free of the malignancy for ≥ 5 years with the following exceptions:
Human immunodeficiency virus (HIV) infection, active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections.
Prior treatment with pomalidomide.
Allergic reaction or rash after treatment with thalidomide or lenalidomide
Any of the following laboratory abnormalities:
Uncontrolled hyperthyroidism or hypothyroidism.
Deep venous thrombosis (DVT) or pulmonary embolus (PE) < 6 months before starting study drug
Clinically-important heart disease within the past 6 months
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| Name | Affiliation | Role |
|---|---|---|
| Robert Peter P Gale, MD, Ph.D. | Celgene Corporation | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mayo Clinic | Scottsdale | Arizona | 85259 | United States | ||
| UCLA School of Medicine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27773929 | Derived | Tefferi A, Al-Ali HK, Barosi G, Devos T, Gisslinger H, Jiang Q, Kiladjian JJ, Mesa R, Passamonti F, McMullin MF, Ribrag V, Schiller G, Vannucchi AM, Zhou D, Reiser D, Zhong J, Gale RP. A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. Leukemia. 2017 Apr;31(4):896-902. doi: 10.1038/leu.2016.300. Epub 2016 Oct 24. | |
| 22081489 |
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Participants in the global study were randomized 2:1 to receive blinded pomalidomide or placebo. All participants in the China extension received open-label pomalidomide. Randomization was stratified by age (≤ vs >65 years), white blood cells (< or ≥25 × 10⁹/L) and baseline transfusion requirement (≤ vs >4 units RBC/28 days over the prior 84 days).
Participants in the global study were enrolled at 72 clinical centers in 15 countries. In addition, the China-specific extension study enrolled participants with myeloproliferative neoplasm (MPN)-associated myelofibrosis and severe anemia not receiving red blood cell (RBC)-transfusions at 5 sites in China.
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| ID | Title | Description |
|---|---|---|
| FG000 | Pomalidomide 0.5 mg | 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. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Placebo | Drug | Placebo Comparator to active drug; Placebo capsule taken by mouth once daily |
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| Pomalidomide | Drug | Pomalidomide 0.5 mg capsule taken by mouth once daily. |
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| Duration of RBC-Transfusion Independence | 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. | 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. |
| Time to RBC-Transfusion Independence | 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. | 168 days |
| Number of Participants With Treatment-emergent Adverse Events (TEAE) | 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 | 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. |
| 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. |
| Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score | 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. | Baseline and Days 85 and 169 |
| Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale | 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). | Baseline and Days 85 and 169 |
| Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score | 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. | Baseline and Days 85 and 169 |
| Los Angeles |
| California |
| 90095 |
| United States |
| University of Florida Shands Cancer Center | Gainesville | Florida | 32610 | United States |
| Mayo Clinic | Jacksonville | Florida | 32224 | United States |
| University of Illinois at Chicago | Chicago | Illinois | 60612 | United States |
| University of Michigan Comprehensive Cancer Center | Ann Arbor | Michigan | 48109 | United States |
| Mayo Clinic | Rochester | Minnesota | 55905 | United States |
| Mount Sinai School of Medicine Brookdale University Hospital | Brooklyn | New York | 11212 | United States |
| Weill Medical College of Cornell University | New York | New York | 10021 | United States |
| Ruttenberg Treatment Center | New York | New York | 10029 | United States |
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | United States |
| Medicine Taussig Cancer Institute | Cleveland | Ohio | 44195 | United States |
| Thomas Jefferson University | Philadelphia | Pennsylvania | 19107 | United States |
| Avera Hematology and Transplant | Sioux Falls | South Dakota | 57105 | United States |
| MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| Fred Hutchinson Cancer Center | Seattle | Washington | 98109 | United States |
| Gosford Hospital | Gosford | New South Wales | 2250 | Australia |
| Royal North Shore Hospital | St Leonards | New South Wales | 2065 | Australia |
| Frankston Hospital | Frankston | Victoria | 3199 | Australia |
| Royal Melbourne Hospital | Parkville | Victoria | 3050 | Australia |
| Medizinische Universitatklinik Graz | Graz | A-8036 | Austria |
| Medizinische Universitat Innsbruck | Innsbruck | A-6020 | Austria |
| Medizinische Universitat Wien | Vienna | A-1190 | Austria |
| Algemeen Ziekenhuis Sint-Jan | Bruges | 8000 | Belgium |
| Grand Hopital de Charleroi | Charleroi | 6000 | Belgium |
| Universitaire Ziekenhuis Leuven Gathuisberg | Leuven | 3000 | Belgium |
| Cross Cancer Institute | Edmonton | Alberta | T6G lz2 | Canada |
| Vancouver General Hospital | Vancouver | British Columbia | V5Z 1M9 | Canada |
| Princess Margaret Hospital | Toronto | Ontario | M5G 2M9 | Canada |
| Centre Hospitalier de L'Universite de Montreal | Montreal | Canada |
| Peking University People's Hospital | Beijing | 100044 | China |
| Peking Union Medical College Hospital | Beijing | 100730 | China |
| Jiangsu Province Hospital | Jiangsu | 210029 | China |
| Shanghai Ruijin Hospital | Shanghai | 200025 | China |
| West China Hospital, Sichuan University | Sichuan | 610041 | China |
| Blood Disease Hospital Chinese Academy of Medical Sciences | Tianjin | 300041 | China |
| Hopital Albert Michallon | La Tronche | 38043 | France |
| Hopital Saint Vincent de Paul | Lille | 59020 | France |
| CHU Dupuytren | Limoges | 87042 | France |
| Hopital Saint-Louis | Paris | 75475 | France |
| CHRU - Hopital du Haut Leveque | Pessac | 33604 | France |
| Hopitaux Universitaires de Strasbourg, CHU Haute-Pierre | Strasbourg | 67098 | France |
| Hopital Purpan | Toulouse | 31059 | France |
| Institut Gustave Roussy | Villejuif | 94805 | France |
| Universitatsklinikum Aachen | Aachen | 52074 | Germany |
| Medizinische Hochschule Hannover | Hanover | 30625 | Germany |
| Universitatsklinikum Leipzig | Leipzig | 04103 | Germany |
| Johannes Wesling Klinikum Minden | Minden | 32429 | Germany |
| Universitatsklinikum Ulm | Ulm | 89081 | Germany |
| Azienda Ospedaliera Universitaria Consorziale Policlinico di Bari | Bari | 70124 | Italy |
| Ospedali Riuniti di Bergamo | Bergamo | 24128 | Italy |
| Azienda Ospedaliera Universitaria Careggi | Florence | 50134 | Italy |
| Azienda Ospedaliera Universitaria Federico II di Napoli | Naples | 80131 | Italy |
| Azienda Ospedaliera San Luigi Gonzaga | Orbassano | 10043 | Italy |
| IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Centro per lo Studio della Mielofibrosi | Pavia | 27100 | Italy |
| IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Ematologia | Pavia | 27100 | Italy |
| Ospedale di Circolo e Fondazione Macchi Varese | Varese | 21100 | Italy |
| Juntendo University Hospital | Bunkyou-ku | 113-8431 | Japan |
| Kyushu University Hospital | Fukuoka | 812-8582 | Japan |
| Tokai University Hospital | Isehara | 259-1193 | Japan |
| Kyoto University Hospital | Kyoto | 606-8507 | Japan |
| Nagasaki University Hospital | Nagasaki | 852-8501 | Japan |
| Tokyo Medical University Hospital | Shinjuku | 160-0023 | Japan |
| VU University Medical Center | Amsterdam | 1081 HV | Netherlands |
| Erasmus Medish Centrum | Rotterdam | 3015 CE | Netherlands |
| University Medical Center Utrecht | Utrecht | 3584 CX | Netherlands |
| Wojewodzki Szpital Specjalistyczny im. F.Chopina | Rzeszów | 35-055 | Poland |
| Samodzielny Publiczny Szpital Kliniczny Nr 1 PAM | Szczecin | 71-242 | Poland |
| Centralny Szpital Kliniczny MSWiA | Warsaw | 02-507 | Poland |
| Russian Scientific Haematology Centre | Moscow | 125167 | Russia |
| Federal State Institution Russian Scientific-research Institute of Hematology and Transfusiology of Federal Medical-Biological Agency of Russia | Saint Petersburg | 191024 | Russia |
| State Pavlov Medical University | Saint Petersburg | 196022 | Russia |
| Federal State Institution "Federal Centre of Heart, Blood and Endocrinology of Rosmedtechnologies named after V.A. Almazov" | Saint Petersburg | 197341 | Russia |
| Hospital Clinic I Provincial de Barcelona | Barcelona | 08036 | Spain |
| Hospital Universitario Puerta De Hierro Majadahonda | Majadahonda | 28222 | Spain |
| Hospital Clinico de Salamanca | Salamanca | 37007 | Spain |
| Hospital Clinico de Valencia | Valencia | 46010 | Spain |
| Skane University Hospital | Lund | 22185 | Sweden |
| Karolinska University Hospital Huddinge | Stockholm | 14185 | Sweden |
| Belfast City Hospital | Belfast | BT9 7AB | United Kingdom |
| Beatson Oncology Centre | Glasgow | G12 0YN | United Kingdom |
| John Radcliffe Hospital NHS Trust | Headington | OX3 9DU | United Kingdom |
| St. Thomas Hospital | London | SE1 9RT | United Kingdom |
| Hammersmith Hospital | London | W12 ONN | United Kingdom |
| Freeman Hospital | Newcastle upon Tyne | NE7 7DN | United Kingdom |
| Royal Hallamshire Hospital | Sheffield | S10 2JF | United Kingdom |
| Derived |
| Begna KH, Pardanani A, Mesa R, Litzow MR, Hogan WJ, Hanson CA, Tefferi A. Long-term outcome of pomalidomide therapy in myelofibrosis. Am J Hematol. 2012 Jan;87(1):66-8. doi: 10.1002/ajh.22233. Epub 2011 Nov 12. |
| FG001 | Placebo | 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. |
| FG002 | China Extension: Pomalidomide 0.5 mg | 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. |
| Received Study Drug |
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| COMPLETED | Completed study, no longer in follow-up |
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| NOT COMPLETED |
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Intent to Treat (ITT) population included all randomized (global study) or enrolled (China extension study) participants
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| ID | Title | Description |
|---|---|---|
| BG000 | Pomalidomide 0.5 mg | 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 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 the definition of clinical benefit was no longer met or other criteria for treatment discontinuation applied. |
| BG001 | Placebo | 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. |
| BG002 | China Extension: Pomalidomide 0.5 mg | 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. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Results of the global study and China extension study were analyzed separately. | Median | Full Range | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Baseline RBC Transfusion Burden | Defined as the average number of RBC-transfusion-units per 28 days over the 84 days immediately prior to randomization. | Data for the global study and China extension study were analyzed separately. | Median | Inter-Quartile Range | units per 28 days |
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| Eastern Cooperative Oncology Group (ECOG) Performance Status | ECOG performance status is used to describe a patient's level of functioning in terms of their ability to care for themselves, daily activity, and physical ability (walking, working, etc.). The scale ranges from 0 to 5: -0 = Fully active, no restrictions; -1 = Restricted activity but ambulatory, able to carry out work of a light nature; -2 = Ambulatory and capable of all self-care but unable to carry out work activities; -3 = Limited self-care, confined to bed or chair more than 50% of waking hours; -4 = Completely disabled, no self-care, confined to bed or chair; -5 = Dead | Count of Participants | Participants |
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| Disease Sub-type | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Percentage of Participants Who Achieved RBC-Transfusion Independence | RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval. | 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. | Posted | Number | 95% Confidence Interval | percentage of participants | 168 days |
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| Primary | China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days | 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. | China extension intent-to-treat population, which includes all participants enrolled in the China extension study. | Posted | Count of Participants | Participants | From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks. |
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| Secondary | Overall Survival | 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. | Global study intent-to-treat population | Posted | Median | 95% Confidence Interval | months | 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. |
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| Secondary | Duration of RBC-Transfusion Independence | 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. | Global study intent-to-treat population with an 84-day RBC-transfusion independence response | Posted | Median | 95% Confidence Interval | months | 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. |
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| Secondary | Time to RBC-Transfusion Independence | 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. | Global study intent-to-treat population with an 84-day RBC-transfusion independence response | Posted | Median | Full Range | weeks | 168 days |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAE) | 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 | Participants who received at least 1 dose of study drug | Posted | Count of Participants | Participants | 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. |
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| Secondary | Healthcare Resource Utilization | Analysis of healthcare resource utilization data were not collected during the study and no analysis were performed. | Posted | 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. |
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| Secondary | Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score | 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. | Global study intent-to-treat population with available data at baseline and each time point | Posted | Mean | Standard Deviation | score on a scale | Baseline and Days 85 and 169 |
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| Secondary | Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale | 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). | Global study intent-to-treat population with available data at baseline and each time point. | Posted | Mean | Standard Deviation | units on a scale | Baseline and Days 85 and 169 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score | 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. | Global study intent-to-treat population with available data at baseline and each time point. | Posted | Mean | Standard Deviation | units on a scale | Baseline and Days 85 and 169 |
|
All-cause mortality data are reported from first dose of study drug up to end of study; maximum follow-up time was 85 months. Adverse events are reported 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.
The safety population includes all participants who received at least one dose of study drug; one participant randomized to pomalidomide and one participant randomized to placebo in the global study did not receive treatment and are excluded from the safety population.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Global Study: Pomalidomide 0.5 mg | 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 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 the definition of clinical benefit was no longer met or other criteria for treatment discontinuation applied. | 115 | 167 | 76 | 167 | 155 | 167 |
| EG001 | Global Study: Placebo | Participants received placebo taken by mouth once daily for at least 168 days unless there were unacceptable side effects or disease progression. Participants who experienced clinical benefit could continue to receive placebo until the definition of clinical benefit was no longer met or other criteria for treatment discontinuation applied. | 54 | 83 | 29 | 83 | 76 | 83 |
| EG002 | China Extension: Pomalidomide 0.5 mg | 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. | 8 | 15 | 0 | 15 | 12 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANAEMIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DISSEMINATED INTRAVASCULAR COAGULATION | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HAEMOLYSIS | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HAEMOLYTIC ANAEMIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| NEUTROPENIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PANCYTOPENIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| SPLENIC EMBOLISM | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| SPLENIC INFARCTION | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| SPLENOMEGALY | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| THROMBOCYTOPENIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ACUTE CORONARY SYNDROME | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ACUTE MYOCARDIAL INFARCTION | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ATRIAL FIBRILLATION | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CARDIAC ARREST | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CARDIAC FAILURE | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CARDIAC FAILURE ACUTE | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CARDIAC FAILURE CONGESTIVE | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CONDUCTION DISORDER | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CORONARY ARTERY DISEASE | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ABDOMINAL DISTENSION | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ASCITES | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ENTEROCOLITIS | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| GASTRIC HAEMORRHAGE | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| GASTROINTESTINAL HAEMORRHAGE | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HAEMORRHOIDAL HAEMORRHAGE | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HAEMORRHOIDS | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| MELAENA | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ASTHENIA | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| FATIGUE | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| GENERALISED OEDEMA | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| INFLUENZA LIKE ILLNESS | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| MULTI-ORGAN FAILURE | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| NON-CARDIAC CHEST PAIN | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| OEDEMA PERIPHERAL | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| SUDDEN CARDIAC DEATH | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| SUDDEN DEATH | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CHOLECYSTITIS | Hepatobiliary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ATYPICAL PNEUMONIA | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| BACTEROIDES BACTERAEMIA | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| BRONCHITIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| BRONCHOPULMONARY ASPERGILLOSIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| CELLULITIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| DEVICE RELATED INFECTION | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| ENTEROCOLITIS INFECTIOUS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| ERYSIPELAS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| GASTROINTESTINAL CANDIDIASIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| INFECTIOUS PLEURAL EFFUSION | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| LUNG INFECTION | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| LYMPHADENITIS BACTERIAL | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| NECROTISING FASCIITIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| PARONYCHIA | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| PNEUMOCYSTIS JIROVECI PNEUMONIA | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| PNEUMONIA | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| SEPSIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| SEPTIC SHOCK | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| SIALOADENITIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| STAPHYLOCOCCAL SEPSIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| UROSEPSIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| WOUND INFECTION | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| INJURY | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| SUBDURAL HAEMATOMA | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| SUBDURAL HAEMORRHAGE | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| TRANSFUSION-RELATED CIRCULATORY OVERLOAD | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| ASPARTATE AMINOTRANSFERASE INCREASED | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| PLATELET COUNT INCREASED | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| WHITE BLOOD CELL COUNT INCREASED | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| DECREASED APPETITE | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DEHYDRATION | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DIABETIC KETOACIDOSIS | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HYPOGLYCAEMIA | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| B-CELL LYMPHOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.1 | Systematic Assessment |
| |
| BASAL CELL CARCINOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.1 | Systematic Assessment |
| |
| HISTIOCYTOSIS HAEMATOPHAGIC | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.1 | Systematic Assessment |
| |
| MALIGNANT MELANOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.1 | Systematic Assessment |
| |
| MYELOFIBROSIS | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.1 | Systematic Assessment |
| |
| OESOPHAGEAL ADENOCARCINOMA | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 15.1 | Systematic Assessment |
| |
| CEREBROVASCULAR ACCIDENT | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CONVULSION | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| LOSS OF CONSCIOUSNESS | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| SYNCOPE | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| VIITH NERVE PARALYSIS | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ABNORMAL BEHAVIOUR | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CALCULUS BLADDER | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CALCULUS URETERIC | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| NEPHROLITHIASIS | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| NEPHROTIC SYNDROME | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| RENAL COLIC | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| RENAL FAILURE | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| RENAL FAILURE ACUTE | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| RENAL FAILURE CHRONIC | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| URINARY RETENTION | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| URINARY TRACT OBSTRUCTION | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| EPISTAXIS | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HYPOXIA | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| INTERSTITIAL LUNG DISEASE | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PNEUMONITIS | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PULMONARY EMBOLISM | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PULMONARY HYPERTENSION | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ACUTE FEBRILE NEUTROPHILIC DERMATOSIS | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DRUG ERUPTION | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| LEUKOCYTOCLASTIC VASCULITIS | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PETECHIAE | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| RASH MACULO-PAPULAR | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| SKIN ULCER | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| AXILLARY VEIN THROMBOSIS | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DEEP VEIN THROMBOSIS | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HYPOTENSION | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| INTERMITTENT CLAUDICATION | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| JUGULAR VEIN THROMBOSIS | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PHLEBITIS | Vascular disorders | MedDRA 15.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANAEMIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| NEUTROPENIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| THROMBOCYTOPENIA | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ARRHYTHMIA | Cardiac disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ABDOMINAL DISTENSION | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ABDOMINAL PAIN UPPER | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ASTHENIA | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CHEST PAIN | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| FATIGUE | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| OEDEMA | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| OEDEMA PERIPHERAL | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HEPATIC CYST | Hepatobiliary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HEPATIC FUNCTION ABNORMAL | Hepatobiliary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| BRONCHITIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| NASOPHARYNGITIS | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| PNEUMONIA | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| LIGAMENT SPRAIN | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| WEIGHT DECREASED | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| WHITE BLOOD CELL COUNT DECREASED | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| WHITE BLOOD CELL COUNT INCREASED | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| DECREASED APPETITE | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HYPERGLYCAEMIA | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| HYPERURICAEMIA | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| ARTHRALGIA | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| MUSCLE SPASMS | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DIZZINESS | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| SOMNOLENCE | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| CALCULUS URETERIC | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| NEPHROLITHIASIS | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| DYSPNOEA EXERTIONAL | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| EPISTAXIS | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PLEURAL EFFUSION | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| NIGHT SWEATS | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| PRURITUS | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| RASH | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
|
Results from a center cannot be submitted for publication before results of multicenter study are published unless it is more than 1 year since study completion. Then Investigator can publish if the manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides a publication would hinder drug development, Investigator must delay submission for up to 90 days. Investigator must delete confidential information before submission or defer publication to permit patent applications.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Anne McClain | Celgene Corporation | 1-888-260-1599 | clinicaltrialdisclosure@celgene.com |
| ID | Term |
|---|---|
| D055728 | Primary Myelofibrosis |
| ID | Term |
|---|---|
| D009196 | Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C467566 | pomalidomide |
Not provided
Not provided
Not provided
|
| China extension study |
|
|
|
|
|
|
| China extension study |
|
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|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
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|
|
| Counts |
|---|
| Participants |
|
|
| OG001 | Placebo | 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. |
| OG002 | China Extension: Pomalidomide 0.5 mg | 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. |
|
|
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|
| Counts |
|---|
| Participants |
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