search
Back to results

Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence (RESUME)

Primary Purpose

Primary Myelofibrosis, MPN-associated Myelofibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Pomalidomide 0.5 mg
Placebo
Pomalidomide
Sponsored by
Celgene
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Myelofibrosis focused on measuring Myelofibrosis, Post-polycythemia vera myelofibrosis, Post-essential thrombocythemia myelofibrosis, RBC-transfusion-dependence, Primary Myelofibrosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 without ≥ 1 RBC-transfusion.
    • Only RBC-transfusions given when the hemoglobin ≤ 90 g/L³ are scored in

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.

    • Severe anemia (China-specific extension):
  • ≥ 2 hemoglobin concentrations ≤ 80 g/L for ≥ 84 days immediately before the day of enrollment.

  • No RBC-transfusion within 6 months prior to enrollment.

    • Hemoglobin ≤ 130 g/L at randomization (global study); ≤ 80 g/L at enrollment in the China-specific extension.
    • Bone marrow biopsy within 6 months (global study only).
    • Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin and androgenic steroids
    • Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
    • Agree to follow pregnancy precautions as required by the protocol.
    • Agree to receive counseling related to teratogenic and other risks of pomalidomide.
    • Agree not to donate blood or semen.

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:

    • Carcinoma in situ of the cervix
    • Carcinoma in situ of the breast
    • Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor, nodes, metastasis] clinical staging system)
  • 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:

    • Neutrophils < 0.5x10^9 /L
    • Platelets < 25 x 10^9 /L
    • Estimated glomerular filtration rate (kidney function) < 30 mL/min/1.73 m²
    • Aspartate aminotransferase (AST) and alanine transaminase (ALT) > 3.0 x upper limit of normal (ULN)
    • Total bilirubin ≥ 4 x ULN;
  • 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

Sites / Locations

  • Mayo Clinic
  • UCLA School of Medicine
  • University of Florida Shands Cancer Center
  • Mayo Clinic
  • University of Illinois at Chicago
  • University of Michigan Comprehensive Cancer Center
  • Mayo Clinic
  • Mount Sinai School of Medicine Brookdale University Hospital
  • Weill Medical College of Cornell University
  • Ruttenberg Treatment Center
  • Memorial Sloan Kettering Cancer Center
  • Medicine Taussig Cancer Institute
  • Thomas Jefferson University
  • Avera Hematology and Transplant
  • MD Anderson Cancer Center
  • University of Utah
  • Fred Hutchinson Cancer Center
  • Gosford Hospital
  • Royal North Shore Hospital
  • Frankston Hospital
  • Royal Melbourne Hospital
  • Medizinische Universitatklinik Graz
  • Medizinische Universitat Innsbruck
  • Medizinische Universitat Wien
  • Algemeen Ziekenhuis Sint-Jan
  • Grand Hopital de Charleroi
  • Universitaire Ziekenhuis Leuven Gathuisberg
  • Cross Cancer Institute
  • Vancouver General Hospital
  • Princess Margaret Hospital
  • Centre Hospitalier de L'Universite de Montreal
  • Peking University People's Hospital
  • Peking Union Medical College Hospital
  • Jiangsu Province Hospital
  • Shanghai Ruijin Hospital
  • West China Hospital, Sichuan University
  • Blood Disease Hospital Chinese Academy of Medical Sciences
  • Hopital Albert Michallon
  • Hopital Saint Vincent de Paul
  • CHU Dupuytren
  • Hopital Saint-Louis
  • CHRU - Hopital du Haut Leveque
  • Hopitaux Universitaires de Strasbourg, CHU Haute-Pierre
  • Hopital Purpan
  • Institut Gustave Roussy
  • Universitatsklinikum Aachen
  • Medizinische Hochschule Hannover
  • Universitatsklinikum Leipzig
  • Johannes Wesling Klinikum Minden
  • Universitatsklinikum Ulm
  • Azienda Ospedaliera Universitaria Consorziale Policlinico di Bari
  • Ospedali Riuniti di Bergamo
  • Azienda Ospedaliera Universitaria Careggi
  • Azienda Ospedaliera Universitaria Federico II di Napoli
  • Azienda Ospedaliera San Luigi Gonzaga
  • IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Centro per lo Studio della Mielofibrosi
  • IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Ematologia
  • Ospedale di Circolo e Fondazione Macchi Varese
  • Juntendo University Hospital
  • Kyushu University Hospital
  • Tokai University Hospital
  • Kyoto University Hospital
  • Nagasaki University Hospital
  • Tokyo Medical University Hospital
  • VU University Medical Center
  • Erasmus Medish Centrum
  • University Medical Center Utrecht
  • Wojewodzki Szpital Specjalistyczny im. F.Chopina
  • Samodzielny Publiczny Szpital Kliniczny Nr 1 PAM
  • Centralny Szpital Kliniczny MSWiA
  • Russian Scientific Haematology Centre
  • Federal State Institution Russian Scientific-research Institute of Hematology and Transfusiology of Federal Medical-Biological Agency of Russia
  • State Pavlov Medical University
  • Federal State Institution "Federal Centre of Heart, Blood and Endocrinology of Rosmedtechnologies named after V.A. Almazov"
  • Hospital Clinic I Provincial de Barcelona
  • Hospital Universitario Puerta De Hierro Majadahonda
  • Hospital Clinico de Salamanca
  • Hospital Clinico de Valencia
  • Skane University Hospital
  • Karolinska University Hospital Huddinge
  • Belfast City Hospital
  • Beatson Oncology Centre
  • John Radcliffe Hospital NHS Trust
  • St. Thomas Hospital
  • Hammersmith Hospital
  • Freeman Hospital
  • Royal Hallamshire Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Experimental

Arm Label

Pomalidomide 0.5 mg

Placebo

China Extension: Pomalidomide 0.5 mg

Arm Description

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.

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.

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.

Outcomes

Primary Outcome Measures

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.
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.

Secondary Outcome Measures

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.
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.
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.
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
Healthcare Resource Utilization
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.
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).
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.

Full Information

First Posted
July 15, 2010
Last Updated
June 24, 2019
Sponsor
Celgene
search

1. Study Identification

Unique Protocol Identification Number
NCT01178281
Brief Title
Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence
Acronym
RESUME
Official Title
A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Red Blood Cell-Transfusion-Dependence
Study Type
Interventional

2. Study Status

Record Verification Date
June 2019
Overall Recruitment Status
Completed
Study Start Date
September 8, 2010 (Actual)
Primary Completion Date
January 1, 2013 (Actual)
Study Completion Date
May 15, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Celgene

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Myelofibrosis, MPN-associated Myelofibrosis
Keywords
Myelofibrosis, Post-polycythemia vera myelofibrosis, Post-essential thrombocythemia myelofibrosis, RBC-transfusion-dependence, Primary Myelofibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
267 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Pomalidomide 0.5 mg
Arm Type
Experimental
Arm Description
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 Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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 Title
China Extension: Pomalidomide 0.5 mg
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Pomalidomide 0.5 mg
Other Intervention Name(s)
CC-4047; Pomalyst; Imnovid
Intervention Description
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 Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo Comparator to active drug; Placebo capsule taken by mouth once daily
Intervention Type
Drug
Intervention Name(s)
Pomalidomide
Other Intervention Name(s)
CC-4047, Pomalyst, Imnovid
Intervention Description
Pomalidomide 0.5 mg capsule taken by mouth once daily.
Primary Outcome Measure Information:
Title
Percentage of Participants Who Achieved RBC-Transfusion Independence
Description
RBC-transfusion independence was defined as the absence of RBC transfusions for any consecutive 84-day interval.
Time Frame
168 days
Title
China Extension: Number of Participants Achieving a Hemoglobin Increase of ≥ 15 g/L Compared to Baseline for ≥ 84 Consecutive Days
Description
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.
Time Frame
From the first dose of study drug until treatment discontinuation; median treatment duration was 24.0 weeks.
Secondary Outcome Measure Information:
Title
Overall Survival
Description
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.
Time Frame
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.
Title
Duration of RBC-Transfusion Independence
Description
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.
Time Frame
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.
Title
Time to RBC-Transfusion Independence
Description
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.
Time Frame
168 days
Title
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Description
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
Time Frame
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.
Title
Healthcare Resource Utilization
Time Frame
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.
Title
Change From Baseline in EuroQoL-5D (EQ-5D) Health Index Score
Description
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.
Time Frame
Baseline and Days 85 and 169
Title
Change From Baseline in EuroQoL-5D (EQ-5D) Visual Analog Scale
Description
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).
Time Frame
Baseline and Days 85 and 169
Title
Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Total Score
Description
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.
Time Frame
Baseline and Days 85 and 169

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 without ≥ 1 RBC-transfusion. Only RBC-transfusions given when the hemoglobin ≤ 90 g/L³ are scored in 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. Severe anemia (China-specific extension): ≥ 2 hemoglobin concentrations ≤ 80 g/L for ≥ 84 days immediately before the day of enrollment. No RBC-transfusion within 6 months prior to enrollment. Hemoglobin ≤ 130 g/L at randomization (global study); ≤ 80 g/L at enrollment in the China-specific extension. Bone marrow biopsy within 6 months (global study only). Inappropriate to receive blood cell or bone marrow allotransplant, erythropoietin and androgenic steroids Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2. Agree to follow pregnancy precautions as required by the protocol. Agree to receive counseling related to teratogenic and other risks of pomalidomide. Agree not to donate blood or semen. 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: Carcinoma in situ of the cervix Carcinoma in situ of the breast Incidental histologic finding of prostate cancer (T 1a or T 1b using TNM [tumor, nodes, metastasis] clinical staging system) 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: Neutrophils < 0.5x10^9 /L Platelets < 25 x 10^9 /L Estimated glomerular filtration rate (kidney function) < 30 mL/min/1.73 m² Aspartate aminotransferase (AST) and alanine transaminase (ALT) > 3.0 x upper limit of normal (ULN) Total bilirubin ≥ 4 x ULN; 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert Peter P Gale, MD, Ph.D.
Organizational Affiliation
Celgene Corporation
Official's Role
Study Director
Facility Information:
Facility Name
Mayo Clinic
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
UCLA School of Medicine
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
University of Florida Shands Cancer Center
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Mayo Clinic
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
University of Illinois at Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
University of Michigan Comprehensive Cancer Center
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Mount Sinai School of Medicine Brookdale University Hospital
City
Brooklyn
State/Province
New York
ZIP/Postal Code
11212
Country
United States
Facility Name
Weill Medical College of Cornell University
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Ruttenberg Treatment Center
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Medicine Taussig Cancer Institute
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
Avera Hematology and Transplant
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57105
Country
United States
Facility Name
MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Fred Hutchinson Cancer Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109
Country
United States
Facility Name
Gosford Hospital
City
Gosford
State/Province
New South Wales
ZIP/Postal Code
2250
Country
Australia
Facility Name
Royal North Shore Hospital
City
St. Leonards
State/Province
New South Wales
ZIP/Postal Code
2065
Country
Australia
Facility Name
Frankston Hospital
City
Frankston
State/Province
Victoria
ZIP/Postal Code
3199
Country
Australia
Facility Name
Royal Melbourne Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
Medizinische Universitatklinik Graz
City
Graz
ZIP/Postal Code
A-8036
Country
Austria
Facility Name
Medizinische Universitat Innsbruck
City
Innsbruck
ZIP/Postal Code
A-6020
Country
Austria
Facility Name
Medizinische Universitat Wien
City
Vienna
ZIP/Postal Code
A-1190
Country
Austria
Facility Name
Algemeen Ziekenhuis Sint-Jan
City
Brugge
ZIP/Postal Code
8000
Country
Belgium
Facility Name
Grand Hopital de Charleroi
City
Charleroi
ZIP/Postal Code
6000
Country
Belgium
Facility Name
Universitaire Ziekenhuis Leuven Gathuisberg
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Cross Cancer Institute
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G lz2
Country
Canada
Facility Name
Vancouver General Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V5Z 1M9
Country
Canada
Facility Name
Princess Margaret Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2M9
Country
Canada
Facility Name
Centre Hospitalier de L'Universite de Montreal
City
Montreal,
Country
Canada
Facility Name
Peking University People's Hospital
City
Beijing
ZIP/Postal Code
100044
Country
China
Facility Name
Peking Union Medical College Hospital
City
Beijing
ZIP/Postal Code
100730
Country
China
Facility Name
Jiangsu Province Hospital
City
Jiangsu
ZIP/Postal Code
210029
Country
China
Facility Name
Shanghai Ruijin Hospital
City
Shanghai
ZIP/Postal Code
200025
Country
China
Facility Name
West China Hospital, Sichuan University
City
Sichuan
ZIP/Postal Code
610041
Country
China
Facility Name
Blood Disease Hospital Chinese Academy of Medical Sciences
City
Tianjin
ZIP/Postal Code
300041
Country
China
Facility Name
Hopital Albert Michallon
City
La Tronche
ZIP/Postal Code
38043
Country
France
Facility Name
Hopital Saint Vincent de Paul
City
Lille
ZIP/Postal Code
59020
Country
France
Facility Name
CHU Dupuytren
City
Limoges
ZIP/Postal Code
87042
Country
France
Facility Name
Hopital Saint-Louis
City
Paris
ZIP/Postal Code
75475
Country
France
Facility Name
CHRU - Hopital du Haut Leveque
City
Pessac
ZIP/Postal Code
33604
Country
France
Facility Name
Hopitaux Universitaires de Strasbourg, CHU Haute-Pierre
City
Strasbourg
ZIP/Postal Code
67098
Country
France
Facility Name
Hopital Purpan
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
Institut Gustave Roussy
City
Villejuif
ZIP/Postal Code
94805
Country
France
Facility Name
Universitatsklinikum Aachen
City
Aachen
ZIP/Postal Code
52074
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
ZIP/Postal Code
30625
Country
Germany
Facility Name
Universitatsklinikum Leipzig
City
Leipzig
ZIP/Postal Code
04103
Country
Germany
Facility Name
Johannes Wesling Klinikum Minden
City
Minden
ZIP/Postal Code
32429
Country
Germany
Facility Name
Universitatsklinikum Ulm
City
Ulm
ZIP/Postal Code
89081
Country
Germany
Facility Name
Azienda Ospedaliera Universitaria Consorziale Policlinico di Bari
City
Bari
ZIP/Postal Code
70124
Country
Italy
Facility Name
Ospedali Riuniti di Bergamo
City
Bergamo
ZIP/Postal Code
24128
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Careggi
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Federico II di Napoli
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Azienda Ospedaliera San Luigi Gonzaga
City
Orbassano
ZIP/Postal Code
10043
Country
Italy
Facility Name
IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Centro per lo Studio della Mielofibrosi
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
IRCCS Fondazione Policlinico San Matteo, Universita di Pavia, Ematologia
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
Ospedale di Circolo e Fondazione Macchi Varese
City
Varese
ZIP/Postal Code
21100
Country
Italy
Facility Name
Juntendo University Hospital
City
Bunkyou-ku
ZIP/Postal Code
113-8431
Country
Japan
Facility Name
Kyushu University Hospital
City
Fukuoka City
ZIP/Postal Code
812-8582
Country
Japan
Facility Name
Tokai University Hospital
City
Isehara City
ZIP/Postal Code
259-1193
Country
Japan
Facility Name
Kyoto University Hospital
City
Kyoto City
ZIP/Postal Code
606-8507
Country
Japan
Facility Name
Nagasaki University Hospital
City
Nagasaki City
ZIP/Postal Code
852-8501
Country
Japan
Facility Name
Tokyo Medical University Hospital
City
Shinjuku
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
VU University Medical Center
City
Amsterdam
ZIP/Postal Code
1081 HV
Country
Netherlands
Facility Name
Erasmus Medish Centrum
City
Rotterdam
ZIP/Postal Code
3015 CE
Country
Netherlands
Facility Name
University Medical Center Utrecht
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Facility Name
Wojewodzki Szpital Specjalistyczny im. F.Chopina
City
Rzeszow
ZIP/Postal Code
35-055
Country
Poland
Facility Name
Samodzielny Publiczny Szpital Kliniczny Nr 1 PAM
City
Szczecin
ZIP/Postal Code
71-242
Country
Poland
Facility Name
Centralny Szpital Kliniczny MSWiA
City
Warsaw
ZIP/Postal Code
02-507
Country
Poland
Facility Name
Russian Scientific Haematology Centre
City
Moscow
ZIP/Postal Code
125167
Country
Russian Federation
Facility Name
Federal State Institution Russian Scientific-research Institute of Hematology and Transfusiology of Federal Medical-Biological Agency of Russia
City
Saint-Petersburg
ZIP/Postal Code
191024
Country
Russian Federation
Facility Name
State Pavlov Medical University
City
Saint-Petersburg
ZIP/Postal Code
196022
Country
Russian Federation
Facility Name
Federal State Institution "Federal Centre of Heart, Blood and Endocrinology of Rosmedtechnologies named after V.A. Almazov"
City
Saint-Petersburg
ZIP/Postal Code
197341
Country
Russian Federation
Facility Name
Hospital Clinic I Provincial de Barcelona
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Hospital Universitario Puerta De Hierro Majadahonda
City
Majadahonda
ZIP/Postal Code
28222
Country
Spain
Facility Name
Hospital Clinico de Salamanca
City
Salamanca
ZIP/Postal Code
37007
Country
Spain
Facility Name
Hospital Clinico de Valencia
City
Valencia
ZIP/Postal Code
46010
Country
Spain
Facility Name
Skane University Hospital
City
Lund
ZIP/Postal Code
22185
Country
Sweden
Facility Name
Karolinska University Hospital Huddinge
City
Stockholm
ZIP/Postal Code
14185
Country
Sweden
Facility Name
Belfast City Hospital
City
Belfast
ZIP/Postal Code
BT9 7AB
Country
United Kingdom
Facility Name
Beatson Oncology Centre
City
Glasgow
ZIP/Postal Code
G12 0YN
Country
United Kingdom
Facility Name
John Radcliffe Hospital NHS Trust
City
Headington
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Facility Name
St. Thomas Hospital
City
London
ZIP/Postal Code
SE1 9RT
Country
United Kingdom
Facility Name
Hammersmith Hospital
City
London
ZIP/Postal Code
W12 ONN
Country
United Kingdom
Facility Name
Freeman Hospital
City
Newcastle upon Tyne
ZIP/Postal Code
NE7 7DN
Country
United Kingdom
Facility Name
Royal Hallamshire Hospital
City
Sheffield
ZIP/Postal Code
S10 2JF
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
27773929
Citation
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. Erratum In: Leukemia. 2017 May;31(5):1252.
Results Reference
derived
PubMed Identifier
22081489
Citation
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.
Results Reference
derived

Learn more about this trial

Study of Pomalidomide in Persons With Myeloproliferative-Neoplasm-Associated Myelofibrosis and RBC-Transfusion-Dependence

We'll reach out to this number within 24 hrs