Back to resultsA Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia
Primary Purpose
Myelofibrosis With Myeloid Metaplasia, Myeloid Metaplasia, Myelofibrosis
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Pomalidomide
Prednisone
Placebo to pomalidomide
Placebo to prednisone
Sponsored by
About this trial
This is an interventional treatment trial for Myelofibrosis With Myeloid Metaplasia focused on measuring Celgene, CC-4047, Myelofibrosis, myelofibrosis with myeloid metaplasia, myeloid metaplasia, JAK2, CC-4047-MMM-001, Prednisone, Phase II, pomalidomide, bone marrow histology, imids, MMM, Ashkenazi Jewish Population, exposure to Thorotrast, exposure to solvents (benzene and toluene), acute megakaryocytic leukemia, history of polycythemia vera
Eligibility Criteria
Inclusion Criteria:
- Must sign an informed consent form
- Must be >18 years of age
- Must be diagnosed with myelofibrosis
- Eligibility is based on local pathology review of bone marrow aspirate and biopsy
- Screening total hemoglobin level < 10g/dL or transfusion-dependent anemia defined as per International Working Group (IWG) criteria.
Must have adequate organ function as demonstrated by the following ≤ 14 days prior to starting study drug:
- Alanine aminotransferase (ALT; SGPT)/aspartate aminotransferase (AST; SGOT) ≤ 3 x upper limit of normal (ULN), [unless upon judgment of the treating physician, it is believed to be due to extra-medullary hematopoiesis (EMH)].
- Total Bilirubin <3x ULN or Direct Bilirubin <2 x ULN
- Serum creatinine ≤ 2.0 mg/dL
- Absolute neutrophil count ≥ 1,000/μL (≥ 1 x 10^9/L).
- Platelet count ≥ 50,000 /μL (≥ 50 x 10^9/L).
- Patients must be willing to receive transfusion of blood products
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 at screening.
- Must be able to adhere to the study visit schedule and other protocol requirements.
- No active malignancies with the exception of controlled prostate cancer, basal cell or squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast.
- Must agree to follow pregnancy precautions as required per the protocol
Exclusion Criteria:
- Known positive status for human immunodeficiency virus (HIV), hepatitis B carrier, or active hepatitis C infection.
- Previous untoward reaction to corticosteroid (specifically, prednisone) therapy that was severe enough, in the opinion of the treating physician, to preclude study participation.
- The use of any growth factors, cytotoxic chemotherapeutic agents (e.g. hydroxyurea and anagrelide), corticosteroids, or experimental drug or therapy within a minimum of 28 days of starting CC-4047 and/or lack of recovery from all toxicity from previous therapy to grade 1 or better (e.g. alpha interferon may require 84 days of longer or washout).
- Prior therapy with CC-4047 or, lenalidomide or thalidomide for Myelofibrosis with myeloid metaplasia (MMM). (Prior prednisone use as a therapy for MMM is allowed, but not within 28 days of starting CC-4047).
- History of deep vein thrombosis or pulmonary embolism within one year of starting study medication.
- Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
- Pregnant or lactating females
Sites / Locations
- UCLA School of Medicine Hematology/Oncology
- Mayo Clinic
- Memorial Sloan-Kettering Cancer Center
- New York Presbyterian HospitalWeill Medical College of Cornell University
- MD Anderson Cancer Center Leukemia Department
- Fred Hutchinson Cancer Research Center
- Medical University of Vienna, Department of Internal Medicine, Hematology
- Fondazione IRCCS Policlinico San Matteo
- IRCCS Policlinico S. Matteo
- Hematology DepartmentHospital Clinic
- Royal Hallamshire Hospital Sheffield Teaching Hospitals NHS Trust
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Experimental
Arm Label
Prednisone
Pomalidomide
Pomalidomide 2 mg + Prednisone
Pomalidomide 0.5 mg + Prednisone
Arm Description
Participants received oral prednisone from Day 1-28 of each 28-day cycle for up to 3 cycles (84 days), 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day, and pomalidomide placebo tablets on Days 1-28 for up to 12 cycles in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants were discontinued from the study.
Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and prednisone placebo tablets on Days 1-28 for the first 3 cycles in the Double-Blind Treatment Phase. After the completion of Cycle 12 and upon unblinding, participants determined to have a complete remission (CR), partial remission (PR) or clinical improvement (CI) using the International Working Group (IWG) Response Criteria in the study protocol, were eligible to participate in the extension phase and continue to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle, until disease progression, unacceptable toxicity or voluntary withdrawal.
Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants determined to have a complete remission (CR), partial remission (PR) or clinical improvement (CI) using the International Working Group (IWG) Response Criteria in the study protocol, were eligible to participate in the extension phase and continue to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle, until disease progression, unacceptable toxicity or voluntary withdrawal.
Participants received 0.5 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase. After the completion of cycle 12 and upon unblinding, participants determined to have a complete remission (CR), partial remission (PR) or clinical improvement (CI) using the International Working Group (IWG) Response Criteria in the study protocol, were eligible to participate in the extension phase and continue to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle, until disease progression, unacceptable toxicity or voluntary withdrawal.
Outcomes
Primary Outcome Measures
Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment
A clinical responder was defined as either:
A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or
A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or
A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable.
Participants who discontinued the study early without achieving clinical response were counted as non-responders.
Secondary Outcome Measures
Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment
A clinical responder was defined as either:
A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or
A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or
A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable.
Participants who discontinued the study early without achieving clinical response were counted as non-responders.
Time to the First Clinical Response
The time to the first clinical response achieved within 168 days after the first study drug dosing date was calculated for participants who achieved a clinical response as:
Start date of the first clinical response - the first study drug date +1.
A clinical responder was defined as either:
A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or
A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or
A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable.
Duration of First Clinical Response
For RBC-transfusion-dependent patients, duration of response was calculated as the last day of response - first day of response +1, where the last day of response was the date of the first RBC-transfusion administrated at or more than 56 days after the response started. For patients who did not receive a subsequent transfusion after the response started, the end date of response was censored at the day of last hemoglobin assessment.
For RBC-transfusion-independent patients, the duration of response was calculated as the last day of response - first day of response +1, where the last day of response was the earlier of the date of a hemoglobin increase of < 2.0 g/dL and the date of a RBC transfusion at ≥ 56 days after the response started. For patients whose hemoglobin measurements were always ≥ 2.0 g/dL and never received a RBC transfusion after response started, the end date of the response was censored at the date of last hemoglobin measurement.
Kaplan-Meier methodology was used.
Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Subscale and Total Scores
The FACT-An comprises the four subscales of the 27-item FACT-General Scale (FACT-G), Physical Well-being, Social/Family Well-being, Emotion Well-being, Functional Well-Being, and the Additional Concerns Anemia subscale. Questions are rated on a scale from 0 to 4, where higher scores indicate more impact on quality of life.
Physical Well-being consists of 7 questions, the subscale score ranges from 0-28;
Social/Family Well-being consists of 7 questions, the subscale score ranges from 0-28;
Emotion Well-being consists of 6 questions, the subscale score ranges from 0-24;
Functional Well-Being consists of 7 questions, the subscale score ranges from 0-28;
Anemia subscale consists of 20 questions, the subscale score ranges from 0-80;
Total FACT-An score ranges from 0-188.
Change From Baseline in Hemoglobin Concentration for Responders
Change from Baseline in hemoglobin for participants with a clinical response within the first 6 cycles of treatment.
Change From Baseline in Hemoglobin Concentration for Non-Responders
Change from Baseline in hemoglobin for participants without a clinical response within the first 6 cycles of treatment.
Change From Baseline in Likert Abdominal Pain Scale
Participants rated abdominal discomfort or pain over the previous week on a scale from zero to ten, where zero is no discomfort or pain and ten is the worst pain imaginable.
Percentage of Participants With Clinical Response by Baseline JAK2 Assessment
Percentage of participants who achieved a clinical response, presented by participants with positive and negative janus kinase 2 (JAK2) V617F mutation results at Baseline.
Number of Participants With Adverse Events (AEs)
A serious AE (SAE) was defined as any AE which resulted in death or was life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or constituted an important medical event (events that may have jeopardized the patient or required intervention to prevent one of the outcomes listed above).
The severity of AEs were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 3.0) or according to the following scale:
Grade 1 = Mild; Grade 2 = Moderate; Grade 3 = Severe; Grade 4 = Life-threatening; Grade 5 = Death.
The Investigator determined the relationship between study drug and the occurrence of an AE as "Not Related" or "Related" (since the study was double-blinded, a patient receiving only prednisone could have an AE that was judged as related to pomalidomide, and vice-versa).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00463385
Brief Title
A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia
Official Title
A Phase 2, Prospective, Randomized, Multicenter, Double-blind, Active-control, Parallel-group Study to Determine the Safety of and to Select a Treatment Regimen of CC-4047 (Pomalidomide) Either as Single-agent or in Combination With Prednisone to Study Further in Subjects With Myelofibrosis With Myeloid Metaplasia
Study Type
Interventional
2. Study Status
Record Verification Date
November 2019
Overall Recruitment Status
Completed
Study Start Date
April 1, 2007 (Actual)
Primary Completion Date
May 1, 2009 (Actual)
Study Completion Date
December 31, 2013 (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 purpose of this study is to determine the safety of and to select a treatment regimen of pomalidomide (CC-4047) either as single-agent or in combination with prednisone to study further in patients with myelofibrosis with myeloid metaplasia (MMM).
Detailed Description
Participants received study treatment in the Double Blind Treatment Phase for up to 12 cycles (336 days; 12 cycles of 28 days each). Participants who completed the Double-Blind Treatment Phase were unblinded and, if receiving pomalidomide and determined to have a complete remission (CR), partial remission (PR) or clinical improvement (CI) using the International Working Group (IWG) Response Criteria in the study protocol, could have continued on their current dose of pomalidomide until disease progression. Participants receiving placebo were discontinued from the study.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelofibrosis With Myeloid Metaplasia, Myeloid Metaplasia, Myelofibrosis
Keywords
Celgene, CC-4047, Myelofibrosis, myelofibrosis with myeloid metaplasia, myeloid metaplasia, JAK2, CC-4047-MMM-001, Prednisone, Phase II, pomalidomide, bone marrow histology, imids, MMM, Ashkenazi Jewish Population, exposure to Thorotrast, exposure to solvents (benzene and toluene), acute megakaryocytic leukemia, history of polycythemia vera
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
88 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Prednisone
Arm Type
Experimental
Arm Description
Participants received oral prednisone from Day 1-28 of each 28-day cycle for up to 3 cycles (84 days), 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day, and pomalidomide placebo tablets on Days 1-28 for up to 12 cycles in the Double-Blind Treatment Phase.
After the completion of cycle 12 and upon unblinding, participants were discontinued from the study.
Arm Title
Pomalidomide
Arm Type
Experimental
Arm Description
Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and prednisone placebo tablets on Days 1-28 for the first 3 cycles in the Double-Blind Treatment Phase.
After the completion of Cycle 12 and upon unblinding, participants determined to have a complete remission (CR), partial remission (PR) or clinical improvement (CI) using the International Working Group (IWG) Response Criteria in the study protocol, were eligible to participate in the extension phase and continue to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle, until disease progression, unacceptable toxicity or voluntary withdrawal.
Arm Title
Pomalidomide 2 mg + Prednisone
Arm Type
Experimental
Arm Description
Participants received 2 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase.
After the completion of cycle 12 and upon unblinding, participants determined to have a complete remission (CR), partial remission (PR) or clinical improvement (CI) using the International Working Group (IWG) Response Criteria in the study protocol, were eligible to participate in the extension phase and continue to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle, until disease progression, unacceptable toxicity or voluntary withdrawal.
Arm Title
Pomalidomide 0.5 mg + Prednisone
Arm Type
Experimental
Arm Description
Participants received 0.5 mg oral pomalidomide daily from Day 1-28 of each 28-day cycle for up to 12 cycles (336 days), and oral prednisone tablets on Days 1-28 for the first 3 cycles, 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day in the Double-Blind Treatment Phase.
After the completion of cycle 12 and upon unblinding, participants determined to have a complete remission (CR), partial remission (PR) or clinical improvement (CI) using the International Working Group (IWG) Response Criteria in the study protocol, were eligible to participate in the extension phase and continue to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle, until disease progression, unacceptable toxicity or voluntary withdrawal.
Intervention Type
Drug
Intervention Name(s)
Pomalidomide
Other Intervention Name(s)
CC-4047, Pomalyst
Intervention Type
Drug
Intervention Name(s)
Prednisone
Intervention Description
Participants will take oral prednisone in the evening for 3 cycles of 28 days each (up to 84 days). The dose will be as follows: 1st cycle = 30 mg daily, 2nd cycle = 15 mg daily, 3rd cycle = 15 mg every other day.
Intervention Type
Drug
Intervention Name(s)
Placebo to pomalidomide
Intervention Description
Matching pomalidomide placebo tablets
Intervention Type
Drug
Intervention Name(s)
Placebo to prednisone
Intervention Description
Matching prednisone placebo tablets
Primary Outcome Measure Information:
Title
Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment
Description
A clinical responder was defined as either:
A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or
A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or
A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable.
Participants who discontinued the study early without achieving clinical response were counted as non-responders.
Time Frame
Up to 168 days
Secondary Outcome Measure Information:
Title
Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment
Description
A clinical responder was defined as either:
A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or
A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or
A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable.
Participants who discontinued the study early without achieving clinical response were counted as non-responders.
Time Frame
Up to 336 days
Title
Time to the First Clinical Response
Description
The time to the first clinical response achieved within 168 days after the first study drug dosing date was calculated for participants who achieved a clinical response as:
Start date of the first clinical response - the first study drug date +1.
A clinical responder was defined as either:
A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or
A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or
A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable.
Time Frame
Up to 168 days
Title
Duration of First Clinical Response
Description
For RBC-transfusion-dependent patients, duration of response was calculated as the last day of response - first day of response +1, where the last day of response was the date of the first RBC-transfusion administrated at or more than 56 days after the response started. For patients who did not receive a subsequent transfusion after the response started, the end date of response was censored at the day of last hemoglobin assessment.
For RBC-transfusion-independent patients, the duration of response was calculated as the last day of response - first day of response +1, where the last day of response was the earlier of the date of a hemoglobin increase of < 2.0 g/dL and the date of a RBC transfusion at ≥ 56 days after the response started. For patients whose hemoglobin measurements were always ≥ 2.0 g/dL and never received a RBC transfusion after response started, the end date of the response was censored at the date of last hemoglobin measurement.
Kaplan-Meier methodology was used.
Time Frame
Up to 40 months
Title
Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Subscale and Total Scores
Description
The FACT-An comprises the four subscales of the 27-item FACT-General Scale (FACT-G), Physical Well-being, Social/Family Well-being, Emotion Well-being, Functional Well-Being, and the Additional Concerns Anemia subscale. Questions are rated on a scale from 0 to 4, where higher scores indicate more impact on quality of life.
Physical Well-being consists of 7 questions, the subscale score ranges from 0-28;
Social/Family Well-being consists of 7 questions, the subscale score ranges from 0-28;
Emotion Well-being consists of 6 questions, the subscale score ranges from 0-24;
Functional Well-Being consists of 7 questions, the subscale score ranges from 0-28;
Anemia subscale consists of 20 questions, the subscale score ranges from 0-80;
Total FACT-An score ranges from 0-188.
Time Frame
Baseline and Cycle 6 (168 days).
Title
Change From Baseline in Hemoglobin Concentration for Responders
Description
Change from Baseline in hemoglobin for participants with a clinical response within the first 6 cycles of treatment.
Time Frame
Baseline, Cycle 6 (168 days)
Title
Change From Baseline in Hemoglobin Concentration for Non-Responders
Description
Change from Baseline in hemoglobin for participants without a clinical response within the first 6 cycles of treatment.
Time Frame
Baseline, Cycle 6 (168 days)
Title
Change From Baseline in Likert Abdominal Pain Scale
Description
Participants rated abdominal discomfort or pain over the previous week on a scale from zero to ten, where zero is no discomfort or pain and ten is the worst pain imaginable.
Time Frame
Baseline and Cycle 6 (168 days)
Title
Percentage of Participants With Clinical Response by Baseline JAK2 Assessment
Description
Percentage of participants who achieved a clinical response, presented by participants with positive and negative janus kinase 2 (JAK2) V617F mutation results at Baseline.
Time Frame
Up to 336 days
Title
Number of Participants With Adverse Events (AEs)
Description
A serious AE (SAE) was defined as any AE which resulted in death or was life-threatening, required or prolonged inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, or constituted an important medical event (events that may have jeopardized the patient or required intervention to prevent one of the outcomes listed above).
The severity of AEs were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE, Version 3.0) or according to the following scale:
Grade 1 = Mild; Grade 2 = Moderate; Grade 3 = Severe; Grade 4 = Life-threatening; Grade 5 = Death.
The Investigator determined the relationship between study drug and the occurrence of an AE as "Not Related" or "Related" (since the study was double-blinded, a patient receiving only prednisone could have an AE that was judged as related to pomalidomide, and vice-versa).
Time Frame
From date of the first dose of the study drug until discontinuation or the data cut-off date (up to approximately 45 months).
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Must sign an informed consent form
Must be >18 years of age
Must be diagnosed with myelofibrosis
Eligibility is based on local pathology review of bone marrow aspirate and biopsy
Screening total hemoglobin level < 10g/dL or transfusion-dependent anemia defined as per International Working Group (IWG) criteria.
Must have adequate organ function as demonstrated by the following ≤ 14 days prior to starting study drug:
Alanine aminotransferase (ALT; SGPT)/aspartate aminotransferase (AST; SGOT) ≤ 3 x upper limit of normal (ULN), [unless upon judgment of the treating physician, it is believed to be due to extra-medullary hematopoiesis (EMH)].
Total Bilirubin <3x ULN or Direct Bilirubin <2 x ULN
Serum creatinine ≤ 2.0 mg/dL
Absolute neutrophil count ≥ 1,000/μL (≥ 1 x 10^9/L).
Platelet count ≥ 50,000 /μL (≥ 50 x 10^9/L).
Patients must be willing to receive transfusion of blood products
Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 at screening.
Must be able to adhere to the study visit schedule and other protocol requirements.
No active malignancies with the exception of controlled prostate cancer, basal cell or squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix or breast.
Must agree to follow pregnancy precautions as required per the protocol
Exclusion Criteria:
Known positive status for human immunodeficiency virus (HIV), hepatitis B carrier, or active hepatitis C infection.
Previous untoward reaction to corticosteroid (specifically, prednisone) therapy that was severe enough, in the opinion of the treating physician, to preclude study participation.
The use of any growth factors, cytotoxic chemotherapeutic agents (e.g. hydroxyurea and anagrelide), corticosteroids, or experimental drug or therapy within a minimum of 28 days of starting CC-4047 and/or lack of recovery from all toxicity from previous therapy to grade 1 or better (e.g. alpha interferon may require 84 days of longer or washout).
Prior therapy with CC-4047 or, lenalidomide or thalidomide for Myelofibrosis with myeloid metaplasia (MMM). (Prior prednisone use as a therapy for MMM is allowed, but not within 28 days of starting CC-4047).
History of deep vein thrombosis or pulmonary embolism within one year of starting study medication.
Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
Pregnant or lactating females
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Robert Peter Gale, MD, PhD
Organizational Affiliation
Celgene Corporation
Official's Role
Study Director
Facility Information:
Facility Name
UCLA School of Medicine Hematology/Oncology
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Memorial Sloan-Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10021-6007
Country
United States
Facility Name
New York Presbyterian HospitalWeill Medical College of Cornell University
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
MD Anderson Cancer Center Leukemia Department
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Fred Hutchinson Cancer Research Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109-4417
Country
United States
Facility Name
Medical University of Vienna, Department of Internal Medicine, Hematology
City
Vienna
ZIP/Postal Code
A-1090
Country
Austria
Facility Name
Fondazione IRCCS Policlinico San Matteo
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
IRCCS Policlinico S. Matteo
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
Hematology DepartmentHospital Clinic
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Royal Hallamshire Hospital Sheffield Teaching Hospitals NHS Trust
City
Sheffield
ZIP/Postal Code
S10 2JF
Country
United Kingdom
12. IPD Sharing Statement
Citations:
Citation
Barosi G, et al. Decrease Of T Regulatory Cells In Patients With Myelofibrosis Receiving Ruxolitinib. Presented at American Society of Hematology 2013, December 7-10, 2013, New Orleans, LA. Abstract No. 4057. Blood, 2013;122(21)
Results Reference
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Learn more about this trial
A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia
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