Myelofibrosis With Myeloid MetaplasiaPhase 2NCT00463385
A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia
A Phase 2 study testing Pomalidomide and Prednisone in people with Myelofibrosis With Myeloid Metaplasia and Myeloid Metaplasia, run by Celgene at 11 sites in 5 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis With Myeloid Metaplasia, Myeloid Metaplasia, Myelofibrosis
- What is being tested?
- Pomalidomide and Prednisone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 88 participants
- Where
- 11 sitesin 5 countries
- Study dates
- 1 Apr 2007to 31 Dec 2013
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
Show all 16Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
11 sites in 5 countries.
11 sites in 5 countries
United States6 sites
MD Anderson Cancer Center Leukemia Department
Houston, Texas, 77030
UCLA School of Medicine Hematology/Oncology
Los Angeles, California, 90095
Memorial Sloan-Kettering Cancer Center
New York, New York, 10021-6007
New York Presbyterian HospitalWeill Medical College of Cornell University
New York, New York, 10021
Mayo Clinic
Rochester, Minnesota, 55905
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109-4417
Italy2 sites
Fondazione IRCCS Policlinico San Matteo
Pavia, 27100
IRCCS Policlinico S. Matteo
Pavia, 27100
Austria1 site
Medical University of Vienna, Department of Internal Medicine, Hematology
Vienna, A-1090
United Kingdom1 site
Royal Hallamshire Hospital Sheffield Teaching Hospitals NHS Trust
Sheffield, S10 2JF
About this study
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
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).
Read the 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.
- Being tested
- Pomalidomide, Prednisone, Placebo to pomalidomide, Placebo to prednisone
- 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
- Medical subject headings
- Primary Myelofibrosis, Leukemia, Megakaryoblastic, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PomalidomideDrug
Also called CC-4047, Pomalyst
PrednisoneDrug
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.
Placebo to pomalidomideDrug
Matching pomalidomide placebo tablets
Placebo to prednisoneDrug
Matching prednisone placebo tablets
| Study group | Role | What they receive |
|---|---|---|
| Prednisone | Experimental | 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. Receives: Drug: Prednisone; Drug: Placebo to pomalidomide |
| Pomalidomide | Experimental | 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. Receives: Drug: Pomalidomide; Drug: Placebo to prednisone |
| Pomalidomide 2 mg + Prednisone | Experimental | 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. Receives: Drug: Pomalidomide; Drug: Prednisone |
| Pomalidomide 0.5 mg + Prednisone | Experimental | 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. Receives: Drug: Pomalidomide; Drug: Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 168 days
Secondary outcomes (9)
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.
Measured: Up to 336 days
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.
Measured: Up to 168 days
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.
Measured: Up to 40 months
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.
Measured: Baseline and Cycle 6 (168 days).
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.
Measured: Baseline, Cycle 6 (168 days)
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.
Measured: Baseline, Cycle 6 (168 days)
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.
Measured: Baseline and Cycle 6 (168 days)
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.
Measured: Up to 336 days
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).
Measured: From date of the first dose of the study drug until discontinuation or the data cut-off date (up to approximately 45 months).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2007 (estimated)
- Study start1 Apr 2007
- Primary completion1 May 2009
- Study completion31 Dec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myelofibrosis With Myeloid MetaplasiaStudies of the same condition, open and under way.
- More trials run by CelgeneThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 88 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Robert Peter Gale, MD, PhD, Study director, Celgene Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00463385
- Sponsor's protocol ID
- CC-4047-MMM-001
- Status last verified
- Nov 2019
- First submitted
- 19 Apr 2007
- Last updated
- 20 Nov 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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)
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00463385
- Acronym / sponsor ID
- CC-4047-MMM-001
- Keywords
- Celgene, CC-4047, Myelofibrosis, myelofibrosis with myeloid metaplasia, myeloid metaplasia, JAK2, CC-4047-MMM-001, Prednisone
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 88 participants (actual)
- Lead sponsor
- CelgeneIndustry (pharma, biotech, devices)
- Study start
- 1 Apr 2007 (actual)
- Primary completion
- 1 May 2009 (actual)
- Study completion
- 31 Dec 2013 (actual)
- First posted
- 20 Apr 2007 (estimated)
- Results
- Posted 28 Oct 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Prednisone | Experimental | Drug: Prednisone, Drug: Placebo to pomalidomideParticipants 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. |
| Pomalidomide | Experimental | Drug: Pomalidomide, Drug: Placebo to prednisoneParticipants 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. |
| Pomalidomide 2 mg + Prednisone | Experimental | Drug: Pomalidomide, Drug: PrednisoneParticipants 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. |
| Pomalidomide 0.5 mg + Prednisone | Experimental | Drug: Pomalidomide, Drug: PrednisoneParticipants 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PomalidomideAlso: CC-4047, Pomalyst | Drug | Pomalidomide, Pomalidomide 0.5 mg + Prednisone, Pomalidomide 2 mg + Prednisone |
| PrednisoneParticipants 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. | Drug | Pomalidomide 0.5 mg + Prednisone, Pomalidomide 2 mg + Prednisone, Prednisone |
| Placebo to pomalidomideMatching pomalidomide placebo tablets | Drug | Prednisone |
| Placebo to prednisoneMatching prednisone placebo tablets | Drug | Pomalidomide |
Primary outcome
Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment
Time frame: Up to 168 days
A clinical responder was defined as either: 1. A baseline red blood cell (RBC)-transfusion-dependent participant with a ≥ 56 consecutive day RBC transfusion-free period after the first dose of study drug, or 2. A baseline RBC-transfusion-independent participant with an increase in hemoglobin of 2.0 g/dL or more from baseline for ≥ 56 consecutive days in the absence of RBC transfusions, or 3. A participant with either a ≥ 50% reduction in palpable splenomegaly of a spleen that was ≥ 10 cm at baseline or a spleen that was palpable at > 5 cm and became not palpable. Participants who discontinued the study early without achieving clinical response were counted as non-responders.
9 secondary outcomes
Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment
Time frame: Up to 336 days
Time to the First Clinical Response
Time frame: Up to 168 days
Duration of First Clinical Response
Time frame: Up to 40 months
Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Subscale and Total Scores
Time frame: Baseline and Cycle 6 (168 days).
Change From Baseline in Hemoglobin Concentration for Responders
Time frame: Baseline, Cycle 6 (168 days)
Change From Baseline in Hemoglobin Concentration for Non-Responders
Time frame: Baseline, Cycle 6 (168 days)
Change From Baseline in Likert Abdominal Pain Scale
Time frame: Baseline and Cycle 6 (168 days)
Percentage of Participants With Clinical Response by Baseline JAK2 Assessment
Time frame: Up to 336 days
Number of Participants With Adverse Events (AEs)
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).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- BackgroundBarosi 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)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert Peter Gale, MD, PhD study director
Celgene Corporation
11 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pomalidomide and Prednisone in people with Myelofibrosis With Myeloid Metaplasia and Myeloid Metaplasia, run by Celgene at 11 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Italy, Austria, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Oct 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment (time frame: Up to 168 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00463385, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2019.