A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Celgene Corporation
ClinicalTrials.gov Identifier:
NCT00463385
First received: April 19, 2007
Last updated: August 21, 2013
Last verified: August 2013
Results First Received: March 7, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Myelofibrosis With Myeloid Metaplasia
Myeloid Metaplasia
Myelofibrosis
Interventions: Drug: Pomalidomide
Drug: Prednisone
Drug: Placebo to pomalidomide
Drug: Placebo to prednisone

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Data as of the data cutoff date of 31 January 2011.

Reporting Groups
  Description
Prednisone

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.

Pomalidomide 2 mg

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, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Pomalidomide 2 mg + Prednisone

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, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Pomalidomide 0.5 mg + Prednisone

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, eligible participants continued to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.


Participant Flow:   Overall Study
    Prednisone     Pomalidomide 2 mg     Pomalidomide 2 mg + Prednisone     Pomalidomide 0.5 mg + Prednisone  
STARTED     22     22     22     22  
Treated     22     22     19     22  
COMPLETED     22 [1]   19 [1]   22 [1]   19 [1]
NOT COMPLETED     0     3     0     3  
Active in study at time of data cut-off                 0                 3                 0                 3  
[1] Completed represents participants who are no longer on study.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Prednisone

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.

Pomalidomide 2 mg

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, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Pomalidomide 2 mg + Prednisone

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, eligible participants continued to receive oral pomalidomide 2 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Pomalidomide 0.5 mg + Prednisone

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, eligible participants continued to receive oral pomalidomide 0.5 mg daily, from Days 1-28 of each cycle. Participants could remain on study treatment in the Extension Phase until disease progression, unacceptable toxicity or voluntary withdrawal.

Total Total of all reporting groups

Baseline Measures
    Prednisone     Pomalidomide 2 mg     Pomalidomide 2 mg + Prednisone     Pomalidomide 0.5 mg + Prednisone     Total  
Number of Participants  
[units: participants]
  22     22     22     22     88  
Age  
[units: years]
Median ( Full Range )
  66.0  
  ( 44.0 to 80.0 )  
  68.0  
  ( 50.0 to 83.0 )  
  67.5  
  ( 36.0 to 82.0 )  
  69.5  
  ( 43.0 to 86.0 )  
  67.5  
  ( 36.0 to 86.0 )  
Gender  
[units: participants]
         
Female     8     5     7     9     29  
Male     14     17     15     13     59  
Race/Ethnicity, Customized  
[units: participants]
         
White     21     22     21     22     86  
Black     0     0     1     0     1  
Hispanic     1     0     0     0     1  
Janus kinase 2 (JAK2) Mutation [1]
[units: participants]
         
Negative     6     7     8     8     29  
Positive     13     11     10     9     43  
Missing     3     4     4     5     16  
Eastern Cooperative Oncology Group (ECOG) Performance Status [2]
[units: participants]
         
Grade 0     12     11     6     14     43  
Grade 1     8     9     10     6     33  
Grade 2     2     2     5     2     11  
Missing     0     0     1     0     1  
Myelofibrosis with myeloid metaplasia Subtype  
[units: participants]
         
Agnogenic Myeloid Metaplasia (AMM)     16     16     16     13     61  
Postpolycythemic Myeloid Metaplasia (PPMM)     3     4     4     2     13  
Postthromocythemic Myeloid Metaplasia (PTMM)     3     2     2     7     14  
Time Since Myelofibrosis Diagnosis  
[units: years]
Median ( Full Range )
  1.5  
  ( 0.0 to 14.3 )  
  0.6  
  ( 0.0 to 6.3 )  
  1.1  
  ( 0.0 to 13.0 )  
  1.7  
  ( 0.0 to 10.9 )  
  1.1  
  ( 0.0 to 14.3 )  
Red Blood Cell (RBC) Transfusion Dependence [3]
[units: participants]
         
Yes     12     10     9     12     43  
No     10     12     13     10     45  
RBC Transfusion Burden  
[units: units/28┬ádays]
Median ( Full Range )
  2.0  
  ( 0.0 to 7.0 )  
  1.0  
  ( 0.0 to 6.0 )  
  0.0  
  ( 0.0 to 6.0 )  
  2.0  
  ( 0.0 to 4.0 )  
  1.0  
  ( 0.0 to 7.0 )  
[1] JAK2^V617F mutation result based on quantitative polymerase chain reaction (PCR) analysis in neutrophil preparation.
[2]

ECOG performance status scale and criteria used to assess disease progression, how the disease affects the daily living abilities of the patient, and determine appropriate treatment:

0=Fully active, able to carry on all pre-disease activities;

  1. Restricted in physically strenuous activity; ambulatory and able to carry out light work;
  2. Ambulatory and capable of all selfcare; unable to perform work activities. Up and about > 50% of waking hours;
  3. Capable of only limited selfcare, confined to bed/chair > 50% of waking hours;
  4. Completely disabled. Confined to bed or chair;
  5. Dead
[3] A patient who receives at least a total of two units of RBC transfusion within 28 days on or prior to the first study drug dosing date is an RBC-transfusion-dependent patient. Otherwise a patient is an RBC-transfusion-independent patient.



  Outcome Measures
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1.  Primary:   Percentage of Participants With a Clinical Response Within the First 6 Cycles of Treatment   [ Time Frame: Up to 168 days ]

2.  Secondary:   Percentage of Participants With a Clinical Response Within the First 12 Cycles of Treatment   [ Time Frame: Up to 336 days ]

3.  Secondary:   Time to the First Clinical Response   [ Time Frame: Up to 168 days ]

4.  Secondary:   Duration of First Clinical Response   [ Time Frame: Up to 40 months ]

5.  Secondary:   Change From Baseline in Functional Assessment of Cancer Therapy-Anemia (FACT-An) Subscale and Total Scores   [ Time Frame: Baseline and Cycle 6 (168 days). ]

6.  Secondary:   Change From Baseline in Hemoglobin Concentration for Responders   [ Time Frame: Baseline, Cycle 6 (168 days) ]

7.  Secondary:   Change From Baseline in Hemoglobin Concentration for Non-Responders   [ Time Frame: Baseline, Cycle 6 (168 days) ]

8.  Secondary:   Change From Baseline in Likert Abdominal Pain Scale   [ Time Frame: Baseline and Cycle 6 (168 days) ]

9.  Secondary:   Percentage of Participants With Clinical Response by Baseline JAK2 Assessment   [ Time Frame: Up to 336 days ]

10.  Secondary:   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). ]


  Serious Adverse Events


  Other Adverse Events


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Associate Director, Clinical Trials Disclosure
Organization: Celgene Corporation
phone: 1-888-260-1599
e-mail: clinicaltrialdisclosure@celgene.com


No publications provided


Responsible Party: Celgene Corporation
ClinicalTrials.gov Identifier: NCT00463385     History of Changes
Other Study ID Numbers: CC-4047-MMM-001
Study First Received: April 19, 2007
Results First Received: March 7, 2013
Last Updated: August 21, 2013
Health Authority: United States: Food and Drug Administration