A Phase II Study of Pomalidomide in Myelofibrosis With Myeloid Metaplasia

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Celgene ( Celgene Corporation )

ClinicalTrials.gov Identifier:

NCT00463385

First received: April 19, 2007

Last updated: June 4, 2015

Last verified: June 2015

History of Changes

Results First Received: March 7, 2013

Study Type:	Interventional
Study Design:	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, 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
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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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	0	0	0	0
NOT COMPLETED	22	22	22	22
Unspecified	4	3	6	4
Adverse Event	5	8	4	2
Lack of Efficacy	6	6	7	9
Withdrawal by Subject	3	3	3	4
Lost to Follow-up	0	0	0	1
Death	2	1	2	0
Missing	2	0	0	2
1 participant received commercial drug	0	1	0	0

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.
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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

Overall Participants Analyzed
[Units: Participants]

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

Male

Race/Ethnicity, Customized
[Units: Participants]

White

Black

Hispanic

Janus kinase 2 (JAK2) Mutation ^[1]
[Units: Participants]

Negative

Positive

Missing

^[1]	JAK2^V617F mutation result based on quantitative polymerase chain reaction (PCR) analysis in neutrophil preparation.

Eastern Cooperative Oncology Group (ECOG) Performance Status ^[1]
[Units: Participants]

Grade 0

Grade 1

Grade 2

Missing

^[1]

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;

Restricted in physically strenuous activity; ambulatory and able to carry out light work;
Ambulatory and capable of all selfcare; unable to perform work activities. Up and about > 50% of waking hours;
Capable of only limited selfcare, confined to bed/chair > 50% of waking hours;
Completely disabled. Confined to bed or chair;
Dead

Myelofibrosis with myeloid metaplasia Subtype
[Units: Participants]

Agnogenic Myeloid Metaplasia (AMM)

Postpolycythemic Myeloid Metaplasia (PPMM)

Postthromocythemic Myeloid Metaplasia (PTMM)

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 ^[1]
[Units: Participants]

Yes

^[1]	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.

RBC Transfusion Burden
[Units: Units/28 days]
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)

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 ]

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2. Secondary:

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

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3. Secondary:

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

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4. Secondary:

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

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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). ]

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6. Secondary:

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

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7. Secondary:

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

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8. Secondary:

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

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9. Secondary:

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

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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). ]

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Serious Adverse Events

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Other Adverse Events

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Limitations and Caveats

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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
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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:

	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.
	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.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The investigator shall have the right to publish and/or present study data provided that the investigator shall (i) furnish the sponsor a copy of any proposed publication or presentation generally thirty (60) days in advance of the submission, (ii) delete any confidential information of the sponsor, and (iii) delay submission for generally up to ninety (90) days to permit the preparation and filing of intellectual property applications or until sponsor gives its consent in a timely manner.

Results Point of Contact:

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

Responsible Party:	Celgene ( 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:	June 4, 2015

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