Trial Outcomes & Findings for Pomalidomide for Myelofibrosis Patients (NCT NCT00946270)

NCT ID: NCT00946270

Last Updated: 2019-06-10

Results Overview

Primary endpoint is best overall response. An evaluable subject classified as a treatment success for the primary endpoint if the subject's best overall response is clinical improvement (CI) as determined by International Working Group Criteria over the first 6 cycles of study treatment. International Working Group (IWG) consensus criteria for treatment response in myelofibrosis - Clinical improvement (CI) in anemia 1/ A minimum 20g/L increase in hemoglobin level or 2. becoming transfusion independent for at least 8 week duration.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

70 participants

Primary outcome timeframe

6 months

Results posted on

2019-06-10

Participant Flow

Recruitment Period: July 2009 to March 2013.

Participant milestones

Participant milestones
Measure
Group 1: Oral CC-4047 3.0 mg
CC-4047 3.0 mg orally daily. CC-4047: 3.0 mg orally daily starting on day 1 through 21.
Group 2: Oral CC-4047 0.5 mg
CC-4047 0.5 mg orally daily . CC-4047: 0.5 mg orally daily starting on day 1 through 28.
Group 3: Oral CC-4047 0.5 mg + Prednisone
CC-4047 0.5 mg orally daily. Prednisone given during first 3 cycles of therapy. It will be dosed orally at the dose of 30 mg/day during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued. CC-4047: 0.5 mg capsules daily by mouth day 1 through day 28. Prednisone: 30 mg by mouth daily during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.
Overall Study
STARTED
21
20
29
Overall Study
COMPLETED
21
20
29
Overall Study
NOT COMPLETED
0
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pomalidomide for Myelofibrosis Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Group 1 CC-4047
n=21 Participants
CC-4047 3.0 mg orally daily starting on day 1 through 21.
Group 2 CC-4047
n=20 Participants
CC-4047 0.5 mg orally daily starting on day 1 through 28.
Group 3 CC-4047 + Predniaone
n=29 Participants
CC-4047 0.5 mg orally daily. Prednisone given during first 3 cycles of therapy. It will be dosed orally at the dose of 30 mg/day during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued. CC-4047: 0.5 mg capsules daily by mouth day 1 through day 28. Prednisone: 30 mg by mouth daily during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.
Total
n=70 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
0 Participants
n=483 Participants
Age, Categorical
Between 18 and 65 years
10 Participants
n=93 Participants
7 Participants
n=4 Participants
8 Participants
n=27 Participants
25 Participants
n=483 Participants
Age, Categorical
>=65 years
11 Participants
n=93 Participants
13 Participants
n=4 Participants
21 Participants
n=27 Participants
45 Participants
n=483 Participants
Age, Continuous
66 years
n=93 Participants
69 years
n=4 Participants
69 years
n=27 Participants
68 years
n=483 Participants
Sex: Female, Male
Female
10 Participants
n=93 Participants
10 Participants
n=4 Participants
9 Participants
n=27 Participants
29 Participants
n=483 Participants
Sex: Female, Male
Male
11 Participants
n=93 Participants
10 Participants
n=4 Participants
20 Participants
n=27 Participants
41 Participants
n=483 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
0 Participants
n=483 Participants
Race (NIH/OMB)
Asian
0 Participants
n=93 Participants
1 Participants
n=4 Participants
0 Participants
n=27 Participants
1 Participants
n=483 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
0 Participants
n=483 Participants
Race (NIH/OMB)
Black or African American
2 Participants
n=93 Participants
4 Participants
n=4 Participants
1 Participants
n=27 Participants
7 Participants
n=483 Participants
Race (NIH/OMB)
White
19 Participants
n=93 Participants
15 Participants
n=4 Participants
28 Participants
n=27 Participants
62 Participants
n=483 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
0 Participants
n=483 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
0 Participants
n=483 Participants
Region of Enrollment
United States
21 participants
n=93 Participants
20 participants
n=4 Participants
29 participants
n=27 Participants
70 participants
n=483 Participants

PRIMARY outcome

Timeframe: 6 months

Primary endpoint is best overall response. An evaluable subject classified as a treatment success for the primary endpoint if the subject's best overall response is clinical improvement (CI) as determined by International Working Group Criteria over the first 6 cycles of study treatment. International Working Group (IWG) consensus criteria for treatment response in myelofibrosis - Clinical improvement (CI) in anemia 1/ A minimum 20g/L increase in hemoglobin level or 2. becoming transfusion independent for at least 8 week duration.

Outcome measures

Outcome measures
Measure
Group 1 CC-4047
n=21 Participants
CC-4047 3.0 mg orally daily starting on day 1 through 21
Group 2
n=20 Participants
CC-4047 0.5 mg orally daily starting on day 1 through 28.
Group 3 CC-4047 + Prednisone
n=29 Participants
CC-4047 0.5 mg orally daily. Prednisone given during first 3 cycles of therapy. It will be dosed orally at the dose of 30 mg/day during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued. CC-4047: 0.5 mg capsules daily by mouth day 1 through day 28. Prednisone: 30 mg by mouth daily during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.
Number of Participants With Best Overall Response
0 Participants
3 Participants
6 Participants

Adverse Events

Group 1 CC-4047

Serious events: 15 serious events
Other events: 13 other events
Deaths: 2 deaths

Group 2 CC-4047

Serious events: 8 serious events
Other events: 10 other events
Deaths: 3 deaths

Group 3 CC-4047 + Prednisone

Serious events: 11 serious events
Other events: 14 other events
Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure
Group 1 CC-4047
n=21 participants at risk
CC-4047 3.0 mg orally daily starting on day 1 through 21.
Group 2 CC-4047
n=20 participants at risk
CC-4047 0.5 mg orally daily starting on day 1 through 28.
Group 3 CC-4047 + Prednisone
n=29 participants at risk
CC-4047 0.5 mg orally daily. Prednisone given during first 3 cycles of therapy. It will be dosed orally at the dose of 30 mg/day during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued. CC-4047: 0.5 mg capsules daily by mouth day 1 through day 28. Prednisone: 30 mg by mouth daily during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.
Blood and lymphatic system disorders
Anemia
19.0%
4/21 • Number of events 4 • Up to 3 years
5.0%
1/20 • Number of events 4 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Respiratory, thoracic and mediastinal disorders
Aspiration
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Musculoskeletal and connective tissue disorders
Asthenia
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Cardiac disorders
Atrial Fibrillation
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Cardiac disorders
Cardiac ischemia/infarction
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Cardiac disorders
Cardiac Valve disease
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Cardiac disorders
Chest Pain
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Nervous system disorders
Confusion
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Cardiac disorders
Congestive Heart Failure
0.00%
0/21 • Up to 3 years
10.0%
2/20 • Number of events 4 • Up to 3 years
3.4%
1/29 • Number of events 2 • Up to 3 years
General disorders
Death
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
General disorders
Device Complication
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Respiratory, thoracic and mediastinal disorders
Dyspnea
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Blood and lymphatic system disorders
Edema Limb
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Blood and lymphatic system disorders
Thrombocytopenia
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Immune system disorders
Hypersensitivity
9.5%
2/21 • Number of events 2 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
General disorders
Fatigue
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
General disorders
Fever
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Gastrointestinal disorders
Fistula Rectum
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Musculoskeletal and connective tissue disorders
Fracture
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
General disorders
Fluid Overload - post transfusion
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Blood and lymphatic system disorders
Hemorrhage
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Metabolism and nutrition disorders
Hyperkalemia
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Infections and infestations
Infection
0.00%
0/21 • Up to 3 years
10.0%
2/20 • Number of events 2 • Up to 3 years
10.3%
3/29 • Number of events 4 • Up to 3 years
Surgical and medical procedures
Intra-operative injury
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Cardiac disorders
Left Ventricular Systolic Dysfunction
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Nervous system disorders
Neuropathy
4.8%
1/21 • Number of events 2 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Blood and lymphatic system disorders
Neutropenia
23.8%
5/21 • Number of events 8 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Infections and infestations
Neutropenic Fever
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
General disorders
Pain
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
6.9%
2/29 • Number of events 2 • Up to 3 years
Vascular disorders
Peripheral Edema
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
6.9%
2/29 • Number of events 2 • Up to 3 years
Infections and infestations
Pneumonia
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
10.3%
3/29 • Number of events 3 • Up to 3 years
Infections and infestations
Pneumonitis
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
10.3%
3/29 • Number of events 3 • Up to 3 years
Cardiac disorders
Pulmonary Hypertension
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Skin and subcutaneous tissue disorders
Rash
9.5%
2/21 • Number of events 2 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Renal and urinary disorders
Renal Failure
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
General disorders
Rigors/Chills
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Nervous system disorders
Seizure
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Gastrointestinal disorders
Small bowel obstruction
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Vascular disorders
Splenic infarct
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Gastrointestinal disorders
Stricture/Stenosis Esophagus
0.00%
0/21 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Blood and lymphatic system disorders
Subdural Hematoma
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years

Other adverse events

Other adverse events
Measure
Group 1 CC-4047
n=21 participants at risk
CC-4047 3.0 mg orally daily starting on day 1 through 21.
Group 2 CC-4047
n=20 participants at risk
CC-4047 0.5 mg orally daily starting on day 1 through 28.
Group 3 CC-4047 + Prednisone
n=29 participants at risk
CC-4047 0.5 mg orally daily. Prednisone given during first 3 cycles of therapy. It will be dosed orally at the dose of 30 mg/day during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued. CC-4047: 0.5 mg capsules daily by mouth day 1 through day 28. Prednisone: 30 mg by mouth daily during cycle 1, 15 mg/day during cycle 2, and 15 mg every other day during cycle 3, and then it will be discontinued.
Gastrointestinal disorders
Abdominal hematoma
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
General disorders
Pain
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
6.9%
2/29 • Number of events 2 • Up to 3 years
Metabolism and nutrition disorders
Hyperuricemia
0.00%
0/21 • Up to 3 years
10.0%
2/20 • Number of events 2 • Up to 3 years
0.00%
0/29 • Up to 3 years
Blood and lymphatic system disorders
Leukocytosis
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Cardiac disorders
Cardiac general
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Cardiac disorders
Cerebrovascualr Ischemia
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Gastrointestinal disorders
Constipation
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Nervous system disorders
Dizziness
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Respiratory, thoracic and mediastinal disorders
Dyspena
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
General disorders
Fall
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
General disorders
Fatigue
14.3%
3/21 • Number of events 3 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
6.9%
2/29 • Number of events 2 • Up to 3 years
General disorders
Abnormal Gait
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
General disorders
Headache
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Blood and lymphatic system disorders
Anemia
19.0%
4/21 • Number of events 4 • Up to 3 years
10.0%
2/20 • Number of events 2 • Up to 3 years
6.9%
2/29 • Number of events 2 • Up to 3 years
Blood and lymphatic system disorders
Hemolysis
9.5%
2/21 • Number of events 2 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
Blood and lymphatic system disorders
Hemorrhage
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
6.9%
2/29 • Number of events 2 • Up to 3 years
Cardiac disorders
Hypertension
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Blood and lymphatic system disorders
Iron increased
4.8%
1/21 • Number of events 1 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Blood and lymphatic system disorders
Neutropenia
23.8%
5/21 • Number of events 5 • Up to 3 years
0.00%
0/20 • Up to 3 years
6.9%
2/29 • Number of events 2 • Up to 3 years
Infections and infestations
Pneumonia
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Eye disorders
Retinal Detachment
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Metabolism and nutrition disorders
Hypokalemia
0.00%
0/21 • Up to 3 years
5.0%
1/20 • Number of events 1 • Up to 3 years
0.00%
0/29 • Up to 3 years
Nervous system disorders
Syncope
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
3.4%
1/29 • Number of events 1 • Up to 3 years
Skin and subcutaneous tissue disorders
Urticaria
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years
General disorders
Weight gain
4.8%
1/21 • Number of events 1 • Up to 3 years
0.00%
0/20 • Up to 3 years
0.00%
0/29 • Up to 3 years

Additional Information

Dr. Srdan Verstovsek, MD./Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-745-3429

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place