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.
COMPLETED
PHASE2
70 participants
6 months
2019-06-10
Participant Flow
Recruitment Period: July 2009 to March 2013.
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
| 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 monthsPrimary 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
| 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
Group 2 CC-4047
Group 3 CC-4047 + Prednisone
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
| 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
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place