Trial Outcomes & Findings for A Phase II Study of CC-5013 in Myelofibrosis (NCT NCT00087672)
NCT ID: NCT00087672
Last Updated: 2012-08-07
Results Overview
Response evaluation, sustained for 2 weeks: Complete Remission (Neutrophil count between 1 to 10 x 10\^9/L without peripheral blasts in blood or bone marrow); Partial Hematologic Response/Partial Remission (Increase in neutrophil by 50% + above 10\^9/L for neutropenia); Hematologic Improvement (increase in Neutrophil count, hemoglobin, platelet count or reduction in blood/marrow blasts) or No Response. If nine or \< patients respond to therapy (response other than 'No Response'), therapy declared ineffective. However, if 11 or \> patients respond to therapy, therapy considered efficacious.
COMPLETED
PHASE2
41 participants
3 - 4 Months for all patients; 24 months for responders
2012-08-07
Participant Flow
Recruitment Period: 7/8/04 to 2/27/09. All patients registered at the University of Texas MD Anderson Cancer Center.
The maximum accrual of 41 was met.
Participant milestones
| Measure |
CC-5013
10 mg orally daily
|
|---|---|
|
Overall Study
STARTED
|
41
|
|
Overall Study
COMPLETED
|
41
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Phase II Study of CC-5013 in Myelofibrosis
Baseline characteristics by cohort
| Measure |
CC-5013
n=41 Participants
10 mg orally daily
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
19 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
22 Participants
n=5 Participants
|
|
Age Continuous
|
66 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
18 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
41 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3 - 4 Months for all patients; 24 months for respondersPopulation: Intention to treat: After a total of 41 patients were enrolled in study, nine or more patients responded to the therapy, therapy declared effective.
Response evaluation, sustained for 2 weeks: Complete Remission (Neutrophil count between 1 to 10 x 10\^9/L without peripheral blasts in blood or bone marrow); Partial Hematologic Response/Partial Remission (Increase in neutrophil by 50% + above 10\^9/L for neutropenia); Hematologic Improvement (increase in Neutrophil count, hemoglobin, platelet count or reduction in blood/marrow blasts) or No Response. If nine or \< patients respond to therapy (response other than 'No Response'), therapy declared ineffective. However, if 11 or \> patients respond to therapy, therapy considered efficacious.
Outcome measures
| Measure |
CC-5013
n=41 Participants
10 mg orally daily
|
|---|---|
|
Efficacy of CC-5013 in Myelofibrosis
Partial Remission
|
9 Participants
|
|
Efficacy of CC-5013 in Myelofibrosis
Complete Remission
|
7 Participants
|
|
Efficacy of CC-5013 in Myelofibrosis
Hematologic Improvement
|
12 Participants
|
|
Efficacy of CC-5013 in Myelofibrosis
No Response
|
9 Participants
|
Adverse Events
CC-5013
Serious adverse events
| Measure |
CC-5013
n=41 participants at risk
10 mg orally daily
|
|---|---|
|
General disorders
Neutropenic fever
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Gastrointestinal disorders
Nausea
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Gastrointestinal disorders
Vomiting
|
2.4%
1/41 • Number of events 3 • 1 year and 2 months.
|
|
General disorders
Pain
|
4.9%
2/41 • Number of events 3 • 1 year and 2 months.
|
|
Infections and infestations
Infection
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Infections and infestations
Pneumonia
|
4.9%
2/41 • Number of events 2 • 1 year and 2 months.
|
|
General disorders
Disease Progression
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Infections and infestations
Infection with neutropenia
|
4.9%
2/41 • Number of events 2 • 1 year and 2 months.
|
|
Vascular disorders
Hemorrhage CNS
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
General disorders
Death
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Vascular disorders
Thrombosis
|
2.4%
1/41 • Number of events 2 • 1 year and 2 months.
|
|
Infections and infestations
Infection with unknown Absolute neutrophil count
|
7.3%
3/41 • Number of events 3 • 1 year and 2 months.
|
|
Cardiac disorders
Edema
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Cardiac disorders
Tachycardia
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Gastrointestinal disorders
Obstruction Gastrointestinal
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Hepatobiliary disorders
Cholecystitis
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
|
Nervous system disorders
Syncope, sensory neuropathy
|
2.4%
1/41 • Number of events 1 • 1 year and 2 months.
|
Other adverse events
| Measure |
CC-5013
n=41 participants at risk
10 mg orally daily
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
7.3%
3/41 • Number of events 3 • 1 year and 2 months.
|
|
Blood and lymphatic system disorders
Hypomagnesemia
|
7.3%
3/41 • Number of events 4 • 1 year and 2 months.
|
|
Infections and infestations
Infection
|
4.9%
2/41 • Number of events 3 • 1 year and 2 months.
|
|
Blood and lymphatic system disorders
Metabolic/Laboratory (other)
|
14.6%
6/41 • Number of events 6 • 1 year and 2 months.
|
|
Musculoskeletal and connective tissue disorders
muscle cramps
|
7.3%
3/41 • Number of events 3 • 1 year and 2 months.
|
|
Gastrointestinal disorders
Nausea
|
17.1%
7/41 • Number of events 8 • 1 year and 2 months.
|
|
Nervous system disorders
Neuropathy Sensory
|
14.6%
6/41 • Number of events 6 • 1 year and 2 months.
|
|
Infections and infestations
Neutrophils (ANC/AGC)
|
34.1%
14/41 • Number of events 14 • 1 year and 2 months.
|
|
General disorders
pain (headache)
|
4.9%
2/41 • Number of events 3 • 1 year and 2 months.
|
|
Blood and lymphatic system disorders
Platelets
|
26.8%
11/41 • Number of events 11 • 1 year and 2 months.
|
|
Skin and subcutaneous tissue disorders
pruritis
|
29.3%
12/41 • Number of events 13 • 1 year and 2 months.
|
|
Skin and subcutaneous tissue disorders
Rash/Desquamation
|
39.0%
16/41 • Number of events 21 • 1 year and 2 months.
|
|
Gastrointestinal disorders
Vomiting
|
7.3%
3/41 • Number of events 3 • 1 year and 2 months.
|
Additional Information
Jorge Cortes, M.D./ Professor
The University of Texas M. D. Anderson Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place