Trial Outcomes & Findings for CEP-701 for PH-negative Myelofibrosis (NCT NCT00494585)
NCT ID: NCT00494585
Last Updated: 2012-06-25
Results Overview
Objective response = Complete Response, absence sign/symptoms of disease (without use of growth factors, hydroxyurea, anagrelide, or transfusions for \> 1 month); Partial Response, absence of progressive disease (PD), and improvement in 2+ parameters (if abnormal): Absolute neutrophil count (ANC), hemoglobin, platelets, transfusions, splenomegaly, or bone marrow blasts; Clinical Improvement, absence of PD, and improvement in 1 parameter: ANC, hemoglobin, platelets, transfusions, splenomegaly, or bone marrow blasts). \[International Working Group on Myelofibrosis Research and Treatment\]
COMPLETED
PHASE2
27 participants
Response assessed after each 3 cycles (cycle = 30 days)
2012-06-25
Participant Flow
Recruitment Period: 6/28/2007 through 10/25/2007. All participants recruited at UT MD Anderson Cancer Center.
Of the 27 participants registered, 5 participants were ineligible to participate.
Participant milestones
| Measure |
CEP-701
80 mg orally twice daily for 30 days
|
|---|---|
|
Overall Study
STARTED
|
22
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
CEP-701 for PH-negative Myelofibrosis
Baseline characteristics by cohort
| Measure |
CEP-701
n=22 Participants
80 mg orally twice daily for 30 days
|
|---|---|
|
Age Continuous
|
61 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
16 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
22 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Response assessed after each 3 cycles (cycle = 30 days)Population: Intention to treat.
Objective response = Complete Response, absence sign/symptoms of disease (without use of growth factors, hydroxyurea, anagrelide, or transfusions for \> 1 month); Partial Response, absence of progressive disease (PD), and improvement in 2+ parameters (if abnormal): Absolute neutrophil count (ANC), hemoglobin, platelets, transfusions, splenomegaly, or bone marrow blasts; Clinical Improvement, absence of PD, and improvement in 1 parameter: ANC, hemoglobin, platelets, transfusions, splenomegaly, or bone marrow blasts). \[International Working Group on Myelofibrosis Research and Treatment\]
Outcome measures
| Measure |
CEP-701
n=22 Participants
80 mg orally twice daily for 30 days
|
|---|---|
|
Number of Participants With Objective Response
Complete Response (CR)
|
0 Participants
|
|
Number of Participants With Objective Response
Partial Response (PR)
|
0 Participants
|
|
Number of Participants With Objective Response
Clinical Improvement (CI)
|
6 Participants
|
Adverse Events
CEP-701
Serious adverse events
| Measure |
CEP-701
n=22 participants at risk
80 mg orally twice daily for 30 days
|
|---|---|
|
Blood and lymphatic system disorders
Hematoma
|
4.5%
1/22 • Number of events 1 • 3 years 10 months
|
|
General disorders
Debridement surgery with VAC placement
|
4.5%
1/22 • Number of events 1 • 3 years 10 months
|
|
Blood and lymphatic system disorders
Hemorrhage
|
4.5%
1/22 • Number of events 1 • 3 years 10 months
|
|
General disorders
Death
|
18.2%
4/22 • Number of events 4 • 3 years 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Chronic Interstitial Pulmonary Fibrosis
|
4.5%
1/22 • Number of events 1 • 3 years 10 months
|
|
General disorders
Dehydration
|
4.5%
1/22 • Number of events 1 • 3 years 10 months
|
|
Infections and infestations
Infection
|
4.5%
1/22 • Number of events 1 • 3 years 10 months
|
Other adverse events
| Measure |
CEP-701
n=22 participants at risk
80 mg orally twice daily for 30 days
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
27.3%
6/22 • Number of events 6 • 3 years 10 months
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
22.7%
5/22 • Number of events 5 • 3 years 10 months
|
|
Gastrointestinal disorders
Diarrhea
|
72.7%
16/22 • Number of events 16 • 3 years 10 months
|
|
Gastrointestinal disorders
Nausea
|
50.0%
11/22 • Number of events 11 • 3 years 10 months
|
|
Nervous system disorders
Headache
|
31.8%
7/22 • Number of events 7 • 3 years 10 months
|
|
Gastrointestinal disorders
Vomiting
|
27.3%
6/22 • Number of events 6 • 3 years 10 months
|
|
Gastrointestinal disorders
Flatulence
|
22.7%
5/22 • Number of events 5 • 3 years 10 months
|
|
Gastrointestinal disorders
Heartburn
|
18.2%
4/22 • Number of events 4 • 3 years 10 months
|
|
Gastrointestinal disorders
Mucositis
|
13.6%
3/22 • Number of events 3 • 3 years 10 months
|
|
Nervous system disorders
Peripheral Neuropathy
|
13.6%
3/22 • Number of events 3 • 3 years 10 months
|
|
Gastrointestinal disorders
Anorexia
|
9.1%
2/22 • Number of events 2 • 3 years 10 months
|
|
General disorders
Fatigue
|
9.1%
2/22 • Number of events 2 • 3 years 10 months
|
|
Hepatobiliary disorders
Elevated aspartate aminotransferase
|
27.3%
6/22 • Number of events 6 • 3 years 10 months
|
|
Hepatobiliary disorders
Elevated alanine aminotransferase
|
27.3%
6/22 • Number of events 6 • 3 years 10 months
|
|
Hepatobiliary disorders
Elevated alkaline phosphatase
|
9.1%
2/22 • Number of events 2 • 3 years 10 months
|
|
Hepatobiliary disorders
Hyperbilirubinemia
|
9.1%
2/22 • Number of events 2 • 3 years 10 months
|
Additional Information
Srdan Verstovsek M.D./Associate Professor
The University of Texas M. D. Anderson Cancer Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place