Trial Outcomes & Findings for Bortezomib for Low or Intermediate-1 Myelodysplastic Syndrome (MDS) With p65 Activation (NCT NCT01891968)
NCT ID: NCT01891968
Last Updated: 2018-04-23
Results Overview
Primary outcome is overall response (OR) including hematologic improvement defined by International Working Group (IWG), complete remission, partial remission and marrow complete remission.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
8 weeks
Results posted on
2018-04-23
Participant Flow
Recruitment Period: 8/2013to 01/2017
Of the 15 participants registered, all were treated with the study medication.
Participant milestones
| Measure |
Bortezomib
Bortezomib administered via subcutaneous route at a dose of 1.3 mg/m2 on days 1, 4, 8 and 11 of a 21 day cycle. A course of treatment will be 21 days.
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Bortezomib for Low or Intermediate-1 Myelodysplastic Syndrome (MDS) With p65 Activation
Baseline characteristics by cohort
| Measure |
Bortezomib
n=15 Participants
Bortezomib administered via subcutaneous route at a dose of 1.3 mg/m2 on days 1, 4, 8 and 11 of a 21 day cycle. A course of treatment will be 21 days.
|
|---|---|
|
Age, Continuous
|
71 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 8 weeksPrimary outcome is overall response (OR) including hematologic improvement defined by International Working Group (IWG), complete remission, partial remission and marrow complete remission.
Outcome measures
| Measure |
Bortezomib
n=15 Participants
Bortezomib administered via subcutaneous route at a dose of 1.3 mg/m2 on days 1, 4, 8 and 11 of a 21 day cycle. A course of treatment will be 21 days.
|
|---|---|
|
Overall Response (OR)
|
3 Participants
|
Adverse Events
Bortezomib
Serious events: 6 serious events
Other events: 15 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Bortezomib
n=15 participants at risk
Bortezomib administered via subcutaneous route at a dose of 1.3 mg/m2 on days 1, 4, 8 and 11 of a 21 day cycle. A course of treatment will be 21 days.
|
|---|---|
|
Cardiac disorders
Congestive Heart Failure
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Cardiac disorders
Hypotension
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Cardiac disorders
Stroke
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Cardiac disorders
Transient Ischemic Attack
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Renal and urinary disorders
Acute Kidney Injury
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Gastrointestinal disorders
Ascites
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Gastrointestinal disorders
Diarrhea
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Gastrointestinal disorders
Gastroenteritis
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Gastrointestinal disorders
Nausea/Vomiting
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Death
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Fatigue
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Fever
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Infections and infestations
Lung Infection
|
6.7%
1/15 • Number of events 3 • 2 years, 9 months
|
|
Infections and infestations
Neutropenic Fever
|
13.3%
2/15 • Number of events 2 • 2 years, 9 months
|
|
Infections and infestations
Septic Shock
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Infections and infestations
Pneumonia
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
Other adverse events
| Measure |
Bortezomib
n=15 participants at risk
Bortezomib administered via subcutaneous route at a dose of 1.3 mg/m2 on days 1, 4, 8 and 11 of a 21 day cycle. A course of treatment will be 21 days.
|
|---|---|
|
Eye disorders
Otitis
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Gastrointestinal disorders
Ascites
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Gastrointestinal disorders
Diarrhea
|
20.0%
3/15 • Number of events 3 • 2 years, 9 months
|
|
Gastrointestinal disorders
Nausea
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Edema
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Fatigue
|
13.3%
2/15 • Number of events 2 • 2 years, 9 months
|
|
General disorders
Fever
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Headache
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Irritability
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Pain, leg
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
General disorders
Pain, right flank
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Infections and infestations
Enteroccocus & pseudomans aeruginosa infections
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Metabolism and nutrition disorders
Hyperbilirubinemia
|
20.0%
3/15 • Number of events 3 • 2 years, 9 months
|
|
Metabolism and nutrition disorders
Elevated Creatinine
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Nervous system disorders
Burning Sensation Feet
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Nervous system disorders
Dizziness
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Nervous system disorders
Neuropathy
|
20.0%
3/15 • Number of events 3 • 2 years, 9 months
|
|
Nervous system disorders
Numbness and tingling in fingertips
|
13.3%
2/15 • Number of events 2 • 2 years, 9 months
|
|
Nervous system disorders
Paresthesia (lower extremities)
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Renal and urinary disorders
Urinary Tract Infection
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Skin and subcutaneous tissue disorders
Rash Macro-Papular trunk, legs, face
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Skin and subcutaneous tissue disorders
Redness abdomen
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
|
Skin and subcutaneous tissue disorders
Redness at Injection Site
|
13.3%
2/15 • Number of events 2 • 2 years, 9 months
|
|
Skin and subcutaneous tissue disorders
Skin Lesions
|
6.7%
1/15 • Number of events 1 • 2 years, 9 months
|
Additional Information
Guillermo Garcia-Manero, MD
The University of Texas MD Anderson Cancer Center
Phone: 713-745-3428
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place