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Trial record 42 of 183 for:    carfilzomib OR pr-171

Carfilzomib in Treating Patients With Chronic Graft-Versus-Host Disease

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ClinicalTrials.gov Identifier: NCT02491359
Recruitment Status : Completed
First Posted : July 8, 2015
Results First Posted : February 6, 2019
Last Update Posted : February 6, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Fred Hutchinson Cancer Research Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Chronic Graft Versus Host Disease
Interventions Drug: Carfilzomib
Other: Laboratory Biomarker Analysis
Other: Quality-of-Life Assessment
Other: Questionnaire Administration
Enrollment 20
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Period Title: Overall Study
Started 20
Completed 4
Not Completed 16
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Inter-Quartile Range)
Unit of measure:  Years
Number Analyzed 20 participants
53
(46 to 64)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Female
7
  35.0%
Male
13
  65.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Hispanic or Latino
3
  15.0%
Not Hispanic or Latino
17
  85.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
1
   5.0%
Black or African American
3
  15.0%
White
16
  80.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Primary Disease  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Acute leukemia (ALL/AML)
12
  60.0%
Chronic leukemia (CML/CLL)
4
  20.0%
Lymphoma (NHL/HD)
2
  10.0%
Myeloma
1
   5.0%
Myeloproliferative neoplasm
1
   5.0%
1.Primary Outcome
Title Incidence of Adverse Events
Hide Description according to National Cancer Institute CTCAE, version 4.03
Time Frame Up to 30 days following completion of study treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
Serious Adverse Events 8
Non-Serious Adverse Events 7
2.Primary Outcome
Title Probability of Treatment Failure at 6mo
Hide Description Kaplan-Meier estimate assessed at 6 months for treatment failure, defined as requirement of an additional line of systemic immune-suppressive therapy, recurrent malignancy, or death.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: probability of treatment failure
0.4
3.Secondary Outcome
Title Complete Response Rate
Hide Description Complete response (CR) at 6 months will be determined by both clinician-defined CR, as well as separately calculated according to the proposed response definitions of the NIH Consensus Conference.
Time Frame Up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who could be evaluated for response (not missing data)
Arm/Group Title Response Evaluated by MD Response Evaluated by NIH
Hide Arm/Group Description:
Participants who receive carfilzomib
Participants who receive carfilzomib
Overall Number of Participants Analyzed 16 16
Measure Type: Number
Unit of Measure: participants
partial response 5 4
mixed response 0 4
unchanged 2 1
progressive 2 0
failed 7 7
4.Secondary Outcome
Title Cumulative Incidence of Non-relapse Mortality and Primary Malignancy Relapse
Hide Description The cumulative incidence of non-relapse mortality (defined as death in the absence of primary malignancy relapse after transplant) and relapse (defined as hematologic relapse or any unplanned intervention to prevent progression of disease in patients with evidence (molecular, cytogenetic, flow cytometric, radiographic) of malignant disease after transplantation) will be estimated from time of study therapy initiation. These will be treated as competing-risk events, and estimated at 1 year.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Count of Participants
Unit of Measure: Participants
7
  35.0%
5.Secondary Outcome
Title Probability of Failure-free Survival at 1 Year
Hide Description Kaplain-Meier estimate assessed at 1 year for failure-free survival, defined as absence of death from any cause, relapse, or addition of secondary immune-suppressive agents.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: probability of failure-free survival
.32
6.Secondary Outcome
Title Impact of Proteasome Inhibition
Hide Description The biologic impact of proteasome inhibition in the treatment of chronic GVHD will be assessed at baseline, 3 and 6 months. The association between biologic outcome measures and clinical parameters (response, treatment failure, mortality) will be studied.
Time Frame Up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Data were not collected because biologic studies were not performed. Response to study drug too minimal to justify time and expense.
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Incidence of Discontinuation of All Systemic Immune-suppressive Therapies
Hide Description The incidence of complete discontinuation of all systemic immune-suppressive therapies will be determined at 1 year.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Count of Participants
Unit of Measure: Participants
2
  10.0%
8.Secondary Outcome
Title Overall Response Rate
Hide Description Overall response rate (ORR) (complete response + partial response) at 6 months will be determined by both clinician-defined categories of complete response and partial response, as well as separately calculated according to the proposed response definitions of the NIH Consensus Conference.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
Physician impression ORR 4
NIH ORR 3
9.Secondary Outcome
Title Probability of Overall Survival at 1 Year
Hide Description Kaplan-Meier estimate assessed at 1 year for overall survival, defined as absence of death from any cause.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: probability of overall survival
.65
10.Secondary Outcome
Title Treatment Success
Hide Description Treatment success will be estimated at 1 year with a composite outcome of complete resolution of all reversible chronic GVHD manifestations, discontinuation of all systemic immune suppressive agents, and freedom from death or primary malignancy relapse after transplant.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
0
11.Secondary Outcome
Title Use of Additional Systemic Immune-suppressive Therapies
Hide Description Addition of therapy after carfilzomib constitutes failure, could occur at any time from baseline to 12mo.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
13
12.Secondary Outcome
Title Symptoms as Measured by Patient Self-report--Short Form-36 (SF-36)
Hide Description SF-36 subscales have min=0 and max=100; results given are actual scores at 12mo, with higher scores indicating higher quality of life.
Time Frame baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Only baseline surveys analyzed. Only 7 patients completed surveys at 6mo, not analyzed due to low number.
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Median (Full Range)
Unit of Measure: units on a scale
Norm-based physical functioning
36
(14.9 to 52.8)
norm-based role-physical score
33.6
(17.7 to 54.4)
norm-based bodily pain score
41.4
(19.9 to 62.1)
norm-based general health score
32.9
(23.4 to 48.2)
norm-based vitality score
42.7
(30.2 to 64.6)
norm-based social functioning
40.5
(13.2 to 56.8)
norm-based role-emotional score
40.3
(17 to 55.9)
norm-based mental health score
50
(33.1 to 61.3)
standardized physical component score
37
(15.9 to 52.2)
standardized mental component score
49
(28.6 to 64.1)
13.Secondary Outcome
Title Symptoms as Measured by Patient Self-report--Functional Assessment of Chronic Illness Therapy (FACT)
Hide Description

FACT-BMT subscales have various min/max, see below; results given are actual 12mo scores, with higher scores indicating better functioning.

FACT physical well-being (0-28) FACT social/family well-being (0-28) FACT emotional well-being (0-24) FACT functional well-being (0-28) FACT Bone Marrow Transplant (BMT) subscale (0-40) FACT trial outcome index (0-96) FACT-General (G) (0-108) FACT-BMT total (0-148)

Time Frame baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Only baseline surveys analyzed. Only 7 patients completed surveys at 6mo, not analyzed due to low number.
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Median (Full Range)
Unit of Measure: units on a scale
physical well-being
21.5
(7 to 26)
social/family well-being
21.5
(15 to 28)
emotional well-being
18.5
(8 to 24)
functional well-being
16.5
(5 to 23)
BMT subscale
28
(21 to 33.3)
FACT-G
72.8
(53 to 98)
trial outcome index
65
(39 to 81)
FACT-BMT total
101.3
(75 to 131.3)
14.Secondary Outcome
Title Symptoms as Measured by Patient Self-report--Human Activities Profile (HAP)
Hide Description

HAP subscales have min=0 and max=94; results given are actual 12mo scores, with higher scores indicating better functioning.

Maximum Activity Score (MAS) is highest item number answered still doing. Represents highest oxygen demanding activity that respondent still performs.

Adjusted Activity Score (AAS) is MAS minus total number of stopped doing responses below MAS. A measure of usual daily activities.

Modified AAS is MAS minus total number of stopped doing responses below MAS but not penalized for not doing activities not permitted post transplant. The following items are not counted against the score:11,15,19,20,22,25,34,41,42,47,49,50,52,53,54,57,72,73,77,78.

Time Frame baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Only baseline surveys analyzed. Only 7 patients completed surveys at 6mo, not analyzed due to low number.
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Median (Full Range)
Unit of Measure: units on a scale
maximum activity score
66
(6 to 87)
adjusted activity score
57
(2 to 82)
modified adjusted activity score
60
(2 to 82)
15.Secondary Outcome
Title Symptoms as Measured by Patient Self-report--Lee Chronic GVHD Symptom Scale
Hide Description Lee symptom scale (LSS) has subscales with min=0, max=100; results given are 12mo scores, with higher numbers indicating higher symptom burden.
Time Frame baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Only baseline surveys analyzed. Only 7 patients completed surveys at 6mo, not analyzed due to low number.
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description:
Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 20
Median (Full Range)
Unit of Measure: units on a scale
skin scale
32.5
(0 to 100)
energy scale
42.9
(7.1 to 96.4)
lung scale
5
(0 to 30)
eye scale
62.5
(0 to 100)
nutrition scale
5
(0 to 20)
psychological scale
25
(0 to 66.7)
mouth scale
0
(0 to 75)
overall summary score
21.6
(8.2 to 49.3)
Time Frame 30 days after stopping study drug
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Treatment (Carfilzomib)
Hide Arm/Group Description Patients receive carfilzomib IV over approximately 30 minutes on days 1, 8, and 15. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
All-Cause Mortality
Treatment (Carfilzomib)
Affected / at Risk (%)
Total   2/20 (10.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Treatment (Carfilzomib)
Affected / at Risk (%) # Events
Total   8/20 (40.00%)    
Gastrointestinal disorders   
nausea/vomitting/diarrhea  1  1/20 (5.00%)  1
General disorders   
fever/infusion reaction  1  1/20 (5.00%)  1
Infections and infestations   
sepsis  1  2/20 (10.00%)  2
Injury, poisoning and procedural complications   
fall  1  1/20 (5.00%)  2
Renal and urinary disorders   
acute renal failure/sepsis  1  1/20 (5.00%)  1
Respiratory, thoracic and mediastinal disorders   
pneumothorax  1  1/20 (5.00%)  1
hypoxia  1  1/20 (5.00%)  1
pneumonia  1  1/20 (5.00%)  1
respiratory failure  1  1/20 (5.00%)  1
Skin and subcutaneous tissue disorders   
leg ulcers  1  1/20 (5.00%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Treatment (Carfilzomib)
Affected / at Risk (%) # Events
Total   7/20 (35.00%)    
Blood and lymphatic system disorders   
decreased lymphocyte count  1  2/20 (10.00%)  2
Gastrointestinal disorders   
diarrhea  1  1/20 (5.00%)  1
esophageal varices  1  1/20 (5.00%)  1
Hepatobiliary disorders   
increased ALT  1  1/20 (5.00%)  2
Infections and infestations   
fungal lung infection  1  1/20 (5.00%)  1
rhinovirus  1  1/20 (5.00%)  1
Metabolism and nutrition disorders   
hypokalemia  1  1/20 (5.00%)  1
intermittent hyperglycemia  1  1/20 (5.00%)  1
Musculoskeletal and connective tissue disorders   
Left hip hemiarthroplasty  1  1/20 (5.00%)  1
myositis  1  1/20 (5.00%)  1
Skin and subcutaneous tissue disorders   
bullous dermatitis  1  1/20 (5.00%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Joseph Pidala
Organization: H. Lee Moffitt Cancer Center
Phone: 813-745-2556
EMail: joseph.pidala@moffitt.org
Layout table for additonal information
Responsible Party: Fred Hutchinson Cancer Research Center
ClinicalTrials.gov Identifier: NCT02491359     History of Changes
Other Study ID Numbers: 9228
NCI-2015-00809 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
9228.00
9228 ( Other Identifier: Fred Hutch/University of Washington Cancer Consortium )
P30CA015704 ( U.S. NIH Grant/Contract )
First Submitted: June 10, 2015
First Posted: July 8, 2015
Results First Submitted: December 19, 2018
Results First Posted: February 6, 2019
Last Update Posted: February 6, 2019