Trial Outcomes & Findings for Carfilzomib in Refractory Renal Cell Carcinoma (RCC) (NCT NCT01775930)
NCT ID: NCT01775930
Last Updated: 2020-03-04
Results Overview
Progression free survival defined as time from enrollment to progression or death, whichever comes first. Progression defined according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. Any patients who are alive and free of disease at time of analysis censored at date of most recent tumor assessment.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
The number of months from enrollment to progression of cancer or death, whichever comes first up to 4 months
Results posted on
2020-03-04
Participant Flow
This was a single arm, single-center, non-randomized study of carfilzomib in participants with refractory ccRCC. Participants were accrued over a period of 195 days.
Participant milestones
| Measure |
Carfilzomib Treatment
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Carfilzomib Treatment
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Overall Study
Futility stopping boundary met
|
1
|
Baseline Characteristics
Carfilzomib in Refractory Renal Cell Carcinoma (RCC)
Baseline characteristics by cohort
| Measure |
Carfilzomib Treatment
n=9 Participants
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Age, Continuous
|
59 Years
n=99 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=99 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status (PS)
ECOG PS 0
|
6 Participants
n=99 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status (PS)
ECOG PS 1
|
2 Participants
n=99 Participants
|
|
Eastern Cooperative Oncology Group (ECOG) Performance Status (PS)
ECOG PS 2
|
1 Participants
n=99 Participants
|
|
Memorial Sloan Kettering Cancer Center (MSKCC) Risk Factor
Good
|
1 Participants
n=99 Participants
|
|
Memorial Sloan Kettering Cancer Center (MSKCC) Risk Factor
Intermediate
|
6 Participants
n=99 Participants
|
|
Memorial Sloan Kettering Cancer Center (MSKCC) Risk Factor
Poor
|
2 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: The number of months from enrollment to progression of cancer or death, whichever comes first up to 4 monthsProgression free survival defined as time from enrollment to progression or death, whichever comes first. Progression defined according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. Any patients who are alive and free of disease at time of analysis censored at date of most recent tumor assessment.
Outcome measures
| Measure |
Carfilzomib Treatment
n=9 Participants
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Progression Free Survival (PFS) of Carfilzomib Therapy in Participants With Refractory Or Intolerant to Prior Therapy
|
1.8 Months
Interval 0.08 to 3.6
|
SECONDARY outcome
Timeframe: Participants response was evaluated every 8 weeks from the first dose of carfilzomib until progression od disease (PD), up to 4 monthsThe number of participants had a complete response (CR, complete reduction in tumor burden) or partial response (PR, a reduction in tumor burden of at least 30%) as determined for radiographic imaging such as a CT scan. Participants who do not have a reduction in tumor burden will either have stable disease (SD) or progressive disease (PD, which is an increase in tumor burden of at least 20%). The results are based on the best response that each participant achieved while on treatment.
Outcome measures
| Measure |
Carfilzomib Treatment
n=9 Participants
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Overall Response Rate (ORR)
Complete response (CR)
|
0 Participants
|
|
Overall Response Rate (ORR)
Partial response (PR)
|
0 Participants
|
|
Overall Response Rate (ORR)
Stable disease (SD)
|
3 Participants
|
|
Overall Response Rate (ORR)
Progression of disease (PD)
|
6 Participants
|
SECONDARY outcome
Timeframe: 15 monthsThe number of months from the time of enrollment until death per participant
Outcome measures
| Measure |
Carfilzomib Treatment
n=9 Participants
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Overall Survival (OS)
|
13.2 Months
Interval 1.9 to 14.3
|
SECONDARY outcome
Timeframe: 4 monthsReason for stopping therapy
Outcome measures
| Measure |
Carfilzomib Treatment
n=9 Participants
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Safety of Carfilzomib
Progressive disease
|
9 Participants
|
|
Safety of Carfilzomib
Toxicity
|
0 Participants
|
|
Safety of Carfilzomib
Patient decision
|
0 Participants
|
SECONDARY outcome
Timeframe: No data collectedPopulation: The futility stopping rule was meet due to the low response rate to the treatment. None of our patients had a better response than stable disease (SD). Consequently the correlative analyses were not performed for VHL mutation subtype only for PFS and ORR which is reported as Outcomes.
Outcome measures
Outcome data not reported
Adverse Events
Carfilzomib Treatment
Serious events: 6 serious events
Other events: 8 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
Carfilzomib Treatment
n=9 participants at risk
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Renal and urinary disorders
Acute kidney injury
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Blood and lymphatic system disorders
Anemia
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Renal and urinary disorders
Creatinine increased
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
General disorders
Fatigue
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Vascular disorders
Hypertension
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Investigations
BUN elevated
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Blood and lymphatic system disorders
Lymphocyte count decreased
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Renal and urinary disorders
Proteinuria
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
Other adverse events
| Measure |
Carfilzomib Treatment
n=9 participants at risk
Dosed with 20 mg/mm2 infused on Day 1 and 2. Dosed with 56 mg/mm2 infused on Day 8, 9, 15, and 16.
|
|---|---|
|
Investigations
Alanine aminotransferase increased
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Investigations
Alkaline phosphatase increased
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Blood and lymphatic system disorders
Anemia
|
66.7%
6/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Anorexia
|
33.3%
3/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Investigations
Aspartate aminotransferase increased
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
General disorders
Chills
|
33.3%
3/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Gastrointestinal disorders
Constipation
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
3/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Renal and urinary disorders
Creatinine increased
|
66.7%
6/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Gastrointestinal disorders
Diarrhea
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
55.6%
5/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
General disorders
Edema
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
General disorders
Fatigue
|
33.3%
3/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
General disorders
Fever
|
33.3%
3/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Nervous system disorders
Headache
|
33.3%
3/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Renal and urinary disorders
Hemoglobinuria
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Vascular disorders
Hypertension
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
44.4%
4/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Investigations
BUN elevated
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Blood and lymphatic system disorders
Lymphocyte count decreased
|
66.7%
6/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Gastrointestinal disorders
Nausea
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Blood and lymphatic system disorders
Neutrophil count decreased
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Gastrointestinal disorders
Oral pain
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
General disorders
Pain
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Nervous system disorders
Paresthesia
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Blood and lymphatic system disorders
Platelet count decreased
|
44.4%
4/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
22.2%
2/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory infection
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
1/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
|
Blood and lymphatic system disorders
White blood cell decreased
|
44.4%
4/9 • Adverse events will be followed for 30 days beyond discontinuation of treatment for any reason; duration of treatment up to 4 months. All-Cause Mortality will be assessed up to 15 months.
|
Additional Information
Dr. Eric Jonasch, MD / Professor, Genitourinary Medical Oncology
UT MD Anderson Cancer Center
Phone: 713- 563-7232
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place