Trial Outcomes & Findings for Carfilzomib in Treating Patients With Multiple Myeloma in First Relapse or Refractory to First-Line Therapy (NCT NCT02020941)
NCT ID: NCT02020941
Last Updated: 2016-11-02
Results Overview
: Evaluate the overall response rate of patients receiving therapy. Patients are considered as having a response if their overall response is Partial Response or better. The percentage of patients achieving this and the exact 95% confidence interval will be calculated. Responses will be defined using the International Myeloma Working Group-Uniform Response Criteria (IMWG-URC).
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
At 32 weeks
Results posted on
2016-11-02
Participant Flow
This protocol was based on enrolling 23 patients. Due to slow accrual, the study was closed with 10 patients enrolled.
Participant milestones
| Measure |
Treatment (Carfilzomib, Dexamethasone)
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Treatment (Carfilzomib, Dexamethasone)
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Physician Decision
|
2
|
|
Overall Study
Withdrawal by Subject
|
1
|
|
Overall Study
Disease Progression
|
4
|
Baseline Characteristics
Carfilzomib in Treating Patients With Multiple Myeloma in First Relapse or Refractory to First-Line Therapy
Baseline characteristics by cohort
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=10 Participants
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
9 Participants
n=5 Participants
|
|
Age, Continuous
|
70.1 years
STANDARD_DEVIATION 6.79 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 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
|
0 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
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At 32 weeksPopulation: All patients receiving at least one dose of study drug and having at least one evaluable post-baseline visit
: Evaluate the overall response rate of patients receiving therapy. Patients are considered as having a response if their overall response is Partial Response or better. The percentage of patients achieving this and the exact 95% confidence interval will be calculated. Responses will be defined using the International Myeloma Working Group-Uniform Response Criteria (IMWG-URC).
Outcome measures
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=9 Participants
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Overall Response Rate (ORR) After 8 Courses of Treatment
|
77.8 percentage of participants
Interval 40.0 to 97.2
|
SECONDARY outcome
Timeframe: At 16 weeksPopulation: All patients receiving at least one dose of study drug and having at least one evaluable post-baseline visit
Evaluate the overall response rate of patients receiving therapy. Patients are considered as having a response if their overall response is Partial Response or better. The percentage of patients achieving this and the exact 95% confidence interval will be calculated. Responses will be defined using the International Myeloma Working Group-Uniform Response Criteria (IMWG-URC).
Outcome measures
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=9 Participants
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Overall Response Rate (ORR) After 4 Courses of Treatment
|
77.8 percentage of participants
Interval 40.0 to 97.2
|
SECONDARY outcome
Timeframe: Time from first dose to first observed disease progression or death, assessed up to 2 yearsPopulation: All patients enrolled and received treatment.
Analysis will be performed using Kaplan-Meier estimates. Time from date on treatment to date of progression for patients who progressed or date of death for patients who died without progressing. The observations of patients remaining alive and progression free were censored at date of last disease evaluation.
Outcome measures
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=10 Participants
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Progression-free Survival (PFS)
|
11.5 months
Interval 2.9 to 16.4
|
SECONDARY outcome
Timeframe: Time from first dose to disease progression, assessed up to 2 yearsPopulation: All patients enrolled and received treatment.
Analysis will be performed using Kaplan-Meier estimates. Time from date on treatment to date of progression. The observations of patients who died or remained alive and progression free were censored at date of death or last disease evaluation, respectively.
Outcome measures
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=10 Participants
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Time to Progression (TTP)
|
11.5 months
Interval 2.9 to 16.4
|
SECONDARY outcome
Timeframe: Time from first evidence of PR or better to disease progression or death, assessed up to 2 yearsPopulation: All patients who had a response of partial response or better
Analysis will be performed using Kaplan-Meier estimates. Time from date of first confirmed response of partial response or better to date of progression or death. Only patients who had a response of partial response or better will be included in this analysis.
Outcome measures
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=7 Participants
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Duration of Response (DOR)
|
8.9 months
Interval 0.1 to 12.0
|
SECONDARY outcome
Timeframe: Up to 30 days after completion of study treatment, up to 2 yearsPopulation: All patients enrolled and received treatment.
Number of unique patients who had a treatment related (possible, probable or definite) adverse events that were graded 3 or greater.
Outcome measures
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=10 Participants
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Treatment Related Adverse Events Grade 3 or Higher
|
5 participants
|
Adverse Events
Treatment (Carfilzomib, Dexamethasone)
Serious events: 2 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=10 participants at risk
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Cardiac disorders
Heart Failure
|
10.0%
1/10 • up to 2 years
|
|
Infections and infestations
Lung infection
|
10.0%
1/10 • up to 2 years
|
|
Injury, poisoning and procedural complications
Hip fracture
|
10.0%
1/10 • up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
10.0%
1/10 • up to 2 years
|
Other adverse events
| Measure |
Treatment (Carfilzomib, Dexamethasone)
n=10 participants at risk
TREATMENT PHASE (COURSES 1-8): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 8, 9, 15, and 16. Treatment repeats every 28 days for 8 courses in the absence of disease progression or unacceptable toxicity. Patients achieving less than PR also receive dexamethasone PO or IV weekly in courses 4-8.
MAINTENANCE PHASE (COURSES 9-14): Patients receive carfilzomib IV over 30 minutes on days 1, 2, 15, and 16. Patients who received dexamethasone in the Treatment Phase continue to receive dexamethasone PO or IV weekly. Treatment repeats every 28 days for 6 courses in the absence of disease progression or unacceptable toxicity.
carfilzomib: Given IV
dexamethasone: Given IV or PO
laboratory biomarker analysis: Correlative studies
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
10.0%
1/10 • up to 2 years
|
|
Cardiac disorders
Atrial fibrillation
|
10.0%
1/10 • up to 2 years
|
|
Cardiac disorders
Heart failure
|
10.0%
1/10 • up to 2 years
|
|
Ear and labyrinth disorders
Ear pain
|
10.0%
1/10 • up to 2 years
|
|
Ear and labyrinth disorders
Tinnitus
|
10.0%
1/10 • up to 2 years
|
|
Eye disorders
Conjunctivitis
|
10.0%
1/10 • up to 2 years
|
|
Gastrointestinal disorders
Abdominal pain
|
20.0%
2/10 • up to 2 years
|
|
Gastrointestinal disorders
Bloating
|
20.0%
2/10 • up to 2 years
|
|
Gastrointestinal disorders
Constipation
|
10.0%
1/10 • up to 2 years
|
|
Gastrointestinal disorders
Dental caries
|
10.0%
1/10 • up to 2 years
|
|
Gastrointestinal disorders
Diarrhea
|
40.0%
4/10 • up to 2 years
|
|
Gastrointestinal disorders
Flatulence
|
10.0%
1/10 • up to 2 years
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
10.0%
1/10 • up to 2 years
|
|
Gastrointestinal disorders
Nausea
|
40.0%
4/10 • up to 2 years
|
|
Gastrointestinal disorders
Upper gastrointestinal hemorrhage
|
10.0%
1/10 • up to 2 years
|
|
Gastrointestinal disorders
Vomiting
|
30.0%
3/10 • up to 2 years
|
|
General disorders
Chills
|
10.0%
1/10 • up to 2 years
|
|
General disorders
Edema limbs
|
40.0%
4/10 • up to 2 years
|
|
General disorders
Fatigue
|
30.0%
3/10 • up to 2 years
|
|
General disorders
Fever
|
40.0%
4/10 • up to 2 years
|
|
General disorders
Flu like symptoms
|
40.0%
4/10 • up to 2 years
|
|
General disorders
Infusion related reaction
|
30.0%
3/10 • up to 2 years
|
|
General disorders
Irritability
|
10.0%
1/10 • up to 2 years
|
|
General disorders
Pain
|
10.0%
1/10 • up to 2 years
|
|
Infections and infestations
Bronchial infection
|
10.0%
1/10 • up to 2 years
|
|
Infections and infestations
Gum infection
|
10.0%
1/10 • up to 2 years
|
|
Infections and infestations
Upper respiratory infection
|
40.0%
4/10 • up to 2 years
|
|
Injury, poisoning and procedural complications
Fall
|
10.0%
1/10 • up to 2 years
|
|
Investigations
Neutrophil count decreased
|
10.0%
1/10 • up to 2 years
|
|
Investigations
Platelet count decreased
|
10.0%
1/10 • up to 2 years
|
|
Metabolism and nutrition disorders
Anorexia
|
10.0%
1/10 • up to 2 years
|
|
Metabolism and nutrition disorders
Glucose intolerance
|
10.0%
1/10 • up to 2 years
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
10.0%
1/10 • up to 2 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
30.0%
3/10 • up to 2 years
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
10.0%
1/10 • up to 2 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.0%
1/10 • up to 2 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other
|
10.0%
1/10 • up to 2 years
|
|
Nervous system disorders
Ataxia
|
10.0%
1/10 • up to 2 years
|
|
Nervous system disorders
Cognitive disturbance
|
10.0%
1/10 • up to 2 years
|
|
Nervous system disorders
Dizziness
|
20.0%
2/10 • up to 2 years
|
|
Nervous system disorders
Headache
|
20.0%
2/10 • up to 2 years
|
|
Nervous system disorders
Nervous system disorders - Other
|
10.0%
1/10 • up to 2 years
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
20.0%
2/10 • up to 2 years
|
|
Nervous system disorders
Tremor
|
10.0%
1/10 • up to 2 years
|
|
Psychiatric disorders
Depression
|
20.0%
2/10 • up to 2 years
|
|
Psychiatric disorders
Insomnia
|
10.0%
1/10 • up to 2 years
|
|
Renal and urinary disorders
Urinary retention
|
10.0%
1/10 • up to 2 years
|
|
Reproductive system and breast disorders
Pelvic pain
|
10.0%
1/10 • up to 2 years
|
|
Reproductive system and breast disorders
Reproductive system and breast disorders - Other
|
10.0%
1/10 • up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
30.0%
3/10 • up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
30.0%
3/10 • up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
10.0%
1/10 • up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
10.0%
1/10 • up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.0%
1/10 • up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
10.0%
1/10 • up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Voice alteration
|
10.0%
1/10 • up to 2 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.0%
2/10 • up to 2 years
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
30.0%
3/10 • up to 2 years
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
10.0%
1/10 • up to 2 years
|
|
Surgical and medical procedures
Surgical and medical procedures - Other
|
10.0%
1/10 • up to 2 years
|
|
Vascular disorders
Flushing
|
10.0%
1/10 • up to 2 years
|
|
Vascular disorders
Hypotension
|
20.0%
2/10 • up to 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place