Trial Outcomes & Findings for Pomalidomide or Lenalidomide and Dexamethasone in Treating Patients With Relapsed or Refractory Multiple Myeloma Previously Treated With Lenalidomide (NCT NCT01794039)

NCT ID: NCT01794039

Last Updated: 2018-08-21

Results Overview

The proportion of successes will be estimated in each arm independently by the number of successes divided by the total number of evaluable patients. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner. A confirmed tumor response is defined to be a partial response or better noted as the objective status on two consecutive evaluations while receiving lenalidomide and dexmethasone (Arm A) or pomalidomide and dexamethasone (Arm B). All patients meeting the eligibility criteria who have signed a consent form and have begun treatment will be evaluable for response.The International Myeloma Working Group (IMWG) uniform response criteria (Rajkumar et al, 2011) will be used to assess response to therapy.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

Up to 2 years

Results posted on

2018-08-21

Participant Flow

Participant milestones

Participant milestones
Measure
Arm A (Lenalidomide, Dexamethasone)
Patients receive lenalidomide PO daily on days 1-21 and dexamethasone PO on days 1, 8, 15, and 22. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients experiencing progressive disease may crossover to arm B.\> \> Dexamethasone: Given PO\> \> Lenalidomide: Given PO
Arm B (Pomalidomide, Dexamethasone)
Patients receive pomalidomide PO daily on days 1-21 and dexamethasone as in arm A. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.\> \> Dexamethasone: Given PO\> \> Pomalidomide: Given PO
Overall Study
STARTED
5
4
Overall Study
COMPLETED
5
4
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pomalidomide or Lenalidomide and Dexamethasone in Treating Patients With Relapsed or Refractory Multiple Myeloma Previously Treated With Lenalidomide

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Arm A (Lenalidomide, Dexamethasone)
n=5 Participants
Patients receive lenalidomide PO daily on days 1-21 and dexamethasone PO on days 1, 8, 15, and 22. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients experiencing progressive disease may crossover to arm B.\> \> Dexamethasone: Given PO\> \> Lenalidomide: Given PO
Arm B (Pomalidomide, Dexamethasone)
n=4 Participants
Patients receive pomalidomide PO daily on days 1-21 and dexamethasone as in arm A. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.\> \> Dexamethasone: Given PO\> \> Pomalidomide: Given PO
Total
n=9 Participants
Total of all reporting groups
Age, Continuous
67 years
n=99 Participants
68.5 years
n=107 Participants
67 years
n=206 Participants
Sex: Female, Male
Female
2 Participants
n=99 Participants
1 Participants
n=107 Participants
3 Participants
n=206 Participants
Sex: Female, Male
Male
3 Participants
n=99 Participants
3 Participants
n=107 Participants
6 Participants
n=206 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Asian
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
White
5 Participants
n=99 Participants
4 Participants
n=107 Participants
9 Participants
n=206 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants

PRIMARY outcome

Timeframe: Up to 2 years

Population: All patients that received treatment were evaluable for response

The proportion of successes will be estimated in each arm independently by the number of successes divided by the total number of evaluable patients. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner. A confirmed tumor response is defined to be a partial response or better noted as the objective status on two consecutive evaluations while receiving lenalidomide and dexmethasone (Arm A) or pomalidomide and dexamethasone (Arm B). All patients meeting the eligibility criteria who have signed a consent form and have begun treatment will be evaluable for response.The International Myeloma Working Group (IMWG) uniform response criteria (Rajkumar et al, 2011) will be used to assess response to therapy.

Outcome measures

Outcome measures
Measure
Arm A (Lenalidomide, Dexamethasone)
n=5 Participants
Patients receive lenalidomide PO daily on days 1-21 and dexamethasone PO on days 1, 8, 15, and 22. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients experiencing progressive disease may crossover to arm B. \> \> Dexamethasone: Given PO \> \> Lenalidomide: Given PO
Arm B (Pomalidomide, Dexamethasone)
n=4 Participants
Patients receive pomalidomide PO daily on days 1-21 and dexamethasone as in arm A. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Dexamethasone: Given PO \> \> Pomalidomide: Given PO
Proportion of Confirmed Tumor Responses Defined to be a Partial Response or Better Noted as the Objective Status on Two Consecutive Evaluations
.4 proportion of participants
Interval 0.05 to 0.85
.25 proportion of participants
Interval 0.006 to 0.81

SECONDARY outcome

Timeframe: Up to 30 days after last day of study drug treatment

Population: All treated patients are evaluable for Adverse Events

Recorded and reported for each patient, and frequency tables will be reviewed to determine adverse event patterns. These results are reported in the Adverse Events section of this CT.gov report.

Outcome measures

Outcome measures
Measure
Arm A (Lenalidomide, Dexamethasone)
n=5 Participants
Patients receive lenalidomide PO daily on days 1-21 and dexamethasone PO on days 1, 8, 15, and 22. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients experiencing progressive disease may crossover to arm B. \> \> Dexamethasone: Given PO \> \> Lenalidomide: Given PO
Arm B (Pomalidomide, Dexamethasone)
n=4 Participants
Patients receive pomalidomide PO daily on days 1-21 and dexamethasone as in arm A. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Dexamethasone: Given PO \> \> Pomalidomide: Given PO
Number of Participants With Adverse Events, Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0
5 Participants
4 Participants

SECONDARY outcome

Timeframe: Time from registration to death due to any cause, assessed up to 2 years

Population: All patients that received Arm A or Arm B treatment.

The distribution of survival time will be estimated using the method of Kaplan-Meier. Due to an early closer from slow accrual the data from both arms was combined in survival analysis.

Outcome measures

Outcome measures
Measure
Arm A (Lenalidomide, Dexamethasone)
n=9 Participants
Patients receive lenalidomide PO daily on days 1-21 and dexamethasone PO on days 1, 8, 15, and 22. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients experiencing progressive disease may crossover to arm B. \> \> Dexamethasone: Given PO \> \> Lenalidomide: Given PO
Arm B (Pomalidomide, Dexamethasone)
Patients receive pomalidomide PO daily on days 1-21 and dexamethasone as in arm A. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Dexamethasone: Given PO \> \> Pomalidomide: Given PO
Overall Survival
13.4 Months
Interval 9.3 to
Upper confidence interval was not reached

SECONDARY outcome

Timeframe: Time from registration to the earliest date with documentation of disease progression, assessed up to 2 years

Population: All patients that received Arm A or Arm B treatment.

The distribution of time to progression will be estimated using the method of Kaplan-Meier. The International Myeloma Working Group (IMWG) uniform response criteria (Rajkumar et al, 2011) will be used to assess response to therapy.

Outcome measures

Outcome measures
Measure
Arm A (Lenalidomide, Dexamethasone)
n=9 Participants
Patients receive lenalidomide PO daily on days 1-21 and dexamethasone PO on days 1, 8, 15, and 22. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients experiencing progressive disease may crossover to arm B. \> \> Dexamethasone: Given PO \> \> Lenalidomide: Given PO
Arm B (Pomalidomide, Dexamethasone)
Patients receive pomalidomide PO daily on days 1-21 and dexamethasone as in arm A. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Dexamethasone: Given PO \> \> Pomalidomide: Given PO
Time to Progression
10 Months
Interval 4.1 to 12.4

Adverse Events

Arm A (Lenalidomide, Dexamethasone)

Serious events: 3 serious events
Other events: 5 other events
Deaths: 0 deaths

Arm B (Pomalidomide, Dexamethasone)

Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Arm A (Lenalidomide, Dexamethasone)
n=5 participants at risk
Lenalidomide: Given PO
Arm B (Pomalidomide, Dexamethasone)
n=4 participants at risk
Pomalidomide: Given PO
Blood and lymphatic system disorders
Anemia
0.00%
0/5
25.0%
1/4 • Number of events 1
Blood and lymphatic system disorders
Febrile neutropenia
0.00%
0/5
25.0%
1/4 • Number of events 1
Cardiac disorders
Acute coronary syndrome
20.0%
1/5 • Number of events 1
0.00%
0/4
Cardiac disorders
Atrial fibrillation
0.00%
0/5
25.0%
1/4 • Number of events 1
Infections and infestations
Lung infection
40.0%
2/5 • Number of events 2
25.0%
1/4 • Number of events 1
Infections and infestations
Sepsis
0.00%
0/5
25.0%
1/4 • Number of events 2
Infections and infestations
Upper respiratory infection
0.00%
0/5
25.0%
1/4 • Number of events 1
Investigations
Lymphocyte count decreased
20.0%
1/5 • Number of events 1
0.00%
0/4
Investigations
Neutrophil count decreased
20.0%
1/5 • Number of events 1
0.00%
0/4
Investigations
Platelet count decreased
20.0%
1/5 • Number of events 1
0.00%
0/4
Investigations
White blood cell decreased
20.0%
1/5 • Number of events 1
0.00%
0/4
Metabolism and nutrition disorders
Hyperglycemia
20.0%
1/5 • Number of events 1
0.00%
0/4
Musculoskeletal and connective tissue disorders
Pain in extremity
0.00%
0/5
25.0%
1/4 • Number of events 1
Vascular disorders
Thromboembolic event
20.0%
1/5 • Number of events 1
0.00%
0/4

Other adverse events

Other adverse events
Measure
Arm A (Lenalidomide, Dexamethasone)
n=5 participants at risk
Lenalidomide: Given PO
Arm B (Pomalidomide, Dexamethasone)
n=4 participants at risk
Pomalidomide: Given PO
Blood and lymphatic system disorders
Anemia
40.0%
2/5 • Number of events 5
25.0%
1/4 • Number of events 2
Cardiac disorders
Aortic valve disease
0.00%
0/5
25.0%
1/4 • Number of events 1
Gastrointestinal disorders
Constipation
20.0%
1/5 • Number of events 5
50.0%
2/4 • Number of events 4
Gastrointestinal disorders
Diarrhea
80.0%
4/5 • Number of events 23
50.0%
2/4 • Number of events 5
Gastrointestinal disorders
Nausea
0.00%
0/5
25.0%
1/4 • Number of events 1
General disorders
Edema limbs
40.0%
2/5 • Number of events 4
50.0%
2/4 • Number of events 5
General disorders
Fatigue
100.0%
5/5 • Number of events 36
75.0%
3/4 • Number of events 15
Infections and infestations
Infections and infestations - Oth spec
20.0%
1/5 • Number of events 1
0.00%
0/4
Investigations
Lymphocyte count decreased
80.0%
4/5 • Number of events 25
50.0%
2/4 • Number of events 2
Investigations
Neutrophil count decreased
100.0%
5/5 • Number of events 18
50.0%
2/4 • Number of events 7
Investigations
Platelet count decreased
80.0%
4/5 • Number of events 30
50.0%
2/4 • Number of events 7
Investigations
White blood cell decreased
100.0%
5/5 • Number of events 24
75.0%
3/4 • Number of events 8
Metabolism and nutrition disorders
Hyperglycemia
0.00%
0/5
25.0%
1/4 • Number of events 1
Musculoskeletal and connective tissue disorders
Back pain
0.00%
0/5
25.0%
1/4 • Number of events 1
Musculoskeletal and connective tissue disorders
Myalgia
20.0%
1/5 • Number of events 2
0.00%
0/4
Nervous system disorders
Peripheral sensory neuropathy
100.0%
5/5 • Number of events 46
75.0%
3/4 • Number of events 15
Nervous system disorders
Tremor
0.00%
0/5
25.0%
1/4 • Number of events 9
Psychiatric disorders
Agitation
20.0%
1/5 • Number of events 5
0.00%
0/4
Skin and subcutaneous tissue disorders
Hyperhidrosis
20.0%
1/5 • Number of events 2
0.00%
0/4
Skin and subcutaneous tissue disorders
Hypohidrosis
20.0%
1/5 • Number of events 3
0.00%
0/4
Skin and subcutaneous tissue disorders
Rash maculo-papular
20.0%
1/5 • Number of events 2
25.0%
1/4 • Number of events 1

Additional Information

Shaji K. Kumar, M.D.

Mayo Clinic

Phone: (507) 284-2017

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place