Trial Outcomes & Findings for Pomalidomide, Ixazomib Citrate, and Dexamethasone in Treating Patients With Previously Treated Multiple Myeloma or Plasma Cell Leukemia (NCT NCT02547662)

Last Updated: 2023-10-30

Results Overview

A confirmed response is defined as a patient who has achieved a stringent complete response (sCR), complete response (CR), very good partial response (PR), or PR on two consecutive evaluations. The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

17 participants

Primary outcome timeframe

4 years

Results posted on

2023-10-30

Participant Flow

Study closed early due to recruitment concerns

Participant milestones

Participant milestones
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Overall Study STARTED	17
Overall Study COMPLETED	17
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pomalidomide, Ixazomib Citrate, and Dexamethasone in Treating Patients With Previously Treated Multiple Myeloma or Plasma Cell Leukemia

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) n=17 Participants Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Age, Continuous	64.2 years STANDARD_DEVIATION 10.0 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	17 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 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	16 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 4 years

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) n=17 Participants Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Confirmed Response Rate	0.06 proportion of patients Interval 0.0 to 0.29

SECONDARY outcome

Timeframe: 4 years

Population: All patients evaluated for biochemical response

The biochemical response rate will be estimated by the number of responders(patients that achieved partial or complete response) divided by the number of evaluable patients who have measurable disease by serum M-protein, urine M-protein, or serum FLC assay at baseline. Exact binomial confidence intervals will be calculated.

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) n=12 Participants Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Biochemical Response Rate	0.25 proportion of participants Interval 0.05 to 0.57

SECONDARY outcome

Timeframe: 4 years

Population: All patients with extramedullary disease that were evaluated for response.

Extramedullary response, defined as a response by extramedullary plasmacytoma or plasma cell count parameters. The extramedullary response rate will be estimated by the number of responders(patients that achieved a partial or complete response) divided by the number of evaluable patients. Exact binomial confidence intervals will be calculated.

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) n=5 Participants Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Extramedullary Response Rate	0.40 proportion of participants Interval 0.05 to 0.85

SECONDARY outcome

Timeframe: 4 years 1 month

Number of patients with adverse events is reported here, the full list of those adverse events and their frequency is reported in the adverse event section.

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) n=17 Participants Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Incidence of Adverse Events Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0	17 Participants

SECONDARY outcome

Timeframe: 4 years 8 months

The distribution of progression-free survival will be estimated using the method of Kaplan-Meier.

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) n=17 Participants Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Progression-free Survival	4.5 Months Interval 2.0 to 11.8

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5 years

The proportion of patients who achieve MRD negative status will be estimated by the number of patients who are MRD negative divided by the total number of evaluable patients who achieve a sCR or CR. Exact binomial 95% confidence intervals for the true MRD negative rate will be calculated.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone)

Serious events: 5 serious events

Other events: 17 other events

Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) n=17 participants at risk Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Blood and lymphatic system disorders Anemia	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
General disorders Death NOS	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Infections and infestations Infections and infestations - Oth spec	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Infections and infestations Lung infection	17.6% 3/17 • Number of events 4 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Infections and infestations Upper respiratory infection	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Injury, poisoning and procedural complications Ankle fracture	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Alanine aminotransferase increased	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Aspartate aminotransferase increased	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Creatinine increased	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Lymphocyte count decreased	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Platelet count decreased	17.6% 3/17 • Number of events 3 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Musculoskeletal and connective tissue disorders Back pain	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Musculoskeletal and connective tissue disorders Neck pain	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Musculoskeletal and connective tissue disorders Pain in extremity	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, mal, uncpec - Oth spec	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Nervous system disorders Encephalopathy	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Nervous system disorders Intracranial hemorrhage	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Psychiatric disorders Confusion	11.8% 2/17 • Number of events 2 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Respiratory, thoracic and mediastinal disorders Cough	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Respiratory, thoracic and mediastinal disorders Dyspnea	5.9% 1/17 • Number of events 2 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Respiratory, thoracic and mediastinal disorders Hypoxia	11.8% 2/17 • Number of events 2 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Respiratory, thoracic and mediastinal disorders Respiratory failure	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.

Other adverse events

Other adverse events
Measure	Treatment (Ixazomib Citrate, Pomalidomide, Dexamethasone) n=17 participants at risk Patients receive ixazomib citrate PO on days 1, 8, and 15, pomalidomide PO 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. Dexamethasone: Given PO Ixazomib Citrate: Given PO Laboratory Biomarker Analysis: Correlative studies Pomalidomide: Given PO
Blood and lymphatic system disorders Anemia	94.1% 16/17 • Number of events 47 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Eye disorders Eye disorders - Other, specify	5.9% 1/17 • Number of events 6 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Gastrointestinal disorders Constipation	58.8% 10/17 • Number of events 55 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Gastrointestinal disorders Diarrhea	29.4% 5/17 • Number of events 6 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Gastrointestinal disorders Nausea	52.9% 9/17 • Number of events 13 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
General disorders Edema limbs	11.8% 2/17 • Number of events 3 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
General disorders Fatigue	82.4% 14/17 • Number of events 102 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
General disorders Pain	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Infections and infestations Urinary tract infection	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Injury, poisoning and procedural complications Ankle fracture	5.9% 1/17 • Number of events 4 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Creatinine increased	41.2% 7/17 • Number of events 39 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Lymphocyte count decreased	23.5% 4/17 • Number of events 7 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Neutrophil count decreased	76.5% 13/17 • Number of events 40 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations Platelet count decreased	70.6% 12/17 • Number of events 41 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Investigations White blood cell decreased	76.5% 13/17 • Number of events 30 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Metabolism and nutrition disorders Hyperglycemia	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Musculoskeletal and connective tissue disorders Back pain	5.9% 1/17 • Number of events 4 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Musculoskeletal and connective tissue disorders Pain in extremity	5.9% 1/17 • Number of events 2 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Nervous system disorders Peripheral motor neuropathy	11.8% 2/17 • Number of events 2 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Nervous system disorders Peripheral sensory neuropathy	58.8% 10/17 • Number of events 79 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Nervous system disorders Tremor	5.9% 1/17 • Number of events 2 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.
Skin and subcutaneous tissue disorders Rash maculo-papular	5.9% 1/17 • Number of events 1 • Adverse events were followed for 4 years and 1 month, mortality was followed for 4 years and 8 months.

Additional Information

Shaji Kumar MD

Mayo Clinic

Phone: 507 284 2511

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place