Trial Outcomes & Findings for Ixazomib Citrate, Pomalidomide, Dexamethasone, Stem Cell Transplant in Treating Relapsed or Refractory Multiple Myeloma (NCT NCT03202628)

NCT ID: NCT03202628

Last Updated: 2023-09-21

Results Overview

The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for the true success proportion will be calculated. If patients are censored prior to 18 months post registration, a Kaplan Meier (Kaplan, E. and Meier, P., 1958) estimate for PFS18 along with the 95% confidence intervals will be reported.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

18 months

Results posted on

2023-09-21

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) INDUCTION (COURSES 1-4): 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. Treatment repeats every 28 days (courses 1-3) and 56 days (course 4) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> TRANSPLANTATION (COURSE 5): Between 2-4 weeks following Induction, patients undergo ASCT. \> \> CONSOLIDATION (COURSES 6-9): Beginning 60-120 days following ASCT, patients receive ixazomib citrate, pomalidomide, and dexamethasone as in Induction. Treatment repeats every 28 days (courses 6-8) and 56 days (course 9) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> MAINTENANCE (COURSES 10+): Beginning 0-4 weeks following Consolidation, patients receive ixazomib citrate as in Induction. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Autologous Hematopoietic Stem Cell Transplantation: Undergo ASCT \> \> Dexamethasone: Given PO \> \> Ixazomib Citrate: Given PO \> \> Laboratory Biomarker Analysis: Correlative studies \> \> Pomalidomide: Given PO
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Ixazomib Citrate, Pomalidomide, Dexamethasone, Stem Cell Transplant in Treating Relapsed or Refractory Multiple Myeloma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) n=8 Participants INDUCTION (COURSES 1-4): 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. Treatment repeats every 28 days (courses 1-3) and 56 days (course 4) for up to 4 courses in the absence of disease progression or unacceptable toxicity.\> \> TRANSPLANTATION (COURSE 5): Between 2-4 weeks following Induction, patients undergo ASCT.\> \> CONSOLIDATION (COURSES 6-9): Beginning 60-120 days following ASCT, patients receive ixazomib citrate, pomalidomide, and dexamethasone as in Induction. Treatment repeats every 28 days (courses 6-8) and 56 days (course 9) for up to 4 courses in the absence of disease progression or unacceptable toxicity.\> \> MAINTENANCE (COURSES 10+): Beginning 0-4 weeks following Consolidation, patients receive ixazomib citrate as in Induction. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.\> \> Autologous Hematopoietic Stem Cell Transplantation: Undergo ASCT\> \> Dexamethasone: Given PO\> \> Ixazomib Citrate: Given PO\> \> Laboratory Biomarker Analysis: Correlative studies\> \> Pomalidomide: Given PO
Age, Continuous	63.5 years STANDARD_DEVIATION 11.17 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 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	8 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
Region of Enrollment United States	8 participants n=5 Participants
Revised international staging system (R-ISS) myeloma stage at initial diagnosis Stage I	2 Participants n=5 Participants
Revised international staging system (R-ISS) myeloma stage at initial diagnosis Stage II	5 Participants n=5 Participants
Revised international staging system (R-ISS) myeloma stage at initial diagnosis Unknown	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 18 months

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) n=8 Participants INDUCTION (COURSES 1-4): 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. Treatment repeats every 28 days (courses 1-3) and 56 days (course 4) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> TRANSPLANTATION (COURSE 5): Between 2-4 weeks following Induction, patients undergo ASCT. \> \> CONSOLIDATION (COURSES 6-9): Beginning 60-120 days following ASCT, patients receive ixazomib citrate, pomalidomide, and dexamethasone as in Induction. Treatment repeats every 28 days (courses 6-8) and 56 days (course 9) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> MAINTENANCE (COURSES 10+): Beginning 0-4 weeks following Consolidation, patients receive ixazomib citrate as in Induction. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Autologous Hematopoietic Stem Cell Transplantation: Undergo ASCT \> \> Dexamethasone: Given PO \> \> Ixazomib Citrate: Given PO \> \> Laboratory Biomarker Analysis: Correlative studies \> \> Pomalidomide: Given PO
Progression-free Survival at 18 Months (PFS18) Defined as the Proportion of Patients Alive and Free From Disease Progression at 18 Months From Study Entry	0.3750 proportion of participants Interval 0.0852 to 0.7551

SECONDARY outcome

Timeframe: 30 months

Will be estimated by the number of patients who achieve a stringent complete response (sCR), complete response CR, or VGPR divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for the true overall \>= VGPR will be calculated.

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) n=8 Participants INDUCTION (COURSES 1-4): 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. Treatment repeats every 28 days (courses 1-3) and 56 days (course 4) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> TRANSPLANTATION (COURSE 5): Between 2-4 weeks following Induction, patients undergo ASCT. \> \> CONSOLIDATION (COURSES 6-9): Beginning 60-120 days following ASCT, patients receive ixazomib citrate, pomalidomide, and dexamethasone as in Induction. Treatment repeats every 28 days (courses 6-8) and 56 days (course 9) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> MAINTENANCE (COURSES 10+): Beginning 0-4 weeks following Consolidation, patients receive ixazomib citrate as in Induction. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Autologous Hematopoietic Stem Cell Transplantation: Undergo ASCT \> \> Dexamethasone: Given PO \> \> Ixazomib Citrate: Given PO \> \> Laboratory Biomarker Analysis: Correlative studies \> \> Pomalidomide: Given PO
Percentage of Participants With Greater Than or Equal to (>=) Very Good Partial Response (VGPR) Rate	25 percentage of patients Interval 3.19 to 65.09

SECONDARY outcome

Timeframe: 36 months

The number of patients experiencing a grade 3 or greater adverse event will be reported. The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) n=8 Participants INDUCTION (COURSES 1-4): 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. Treatment repeats every 28 days (courses 1-3) and 56 days (course 4) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> TRANSPLANTATION (COURSE 5): Between 2-4 weeks following Induction, patients undergo ASCT. \> \> CONSOLIDATION (COURSES 6-9): Beginning 60-120 days following ASCT, patients receive ixazomib citrate, pomalidomide, and dexamethasone as in Induction. Treatment repeats every 28 days (courses 6-8) and 56 days (course 9) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> MAINTENANCE (COURSES 10+): Beginning 0-4 weeks following Consolidation, patients receive ixazomib citrate as in Induction. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Autologous Hematopoietic Stem Cell Transplantation: Undergo ASCT \> \> Dexamethasone: Given PO \> \> Ixazomib Citrate: Given PO \> \> Laboratory Biomarker Analysis: Correlative studies \> \> Pomalidomide: Given PO
Number of Patients Experiencing Adverse Events Graded According to the Medical Dictionary for Regulatory Activities (MedDRA) Version (v) 12.1	7 Participants

SECONDARY outcome

Timeframe: 30 months

Will be estimated by the number of patients who achieve a stringent complete response (sCR), complete response CR, very good partial response (VGPR), or partial response (PR) divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for the true overall response rate will be calculated.

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) n=8 Participants INDUCTION (COURSES 1-4): 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. Treatment repeats every 28 days (courses 1-3) and 56 days (course 4) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> TRANSPLANTATION (COURSE 5): Between 2-4 weeks following Induction, patients undergo ASCT. \> \> CONSOLIDATION (COURSES 6-9): Beginning 60-120 days following ASCT, patients receive ixazomib citrate, pomalidomide, and dexamethasone as in Induction. Treatment repeats every 28 days (courses 6-8) and 56 days (course 9) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> MAINTENANCE (COURSES 10+): Beginning 0-4 weeks following Consolidation, patients receive ixazomib citrate as in Induction. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Autologous Hematopoietic Stem Cell Transplantation: Undergo ASCT \> \> Dexamethasone: Given PO \> \> Ixazomib Citrate: Given PO \> \> Laboratory Biomarker Analysis: Correlative studies \> \> Pomalidomide: Given PO
Overall Response Rate	50 percentage of patients Interval 15.7 to 84.3

SECONDARY outcome

Timeframe: 30 months

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

Outcome measures

Outcome measures
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) n=8 Participants INDUCTION (COURSES 1-4): 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. Treatment repeats every 28 days (courses 1-3) and 56 days (course 4) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> TRANSPLANTATION (COURSE 5): Between 2-4 weeks following Induction, patients undergo ASCT. \> \> CONSOLIDATION (COURSES 6-9): Beginning 60-120 days following ASCT, patients receive ixazomib citrate, pomalidomide, and dexamethasone as in Induction. Treatment repeats every 28 days (courses 6-8) and 56 days (course 9) for up to 4 courses in the absence of disease progression or unacceptable toxicity. \> \> MAINTENANCE (COURSES 10+): Beginning 0-4 weeks following Consolidation, patients receive ixazomib citrate as in Induction. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. \> \> Autologous Hematopoietic Stem Cell Transplantation: Undergo ASCT \> \> Dexamethasone: Given PO \> \> Ixazomib Citrate: Given PO \> \> Laboratory Biomarker Analysis: Correlative studies \> \> Pomalidomide: Given PO
Percent of Patients Alive at 30 Months	75 percentage of patients Interval 50.3 to 100.0

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline up to 1 year from initiation of maintenance therapy

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 stringent complete response (sCR) or complete response (CR). Exact binomial 95% confidence intervals for the true MRD negative rate will be calculated.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 3 years

Will be measured by assessing the timing of recovery of bone marrow function. Will be summarized using descriptive statistics.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT)

Serious events: 4 serious events

Other events: 8 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) n=8 participants at risk Pomalidomide: Given PO
Blood and lymphatic system disorders Anemia	12.5% 1/8 • Number of events 1 • 36 months
Blood and lymphatic system disorders Blood and lymph sys disorders - Oth Spec	12.5% 1/8 • Number of events 1 • 36 months
Blood and lymphatic system disorders Febrile neutropenia	12.5% 1/8 • Number of events 1 • 36 months
General disorders Fever	12.5% 1/8 • Number of events 1 • 36 months
Investigations Alkaline phosphatase increased	12.5% 1/8 • Number of events 1 • 36 months
Investigations Platelet count decreased	12.5% 1/8 • Number of events 1 • 36 months
Respiratory, thoracic and mediastinal disorders Pneumonitis	12.5% 1/8 • Number of events 1 • 36 months
Skin and subcutaneous tissue disorders Rash maculo-papular	12.5% 1/8 • Number of events 1 • 36 months
Vascular disorders Thromboembolic event	12.5% 1/8 • Number of events 2 • 36 months

Other adverse events

Other adverse events
Measure	Treatment (Ixazomib, Pomalidomide, Dexamethasone, ASCT) n=8 participants at risk Pomalidomide: Given PO
Blood and lymphatic system disorders Anemia	12.5% 1/8 • Number of events 2 • 36 months
Cardiac disorders Cardiac disorders - Other, specify	12.5% 1/8 • Number of events 1 • 36 months
Gastrointestinal disorders Constipation	62.5% 5/8 • Number of events 33 • 36 months
Gastrointestinal disorders Diarrhea	75.0% 6/8 • Number of events 61 • 36 months
Gastrointestinal disorders Nausea	50.0% 4/8 • Number of events 25 • 36 months
Gastrointestinal disorders Vomiting	12.5% 1/8 • Number of events 3 • 36 months
General disorders Fatigue	100.0% 8/8 • Number of events 50 • 36 months
Infections and infestations Urinary tract infection	12.5% 1/8 • Number of events 1 • 36 months
Injury, poisoning and procedural complications Wrist fracture	12.5% 1/8 • Number of events 1 • 36 months
Investigations Creatinine increased	50.0% 4/8 • Number of events 13 • 36 months
Investigations Lymphocyte count decreased	75.0% 6/8 • Number of events 30 • 36 months
Investigations Neutrophil count decreased	100.0% 8/8 • Number of events 23 • 36 months
Investigations Platelet count decreased	87.5% 7/8 • Number of events 16 • 36 months
Investigations White blood cell decreased	100.0% 8/8 • Number of events 26 • 36 months
Metabolism and nutrition disorders Anorexia	12.5% 1/8 • Number of events 2 • 36 months
Metabolism and nutrition disorders Hyperglycemia	25.0% 2/8 • Number of events 3 • 36 months
Metabolism and nutrition disorders Hypoglycemia	12.5% 1/8 • Number of events 1 • 36 months
Nervous system disorders Peripheral sensory neuropathy	62.5% 5/8 • Number of events 61 • 36 months
Nervous system disorders Tremor	12.5% 1/8 • Number of events 1 • 36 months
Psychiatric disorders Insomnia	12.5% 1/8 • Number of events 1 • 36 months
Respiratory, thoracic and mediastinal disorders Hypoxia	12.5% 1/8 • Number of events 1 • 36 months
Respiratory, thoracic and mediastinal disorders Resp, thoracic, mediastinal - Oth spec	12.5% 1/8 • Number of events 1 • 36 months
Skin and subcutaneous tissue disorders Rash maculo-papular	12.5% 1/8 • Number of events 1 • 36 months
Vascular disorders Thromboembolic event	12.5% 1/8 • Number of events 14 • 36 months

Additional Information

Dr. Prashant Kapoor

Mayo Clinic

Phone: 507-284-2511

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place