Trial Outcomes & Findings for Study on the Effect of Ibrutinib on High Risk Smoldering Multiple Myeloma Patients (NCT NCT02943473)

Last Updated: 2020-12-16

Results Overview

Disease response - the proportion of patients with high risk smoldering multiple myeloma who do not progress to symptomatic myeloma as defined by the IMWG.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

up to 1 year

Results posted on

2020-12-16

Participant Flow

Participants enrolled from May 2017 through June 2019

Participant milestones

Participant milestones
Measure	Ibrutinib Ibrutinib (trademark name is IMBRUVICA®). 560 mg dose taken PO daily for 12 cycles (28 days each). Ibrutinib: Ibrutinib is a type of drug called a "kinase inhibitor." Kinases are proteins inside cells that help cells live and grow. Ibrutinib blocks a specific kinase protein in our bodies. This protein is thought to be very important in helping blood cancer cells live and grow. By blocking this kinase protein, ibrutinib stops cancer cells from growing.
Overall Study STARTED	9
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Ibrutinib Ibrutinib (trademark name is IMBRUVICA®). 560 mg dose taken PO daily for 12 cycles (28 days each). Ibrutinib: Ibrutinib is a type of drug called a "kinase inhibitor." Kinases are proteins inside cells that help cells live and grow. Ibrutinib blocks a specific kinase protein in our bodies. This protein is thought to be very important in helping blood cancer cells live and grow. By blocking this kinase protein, ibrutinib stops cancer cells from growing.
Overall Study enrolled but not dosed	1
Overall Study screen failure	1
Overall Study Physician Decision	4

Baseline Characteristics

Study on the Effect of Ibrutinib on High Risk Smoldering Multiple Myeloma Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ibrutinib n=9 Participants Ibrutinib (trademark name is IMBRUVICA®). 560 mg dose administered on a continuous basis Ibrutinib: Ibrutinib is a type of drug called a "kinase inhibitor." Kinases are proteins inside cells that help cells live and grow. Ibrutinib blocks a specific kinase protein in our bodies. This protein is thought to be very important in helping blood cancer cells live and grow. By blocking this kinase protein, ibrutinib stops cancer cells from growing.
Age, Customized 50-59 years old	3 Participants n=5 Participants
Age, Customized 60 years and older	6 Participants n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 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	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to 1 year

Disease response - the proportion of patients with high risk smoldering multiple myeloma who do not progress to symptomatic myeloma as defined by the IMWG.

Outcome measures

Outcome measures
Measure	Ibrutinib n=7 Participants Ibrutinib (trademark name is IMBRUVICA®). 560 mg dose administered on a continuous basis Ibrutinib: Ibrutinib is a type of drug called a "kinase inhibitor." Kinases are proteins inside cells that help cells live and grow. Ibrutinib blocks a specific kinase protein in our bodies. This protein is thought to be very important in helping blood cancer cells live and grow. By blocking this kinase protein, ibrutinib stops cancer cells from growing.
Number of Patients Without Symptomatic Myeloma	5 Participants

SECONDARY outcome

Timeframe: up to 1 year

Overall response rate, defined as partial response or better per IMWG criteria. (IMWG response criteria are - Complete Response, Very good partial response, partial response, Minimal response, stable disease, and progressive disease)

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline and one year

Changes in bone density, particularly in patients with osteopenia (defined as T-score on bone densitometry testing (DEXA) of -1 to -2.5).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: baseline and one year

Changes in PET-MRI, particularly in patients with osteopenia