Trial Outcomes & Findings for Pembrolizumab for the Treatment of Relapsed or Refractory Multiple Myeloma After Anti-BCMA CAR-T Therapies (NCT NCT05191472)

Results Overview

The International Myeloma Working Group (IMWG) uniform response criteria will be used to assess disease response and progression. The response rate is defined as the proportion of patients achieving a partial response (PR), very good partial response (VGPR), complete response (CR), or a stringent complete response (sCR) after 4 cycles of pembrolizumab therapy. A 95% confidence interval will also be reported.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

Up to 4 cycles (1 cycle is equal to 21 days)

Results posted on

2025-01-15

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Pembrolizumab) Participants received pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	3
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pembrolizumab for the Treatment of Relapsed or Refractory Multiple Myeloma After Anti-BCMA CAR-T Therapies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Pembrolizumab) n=3 Participants Participants received pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Age, Customized 50-59 years	1 Participants n=5 Participants
Age, Customized 60-69 years	1 Participants n=5 Participants
Age, Customized 70-79 years	1 Participants n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 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	1 Participants n=5 Participants
Race (NIH/OMB) White	2 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	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 4 cycles (1 cycle is equal to 21 days)

Population: Data not collected

The International Myeloma Working Group (IMWG) uniform response criteria will be used to assess disease response and progression. The response rate is defined as the proportion of patients achieving a partial response (PR), very good partial response (VGPR), complete response (CR), or a stringent complete response (sCR) after 4 cycles of pembrolizumab therapy. A 95% confidence interval will also be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 30 days after discontinuing study treatment, approximately 1 year and 8 months

The safety population will consist of all participants who receive any amount of study treatment. Safety will be assessed by evaluation of AEs using the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Specific AEs will be counted once for each participant for calculating rates but will be presented in total in participant listings including frequency of non-hematologic grade 3 or higher adverse events.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=3 Participants Participants received pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Proportion of Participants With Reported Treatment-related Adverse Events	0 Participants

SECONDARY outcome

Timeframe: Up to 30 days after discontinuing study treatment, approximately 1 year and 8 months

Population: Data not collected

Proportion of participants with reported CRS as defined by the American Society for Transplantation and Cellular Therapy (ASTCT) will be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 30 days after discontinuing study treatment, approximately 1 year and 8 months

Population: Data not collected

The Proportion of participants with reported ICANS as defined by the American Society for Transplantation and Cellular Therapy (ASTCT) will be reported

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 30 days after discontinuing study treatment, approximately 1 year and 8 months

The proportion of participants who reported discontinuing treatment due to a treatment-related toxicity will be reported.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab) n=3 Participants Participants received pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Proportion of Participants Discontinuing Treatment Due to Toxicity	0 proportion of participants

SECONDARY outcome

Timeframe: Approximately 1 year and 8 months

Population: Data not collected

The proportion of patients achieving progressive disease, stable disease, partial response, very good partial response, complete response, stringent complete response for best-achieved response, using the IMWG criteria for assessing response, at any time during pembrolizumab therapy will be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years

Population: Data not collected

For OS, participants who remain alive at the end of study follow-up will be censored at the time when the participants is known to be alive. Results will be summarized using the Kaplan-Meier method. The 12-months percentage of participants alive, 25th percentile, median, and 75th percentile, along with corresponding 95% confidence intervals, will be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 1 year

Population: Data not collected

For PFS, participants without progression or death will be censored at the time of the last evaluable disease assessment, while participants without a disease assessment will be censored at the date of first study treatment received. Results will be summarized using the Kaplan-Meier method. The 12-months event-free probabilities, 25th percentile, median, and 75th percentile, along with corresponding 95% confidence intervals, will be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years

Population: Data not collected

For PFS, participants without progression or death will be censored at the time of the last evaluable disease assessment, while participants without a disease assessment will be censored at the date of first study treatment received. Results will be summarized using the Kaplan-Meier method. The 24-months event-free probabilities, 25th percentile, median, and 75th percentile, along with corresponding 95% confidence intervals, will be reported.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years

Population: Data not collected

Median duration of response among the responders will be reported along with 95% confidence intervals.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 2 years

Population: Data not collected

Competing risks analysis will be performed for analyzing TNT, with death as a competing event. For patients who are alive and do not receive the next line of therapy, TNT will be censored at the last study contact. Cumulative incidence function, along with 95% confidence intervals, will be reported for TNT.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Pembrolizumab)

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Treatment (Pembrolizumab) n=3 participants at risk Participants received pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Gastrointestinal disorders Diarrhea	33.3% 1/3 • Number of events 1 • Up to 6 months
Gastrointestinal disorders Nausea	33.3% 1/3 • Number of events 1 • Up to 6 months
Gastrointestinal disorders Oral pain	33.3% 1/3 • Number of events 1 • Up to 6 months
Blood and lymphatic system disorders Anemia	33.3% 1/3 • Number of events 1 • Up to 6 months
Investigations Weight Loss	33.3% 1/3 • Number of events 1 • Up to 6 months

Additional Information

Dr. Alfred Chung, MD

University of California, San Francisco

Phone: (415) 476-1000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place