Trial Outcomes & Findings for Lymphodepletion and Anti-PD-1 Blockade to Reduce Relapse in AML Patient Not Eligible for Transplant (NCT NCT02771197)

Last Updated: 2023-09-28

Results Overview

Hypothesis is that following lymphodepleting chemotherapy and pembrolizumab, the 2-year relapse risk will decrease to ≤35%

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

2 years

Results posted on

2023-09-28

Participant milestones
Measure	Lymphodepletion Plus Pembrolizumab Fludarabine \& Melphalan followed by autologous stem cell transplantation. Pembrolizumab will begin on Day +1. Fludarabine Melphalan Pembrolizumab
Overall Study STARTED	20
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	5

Withdrawal data not reported

Lymphodepletion and Anti-PD-1 Blockade to Reduce Relapse in AML Patient Not Eligible for Transplant

Baseline characteristics by cohort
Measure	Lymphodepletion Plus Pembrolizumab n=20 Participants Fludarabine \& Melphalan followed by autologous stem cell transplantation. Pembrolizumab will begin on Day +1. Fludarabine Melphalan Pembrolizumab
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants
Age, Categorical >=65 years	11 Participants n=5 Participants
Age, Continuous	64 years n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 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	9 Participants n=5 Participants
Race (NIH/OMB) White	10 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	20 participants n=5 Participants

Timeframe: 2 years

Hypothesis is that following lymphodepleting chemotherapy and pembrolizumab, the 2-year relapse risk will decrease to ≤35%

Outcome measures
Measure	Lymphodepletion Plus Pembrolizumab n=20 Participants Fludarabine \& Melphalan followed by autologous stem cell transplantation. Pembrolizumab will begin on Day +1. Fludarabine Melphalan Pembrolizumab
Number of Patients With 2-year Relapse Risk	10 Participants

Timeframe: 6 months

Assess safety of pembrolizumab in patients with AML following lymphodepleting chemotherapy

Outcome measures
Measure	Lymphodepletion Plus Pembrolizumab n=20 Participants Fludarabine \& Melphalan followed by autologous stem cell transplantation. Pembrolizumab will begin on Day +1. Fludarabine Melphalan Pembrolizumab
Assess Safety of Pembrolizumab by Recording the Number of Participants With Treatment-related Adverse Events	9 Participants

Serious events: 12 serious events

Other events: 20 other events

Deaths: 1 deaths

Serious adverse events
Measure	Lymphodepletion Plus Pembrolizumab n=20 participants at risk Fludarabine \& Melphalan followed by autologous stem cell transplantation. Pembrolizumab will begin on Day +1. Fludarabine Melphalan Pembrolizumab
Infections and infestations Neutropenic fever	35.0% 7/20 • 6 months
Gastrointestinal disorders Diarrhea	20.0% 4/20 • 6 months
Musculoskeletal and connective tissue disorders Weakness	5.0% 1/20 • 6 months
Gastrointestinal disorders Nausea	20.0% 4/20 • 6 months
Musculoskeletal and connective tissue disorders Failure to thrive	10.0% 2/20 • 6 months
Gastrointestinal disorders Vomiting	10.0% 2/20 • 6 months
Gastrointestinal disorders Colitis	10.0% 2/20 • 6 months
Gastrointestinal disorders Mucositis	5.0% 1/20 • 6 months
Musculoskeletal and connective tissue disorders Right flank pain	5.0% 1/20 • 6 months
Gastrointestinal disorders Decreased appetite	5.0% 1/20 • 6 months
Gastrointestinal disorders Abdominal pain	10.0% 2/20 • 6 months
Infections and infestations Non-neutropenic fever	25.0% 5/20 • 6 months
Cardiac disorders Hypotension	5.0% 1/20 • 6 months
Cardiac disorders Atrial fibrillation	5.0% 1/20 • 6 months
Infections and infestations General infection	10.0% 2/20 • 6 months
Blood and lymphatic system disorders Cytopenia	10.0% 2/20 • 6 months
Musculoskeletal and connective tissue disorders Radial fracture	5.0% 1/20 • 6 months

Other adverse events
Measure	Lymphodepletion Plus Pembrolizumab n=20 participants at risk Fludarabine \& Melphalan followed by autologous stem cell transplantation. Pembrolizumab will begin on Day +1. Fludarabine Melphalan Pembrolizumab
Blood and lymphatic system disorders Cytopenias	100.0% 20/20 • 6 months
Gastrointestinal disorders Nausea	25.0% 5/20 • 6 months
Gastrointestinal disorders Vomiting	10.0% 2/20 • 6 months
Gastrointestinal disorders Diarrhea	25.0% 5/20 • 6 months
Gastrointestinal disorders Colitis	10.0% 2/20 • 6 months
Gastrointestinal disorders Mucositis	10.0% 2/20 • 6 months
General disorders Fatigue	10.0% 2/20 • 6 months
Cardiac disorders Hypertension	20.0% 4/20 • 6 months
Metabolism and nutrition disorders Hypokalemia	10.0% 2/20 • 6 months

The Blood and Marrow Transplant Group of Georgia/Northside Hospital

Phone: 404-255-1930