Clinical trial NCT02771197: Lymphodepletion and Anti-PD-1 Blockade to Reduce Relapse in AML Patient Not Eligible for Transplant
Official title: Phase II Trial of Lymphodepletion and Anti-PD-1 Blockade to Reduce Relapse in High Risk AML Patients Who Are Not Eligible for Allogeneic Stem Cell Transplantation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acute Myeloid Leukemia
Keywords: AML
Lead sponsor: Northside Hospital, Inc.
Sponsor class: OTHER
Collaborators: name: Merck Sharp & Dohme LLC
First posted: 2016-05-13
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 78
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: AML is the most common acute leukemia in adults. Most patients can undergo allogeneic stem cell transplantation as a possible cure; however, many patients are not candidates for allogeneic transplant due to age, overall health, psychosocial factors, and/or lack of available donors. Therefore, these patients are unable to receive the therapeutic benefits of the "graft-versus-leukemia" effect of donor immune cells. The aim of this study is to hopefully break immune tolerance to AML cells to provide better outcomes in patients with non-favorable risk AML.

Detailed description: Non-favorable risk AML patients will undergo a preparative regimen of lymphodepletion of Flu/Mel followed by autologous transplantation. Anti-PD-1 therapy of pembrolizumab will begin on Day +1 following stem cell transplantation and will be administered every 3 weeks for a total of 8 doses. According to the literature, the risk of 2-year relapse is estimated to be 60-80% in patients with non-favorable risk AML in CR-1. With this protocol, investigators hypothesize that following lymphodepleting chemotherapy and pembrolizumab, the 2-year relapse risk will decrease to less than or equal to 35%. The one-sided Wald test at 5% significance level will be used to test the hypothesis. The size of 20 patients yields the power of 90.5% assuming that the actual 2-year leukemia-free survival is 60%.

Eligibility criteria: Inclusion Criteria:

* Non-favorable risk AML
* In CR-1 or subsequent CR
* Completed at least one cycle of consolidation chemotherapy
* Collection of at least 2x106/kg CD34+ cells
* KPS of 70% or greater

Exclusion Criteria:

* Received investigational agent within 4 weeks of first dose
* Prior chemotherapy, radiation therapy within 2 weeks of first dose
* Hypersensitivity to pembrolizumab or any of its excipients
* Received prior therapy with anti-PD-1, anti-PD-L1, or anti-PD-L2 agent

Primary outcomes:
measure: Number of Patients With 2-year Relapse Risk
timeFrame: 2 years
Secondary outcomes:
measure: Assess Safety of Pembrolizumab by Recording the Number of Participants With Treatment-related Adverse Events
timeFrame: 6 months
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Blood and Marrow Transplant Group of Georgia
state: Georgia
city: Atlanta
country: United States
facility: Northside Hospital
state: Georgia
Study officials:
affiliation: Blood and Marrow Transplant Group of Georgia
name: Scott Solomon, MD
role: PRINCIPAL_INVESTIGATOR
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