AML
Conditions
Keywords
AML
Brief summary
The aim of this protocol is to investigate a novel form of immune therapy for patients with acute myelogenous leukemia (AML) who are in remission (CR) but who are at high risk for relapse.
Detailed description
Primary Aim: To conduct a Phase 1/2 clinical trial of autologous CTL-mediated immunotherapy in a homogeneous group of patients with AML who have recently received an autologous hematopoietic stem cell transplant. Specifically: Phase 1: To determine the MTD of autologous AML-reactive cultured CTL in patients with AML who have recently received an AHSCT. Phase 2: To determine 1 year progression-free survival of the study group vs institutional historical control group composed of a sequential series of recent patients who have received an AHSCT for AML.
Interventions
BIOLOGICALAMLCTL
Ex-vivo expanded cytotoxic autologous AML-reactive T cells (CTL)
Sponsors
Thomas A. Lane, MD
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
(Initial Eligibility Screen): * Diagnosis of AML, not M3 * At least 10% of circulating leukocytes are AML blast cells * Age 18 through 75 * Sex male or female * Patient is considered a potential candidate for AHSCT
Exclusion criteria
(Initial Eligibility Screen): * Participation in another immunotherapy trial within 30 days * Presence of active malignancy other than AML * History of autoimmune disease requiring systemic treatment * ECOG performance status of 3 or 4 * Major organ system dysfunction * Recent (30 days) or current use of steroids other than topical skin preparations * History of allogeneic transplant * Patients who, for any reason are not deemed candidates for AHSCT Eligibility for autologous CTL Infusion: Inclusion Criteria: * Patient has CTL that are in sufficient number and are suitable for infusion * Patient is stable, afebrile, engrafted, ECOG status 0-2, in CR and received AHSCT 45 - 60 days earlier. Exclusion or delay criteria: * Temperature \> 38 C and/or known to be infected * Absence of engraftment ANC \> 500 and Plt \> 20,000 unsupported * Life expectancy less than 6 weeks * Autoimmune disease requiring systemic treatment. * ECOG performance status of 3 or 4 * Major organ system dysfunction
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 6 Dose Cohorts for Safety Monitoring. Each Cohort is Assessed for DLT for One Month After Autologous Cultured CTL Infusion Prior to Enrolling the Next Cohort. | 2.5 years estimated |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| If Phase I Has Successfully Shown the Target Dose to be Below the MTD Continue Enrolling Until 38 Patients Have Received the Target Dose. Patients Will be Monitored for Safety and Efficacy. | 2.5 years estimated | 6 patients were enrolled, however none of the 6 patients became eligible to receive the AML CTL Infusion. No CTL infusions were administered to any subject, consequently NO outcome measure results are available. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Biologic AML\_CTL cells
AMLCTL: Ex-vivo expanded cytotoxic autologous AML-reactive T cells (CTL) | 6 |
| Total | 6 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Patients Became Ineligible for infusion | 6 |
Baseline characteristics
| Characteristic | Biologic |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 6 Participants |
| ANC at Baseline | 2460.7 1000 cells/mm^3 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 5 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Percent Blast Cells in Bone Marrow | 67.4 percentage of bone marrow |
| Percent Circulating Blasts In Peripheral Blood | 52.7 percentage of peripheral blood |
| Region of Enrollment United States | 6 participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 3 Participants |
| WBC at Baseline | 18.4 1000 cells/mm^3 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 |
Outcome results
Primary
6 Dose Cohorts for Safety Monitoring. Each Cohort is Assessed for DLT for One Month After Autologous Cultured CTL Infusion Prior to Enrolling the Next Cohort.
Time frame: 2.5 years estimated
Population: Not Applicable as none of the enrolled patients became eligible for infusion
Secondary
If Phase I Has Successfully Shown the Target Dose to be Below the MTD Continue Enrolling Until 38 Patients Have Received the Target Dose. Patients Will be Monitored for Safety and Efficacy.
6 patients were enrolled, however none of the 6 patients became eligible to receive the AML CTL Infusion. No CTL infusions were administered to any subject, consequently NO outcome measure results are available.
Time frame: 2.5 years estimated
Population: None. 6 patients were enrolled, however none of the 6 patients became eligible to receive the AML CTL Infusion. No CTL infusions were administered to any subject, consequently NO outcome measure results are available.