AMLCTL: A Clinical Trial of Cytotoxic T Cells Augmenting Autologous Hematopoietic Stem Cell (AHSC) Transplantation for Acute Myeloid Leukemia (AML)

• Sponsor: Thomas A. Lane, MD (Other)
• Overall Status: Terminated
• CT.gov ID: NCT00808080
• Collaborator: (none)
• Enrollment: 6
• Location: 1
• Arm: 1
• Duration (Months): 41
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
AML	Biological: AMLCTL	Phase 1/Phase 2

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 6 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Clinical Trial of Cytotoxic T Cells Augmenting Autologous Hematopoietic Stem Cell Transplantation for Acute Myeloid Leukemia
• Study Start Date: Jan 1, 2010
• Actual Primary Completion Date: Jun 1, 2013
• Actual Study Completion Date: Jun 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Biologic AML_CTL cells	Biological: AMLCTL; Ex-vivo expanded cytotoxic autologous AML-reactive T cells (CTL)

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 75 Years
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UCSD	La Jolla	California	United States	92093

Sponsors and Collaborators

• Thomas A. Lane, MD

Investigators

• Principal Investigator: Thomas Lane, MD, UCSD

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Thomas A. Lane, MD, Professor of Pathology, University of California, San Diego
• ClinicalTrials.gov Identifier: NCT00808080
• Other Study ID Numbers: 070768
• First Posted: Dec 15, 2008
• Last Update Posted: Apr 9, 2019
• Last Verified: Apr 1, 2019

• Keywords provided by Thomas A. Lane, MD, Professor of Pathology, University of California, San Diego: AML
• Additional relevant MeSH terms: Leukemia, Myeloid, Acute

Study Results

Participant Flow

Arm/Group Title	Biologic
Arm/Group Description	AML_CTL cells AMLCTL: Ex-vivo expanded cytotoxic autologous AML-reactive T cells (CTL) 6 patients were enrolled, however none of the 6 patients became eligible to receive the AML CTL Infusion
Period Title: Overall Study
STARTED	6
COMPLETED	0
NOT COMPLETED	6

Baseline Characteristics

Arm/Group Title	Biologic
Arm/Group Description	AML_CTL cells AMLCTL: Ex-vivo expanded cytotoxic autologous AML-reactive T cells (CTL)
Overall Participants	6
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	6 100%
>=65 years	0 0%
Sex: Female, Male (Count of Participants)
Female	3 50%
Male	3 50%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	1 16.7%
Not Hispanic or Latino	5 83.3%
Unknown or Not Reported	0 0%
Region of Enrollment (participants) [Number]
United States	6 100%
Percent Circulating Blasts In Peripheral Blood (percentage of peripheral blood) [Mean (Full Range) ]
Mean (Full Range) [percentage of peripheral blood]	52.7
Percent Blast Cells in Bone Marrow (percentage of bone marrow) [Mean (Full Range) ]
Mean (Full Range) [percentage of bone marrow]	67.4
WBC at Baseline (1000 cells/mm^3) [Mean (Full Range) ]
Mean (Full Range) [1000 cells/mm^3]	18.4
ANC at Baseline (1000 cells/mm^3) [Mean (Full Range) ]
Mean (Full Range) [1000 cells/mm^3]	2460.7

Outcome Measures

1. Primary Outcome

Title	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

Outcome Measure Data

Analysis Population Description

Not Applicable as none of the enrolled patients became eligible for infusion

Arm/Group Title	Biologic
Arm/Group Description	AML_CTL cells AMLCTL: Ex-vivo expanded cytotoxic autologous AML-reactive T cells (CTL) 6 patients were enrolled, however none of the 6 patients became eligible to receive the AML CTL Infusion
Measure Participants	0

2. Secondary Outcome

Title	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.
Description	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

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Biologic
Arm/Group Description	AML_CTL cells AMLCTL: Ex-vivo expanded cytotoxic autologous AML-reactive T cells (CTL) 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, therefore NO outcome measure results are available.
Measure Participants	0

Adverse Events

Adverse Event Reporting Description	No adverse events or serious adverse events occurred related to study treatment as no patients have received their AML CTL infusion.
Arm/Group Title	Biologic
Arm/Group Description	AML_CTL cells AMLCTL: Ex-vivo expanded cytotoxic autologous AML-reactive T cells (CTL) 6 patients were enrolled, however none of the 6 patients became eligible to receive the AML CTL Infusion
All Cause Mortality
	Biologic
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Biologic
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)
Other (Not Including Serious) Adverse Events
	Biologic
	Affected / at Risk (%)	# Events
Total	0/0 (NaN)

Limitations/Caveats

Accrual trial was insufficient. No patient became eligible for CTL infusion. Cell culture information gleaned from a comparison of different culture methods will greatly facilitate future studies of autologous CTL-directed immunotherapy for AML.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.