Clinical trial NCT02667093: Samples From Leukemia Patients and Their Donors to Identify Specific Antigens
Official title: Bone Marrow and Peripheral Blood (PB) Samples From Patients With Leukemia and PB From Their BM Donors (BMD) to Identify Leukemia-Specific Antigens (LSA) and Graft Versus Host Disease Antigens (GVHDA) for Use in Cellular Immunotherapy
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Acute Myeloid Leukemia
Keywords: Acute Leukemia
Acute Myeloid Leukemia
AML
Lead sponsor: PersImmune, Inc
Sponsor class: INDUSTRY
Collaborators: name: University of California, San Diego
First posted: 2016-01-28
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with leukemia and their potential bone marrow donors.

Brief summary: The purpose of this project is to develop a process to identify highly personalized antigens that are uniquely expressed by the patient's own leukemia cells that can be used for cellular immune therapy.

Detailed description: It is well known that tumor cells and leukemia cells express different surface structures (called antigens) that can serve as targets for cancer cell destruction by the immune system. Effective immune therapies are characterized by high specificity and low toxicity. One of the major obstacles impeding the use of these therapies as standard of care is the identification of good target antigens. In acute myeloid leukemia (AML) there is major patient to patient variation in leukemia antigens, so there is no universal AML cell target. Rather, each patient has a unique array of potential cell targets. Thanks to the rapid progress of new DNA/RNA sequencing technologies, the identification of these unique, patient-specific leukemia cell antigen-targets is now possible.

The purpose of this project is to develop a process to identify highly personalized antigens that are uniquely expressed by the patient's own leukemia cells that can be used for cellular immune therapy.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of AML with plan to receive a bone marrow transplant

Exclusion Criteria:

* NA

Primary outcomes:
measure: Genomics of patients with leukemia and their HLA matched bone marrow transplant donors.
timeFrame: 5 years
Secondary outcomes:
measure: Identification of patients' leukemia cell mutations and polymorphisms that are different from their HLA matched bone marrow transplant donors
timeFrame: 5 years
measure: Immunogenic mutant neoantigen peptide selection
timeFrame: 5 years
measure: Peptide immunogenicity confirmation and donor T cell stimulation
timeFrame: 5 years
measure: Data analysis and interpretation
timeFrame: 5 years
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: Atlanta
country: United States
facility: Northside Hospital
state: Georgia
Study officials:
affiliation: University of California, San Diego
name: Edward Ball, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Montagna D, Maccario R, Locatelli F, Rosti V, Yang Y, Farness P, Moretta A, Comoli P, Montini E, Vitiello A. Ex vivo priming for long-term maintenance of antileukemia human cytotoxic T cells suggests a general procedure for adoptive immunotherapy. Blood. 2001 Dec 1;98(12):3359-66. doi: 10.1182/blood.v98.12.3359.
citation: Kreiter S, Castle JC, Tureci O, Sahin U. Targeting the tumor mutanome for personalized vaccination therapy. Oncoimmunology. 2012 Aug 1;1(5):768-769. doi: 10.4161/onci.19727.
citation: Klebanoff CA, Gattinoni L, Restifo NP. Sorting through subsets: which T-cell populations mediate highly effective adoptive immunotherapy? J Immunother. 2012 Nov-Dec;35(9):651-60. doi: 10.1097/CJI.0b013e31827806e6.
citation: Brenner MK. Will T-cell therapy for cancer ever be a standard of care? Cancer Gene Ther. 2012 Dec;19(12):818-21. doi: 10.1038/cgt.2012.74. Epub 2012 Oct 12.