Collection of Samples From Patients With MDS

PersImmune, Inc24 enrolled

Overview

The purpose of this study is to collect information and bone marrow, blood, saliva, cheek cells and skin to be used in the laboratory to assist the sponsor in identifying a new way of treating MDS.

Goals of the study: The purpose of this study is to collect the blood and marrow samples, and non-involved fibroblasts, that are required to identify the unique, personalized array of mutation-driven neoantigens that are expressed by the subject's MDS cells and to assess the feasibility of immunizing and expanding one or more of the patient's T cells ex vivo for investigation of their use as adoptive cellular immunotherapy.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Myelodysplastic Syndromes(MDS)

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Patients must meet the following initial inclusion criteria: * Diagnosis or suspected diagnosis of MDS or CCUS * Age 18 or older Patient exclusion criteria: * Currently receiving or within 3 months has received hypomethylating agent(s), lenalidomide, cytotoxic agents, or within the previous 4 weeks corticosteroids \> 5 mg prednisone daily or any other immunosuppressants * Previous allogenic transplant * Inability to provide consent * Prisoners

Locations (2)

University of California, Irvine

Irvine, California, United States

RECRUITING

University of California San Diego

La Jolla, California, United States

RECRUITING

Outcomes

Primary Outcomes

Genomics of patients with MDS

To sequence the exome and transcriptome obtained from MDS hematopoietic cells and the exome from non-hematopoietic cells (e.g. fibroblasts).

Time frame: 2 years

Secondary Outcomes

Identification of patients' MDS-specific variant

To select variants by comparing MDS versus non-MDS cell exome sequences. MDS-specific variant sequences are defined as those that differ between the two and are not common polymorphisms. We will also compare myeloid and lymphoid hematopoietic cells and assess the number of myeloid-specific vs myeloid and lymphoid MDS-related variants

Time frame: 2 years

Immunogenic mutant neoantigen peptide selection

To select putative mutation-driven neoantigen-related peptides, which represent the sequences obtained from Aim 2, according to their ability to bind to the patient's MHC using PersImmune's licensed and proprietary algorithms.

Time frame: 2 years

Peptide Immunogenicity confirmation and donor T cell stimulation

To test the neoantigen peptides for their in vitro immunogenicity for autologous T lymphocytes.

Time frame: 2 years

Peptide immunogenicity confirmation and donor T cell stimulation

To test the potency and specificity of neoantigen peptide-stimulated T cells for the patient's MDS cells that express the defined neoantigens.

Time frame: 2 years

Data analysis and interpretation

To create a database summarizing the data obtained.

Time frame: 2 years