Collection of Samples From Patients With MDS

Collection of Bone Marrow, Peripheral Blood (PB), Epithelial Tissue, and Saliva Samples From Patients With Myelodysplastic Syndromes (MDS) to Identify MDS-Specific Antigens (MSA) for Use in Cellular Immunotherapy.

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.

Study Overview

• Status: Unknown
• Conditions: Myelodysplastic Syndromes(MDS)

Detailed Description

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 (Anticipated): 24

Contacts and Locations

• Study Contact: Name: Rafael Bejar, MD; Phone Number: 858-822-5485; Email: rabejar@ucsd.edu
• Study Contact Backup: Name: Tiffany Tanaka, MD; Phone Number: 858-534-8575; Email: tntanaka@ucsd.edu

Study Locations

• United States
  • California
    • Irvine, California, United States, 92868
      • Recruiting
      • University of California, Irvine
      • Contact: Chao Family Comprehensive Cancer Center; Phone Number: 877-827-8839; Email: ucstudy@uci.edu
      • Contact: Jessica Limson; Phone Number: 7145096233; Email: rlimson@uci.edu
      • Principal Investigator: Deepa Jeyakumar, M.D.
    • La Jolla, California, United States, 92093
      • Recruiting
      • University of California San Diego
      • Contact: Colin McCarthy; Phone Number: 858-534-8127; Email: c4mccarthy@ucsd.edu
      • Contact: Kimberly Aguilar; Phone Number: 858-534-5201; Email: k1aguilar@ucsd.edu
      • Principal Investigator: Rafael Bejar, M.D., Ph.D.
      • Sub-Investigator: Tiffany Tanaka, M.D.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Approximately 24 Subjects (patients and donors) will be recruited over a 3-year period. Both male and female subjects will be included and there will be no restrictions based on race or ethnicity.

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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.	2 years
Peptide Immunogenicity confirmation and donor T cell stimulation	To test the neoantigen peptides for their in vitro immunogenicity for autologous T lymphocytes.	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.	2 years
Data analysis and interpretation	To create a database summarizing the data obtained.	2 years

Collaborators and Investigators

• Sponsor: PersImmune, Inc
• Collaborators: University of California, San Diego
• Investigators: Principal Investigator: Rafael Bejar, MD, UCSD

Study record dates

Study Major Dates

• Study Start (Actual): April 6, 2017
• Primary Completion (Anticipated): December 5, 2020
• Study Completion (Anticipated): March 5, 2021

Study Registration Dates

• First Submitted: March 1, 2017
• First Submitted That Met QC Criteria: March 1, 2017
• First Posted (Actual): March 7, 2017

Study Record Updates

• Last Update Posted (Actual): March 26, 2019
• Last Update Submitted That Met QC Criteria: March 25, 2019
• Last Verified: March 1, 2019

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Preleukemia
• Other Study ID Numbers: 161345

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No