Collection of Samples From Patients With MDS

• Conditions: Myelodysplastic Syndromes(MDS)

• Registration Number: NCT03072498
• Lead Sponsor: PersImmune, Inc

Brief Summary

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.

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 Timeline

• Study First Submitted: 2017-03-01
• Study First Submitted QC: 2017-03-01
• Study First Posted: 2017-03-07
• Study Start: 2017-04-06
• Status Verified: 2019-03
• Last Update Submitted: 2019-03-25
• Last Update Posted: 2019-03-26
• Primary Completion: 2020-12-05
• Study Completion: 2021-03-05

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Genomics of patients with MDS	2 years	To sequence the exome and transcriptome obtained from MDS hematopoietic cells and the exome from non-hematopoietic cells (e.g. fibroblasts).

Secondary Outcome Measures

Name	Time	Method
Identification of patients' MDS-specific variant	2 years	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
Immunogenic mutant neoantigen peptide selection	2 years	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.
Data analysis and interpretation	2 years	To create a database summarizing the data obtained.
Peptide Immunogenicity confirmation and donor T cell stimulation	2 years	To test the neoantigen peptides for their in vitro immunogenicity for autologous T lymphocytes.
Peptide immunogenicity confirmation and donor T cell stimulation	2 years	To test the potency and specificity of neoantigen peptide-stimulated T cells for the patient's MDS cells that express the defined neoantigens.

Trial Locations

Locations (2)

University of California San Diego; 🇺🇸 La Jolla, California, United States

University of California, Irvine; 🇺🇸 Irvine, California, United States