Collection of Samples From Patients With MDS

Myelodysplastic Syndromes(MDS) Clinical Trial

Official title:

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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03072498
• Other study ID #: 161345
• Status: Recruiting
• Start date: April 6, 2017
• Est. completion date: March 5, 2021

Study information

• Verified date: March 2019
• Source: PersImmune, Inc
• Contact: Rafael Bejar, MD
• Phone: 858-822-5485
• Email: rabejar[@]ucsd.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 24
• Est. completion date: March 5, 2021
• Est. primary completion date: December 5, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Myelodysplastic Syndromes
• Myelodysplastic Syndromes(MDS)
• Preleukemia
• Syndrome

Locations

Country	Name	City	State
United States	University of California, Irvine	Irvine	California
United States	University of California San Diego	La Jolla	California

Sponsors (2)

Lead Sponsor	Collaborator
PersImmune, Inc	University of California, San Diego

Country where clinical trial is conducted

United States, 

Outcome

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