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.

Status

UNKNOWN

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT03072498

Enrollment

Registered

2017-03-07

Start date

2017-04-06

Completion date

2021-03-05

Last updated

2019-03-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myelodysplastic Syndromes(MDS)

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.

Interventions

None listed

Study design

Observational model

CASE_CONTROL

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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.
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.
Data analysis and interpretation	2 years	To create a database summarizing the data obtained.

Countries

United States

Contacts

Primary ContactRafael Bejar, MD

rabejar@ucsd.edu858-822-5485

Backup ContactTiffany Tanaka, MD

tntanaka@ucsd.edu858-534-8575

Outcome results