Collection of Samples From Patients With MDS

• ClinicalTrials.gov Identifier: NCT03072498
• Recruitment Status: Unknown
• First Posted: March 7, 2017
• Last Update Posted: March 26, 2019
• Sponsor: PersImmune, Inc
• Collaborator: University of California, San Diego
• Information provided by (Responsible Party): PersImmune, Inc

Tracking Information

• First Submitted Date: March 1, 2017
• First Posted Date: March 7, 2017
• Last Update Posted Date: March 26, 2019
• Actual Study Start Date: April 6, 2017
• Estimated Primary Completion Date: December 5, 2020 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 1, 2017)

Genomics of patients with MDS [ Time Frame: 2 years ]

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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: March 1, 2017)

• Identification of patients' MDS-specific variant [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 2 years ]
  To test the neoantigen peptides for their in vitro immunogenicity for autologous T lymphocytes.
• Peptide immunogenicity confirmation and donor T cell stimulation [ Time Frame: 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 [ Time Frame: 2 years ]
  To create a database summarizing the data obtained.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Collection of Samples From Patients With MDS
• 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.
• 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 Type: Observational
• Study Design: Observational Model: Case-Control; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided

Biospecimen

Retention:   Samples With DNA

Description:

We intend to obtain cells from bone marrow, peripheral blood, epithelial tissue, and saliva from patients who are likely to be candidates for MDS treatment in the near future.

• 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.
• Condition: Myelodysplastic Syndromes(MDS)
• Intervention: Not Provided
• Study Groups/Cohorts: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: March 1, 2017): 24
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: March 5, 2021
• Estimated Primary Completion Date: December 5, 2020 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03072498
• Other Study ID Numbers: 161345
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: PersImmune, Inc
• Original Responsible Party: Rafael Bejar, University of California, San Diego, Assistant Clinical Professor
• Current Study Sponsor: PersImmune, Inc
• Original Study Sponsor: University of California, San Diego
• Collaborators: University of California, San Diego

Investigators

• Principal Investigator: Rafael Bejar, MD; UCSD

• PRS Account: PersImmune, Inc
• Verification Date: March 2019