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Collection of Samples From Patients With MDS

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ClinicalTrials.gov Identifier: NCT03072498
Recruitment Status : Unknown
Verified March 2019 by PersImmune, Inc.
Recruitment status was:  Recruiting
First Posted : March 7, 2017
Last Update Posted : March 26, 2019
Sponsor:
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
Change History
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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
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
Removed Location Countries  
 
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