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Collection of Samples From Patients With MDS

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ClinicalTrials.gov Identifier: NCT03072498
Recruitment Status : 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

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.

Condition or disease
Myelodysplastic Syndromes(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.


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Study Type : Observational
Estimated Enrollment : 24 participants
Observational Model: Case-Control
Time Perspective: Prospective
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.
Actual Study Start Date : April 6, 2017
Estimated Primary Completion Date : December 5, 2020
Estimated Study Completion Date : March 5, 2021

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. 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).


Secondary Outcome Measures :
  1. 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

  2. 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.

  3. 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.

  4. 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.

  5. Data analysis and interpretation [ Time Frame: 2 years ]
    To create a database summarizing the data obtained.


Biospecimen Retention:   Samples With DNA
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.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03072498


Contacts
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Contact: Rafael Bejar, MD 858-822-5485 rabejar@ucsd.edu
Contact: Tiffany Tanaka, MD 858-534-8575 tntanaka@ucsd.edu

Locations
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United States, California
University of California, Irvine Recruiting
Irvine, California, United States, 92868
Contact: Chao Family Comprehensive Cancer Center    877-827-8839    ucstudy@uci.edu   
Contact: Jessica Limson    7145096233    rlimson@uci.edu   
Principal Investigator: Deepa Jeyakumar, M.D.         
University of California San Diego Recruiting
La Jolla, California, United States, 92093
Contact: Colin McCarthy    858-534-8127    c4mccarthy@ucsd.edu   
Contact: Kimberly Aguilar    858-534-5201    k1aguilar@ucsd.edu   
Principal Investigator: Rafael Bejar, M.D., Ph.D.         
Sub-Investigator: Tiffany Tanaka, M.D.         
Sponsors and Collaborators
PersImmune, Inc
University of California, San Diego
Investigators
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Principal Investigator: Rafael Bejar, MD UCSD

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Responsible Party: PersImmune, Inc
ClinicalTrials.gov Identifier: NCT03072498     History of Changes
Other Study ID Numbers: 161345
First Posted: March 7, 2017    Key Record Dates
Last Update Posted: March 26, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Preleukemia
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms