Myelodysplastic Syndromes(MDS)NCT03072498
Collection of Samples From Patients With MDS
An observational study of Myelodysplastic Syndromes(MDS), run by PersImmune, Inc at 2 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes(MDS)
- How many people
- 24 participants
- Where
- 2 sitesUnited States
- Study dates
- 6 Apr 2017to 5 Mar 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
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
Trial locations and contacts
2 sites in 1 country.
United States2 sites, 2 recruiting
- Recruiting
University of California, Irvine
Irvine, California, 92868
Chao Family Comprehensive Cancer Center, 877-827-8839, ucstudy@uci.edu
Jessica Limson, 7145096233, rlimson@uci.edu
- Recruiting
University of California San Diego
La Jolla, California, 92093
Colin McCarthy, 858-534-8127, c4mccarthy@ucsd.edu
Kimberly Aguilar, 858-534-5201, k1aguilar@ucsd.edu
About this study
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.
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.
Read the 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.
- Conditions
- Myelodysplastic Syndromes(MDS)
- Medical subject headings
- Myelodysplastic Syndromes
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 2 years
Secondary outcomes (5)
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
Measured: 2 years
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.
Measured: 2 years
Peptide Immunogenicity confirmation and donor T cell stimulation
To test the neoantigen peptides for their in vitro immunogenicity for autologous T lymphocytes.
Measured: 2 years
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.
Measured: 2 years
Data analysis and interpretation
To create a database summarizing the data obtained.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2017
- Study start6 Apr 2017
- Primary completion5 Dec 2020 (estimated)
- Study completion5 Mar 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic Syndromes(MDS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PersImmune, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of California, San Diego
- Responsible party
- Sponsor
- Investigators
- Rafael Bejar, MD, Principal investigator, UCSD
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03072498
- Sponsor's protocol ID
- 161345
- Status last verified
- Mar 2019
- First submitted
- 1 Mar 2017
- Last updated
- 26 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT03072498
- Acronym / sponsor ID
- 161345
- Conditions
- Myelodysplastic Syndromes(MDS)
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- PersImmune, IncIndustry (pharma, biotech, devices)
- Collaborators
- University of California, San Diego
- Study start
- 6 Apr 2017 (actual)
- Primary completion
- 5 Dec 2020 (estimated)
- Study completion
- 5 Mar 2021 (estimated)
- First posted
- 7 Mar 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case-control
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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).
5 secondary outcomes
Identification of patients' MDS-specific variant
Time frame: 2 years
Immunogenic mutant neoantigen peptide selection
Time frame: 2 years
Peptide Immunogenicity confirmation and donor T cell stimulation
Time frame: 2 years
Peptide immunogenicity confirmation and donor T cell stimulation
Time frame: 2 years
Data analysis and interpretation
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2019.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rafael Bejar, MD principal investigator
UCSD
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myelodysplastic Syndromes(MDS), run by PersImmune, Inc at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 26 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rafael Bejar, MD (858-822-5485, rabejar@ucsd.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Genomics of patients with MDS (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Mar 2019.