Induced Pluripotent Stem CellsNCT02056613
Blood Collection From Healthy Volunteers and Patients for the Production of Clinical Grade Induced Pluripotent Stem Cell (iPSC) Products
An observational study of Induced Pluripotent Stem Cells, run by National Institutes of Health Clinical Center (CC).
Trial summary
Background: Pluripotent stem cells are cells that can be converted to any type of cell (muscle, nerve, liver, etc.). Researchers are collecting blood samples to develop way to make a special cell product called induced pluripotent stem cells (iPSCs). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Induced Pluripotent Stem Cells
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 5 Feb 2014to 1 Nov 2018
- 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
- Healthy volunteers accepted
- Adult, Older adult
- INCLUSION CRITERIA RELATED OR THIRD PARTY DONORS (ALLOGENEIC CELLS, INCLUDING RESEARCH DONORS) AND AUTOLOGOUS DONORS
Subjects who meet ALL of the following criteria will be considered for enrollment into this study:
- Be greater than or equal to 18 years of age, as of the date of enrollment. There is no upper age limit for donor enrollment.
- Able to provide informed consent.
- Meets the donation requirements established by AABB and FDA for allogeneic or autologous use with the exception of hemoglobin/hematocrit.
SUBJECT EXCLUSION CRITERIA ALLOGENEIC DONORS
Subjects who meet ANY of the following criteria will be excluded from participation in this study as a third party donor or research volunteer:
- Medical history that includes any of the following, as per AABB or FDA requirements for allogeneic use:
- Thrombocytopenia or other blood dyscrasias
- Bleeding diathesis
- Antibiotic use within the prior 48 hours
- History of cancer
- History of exposure to transfusion transmitted diseases including HIV and hepatitis B and C as defined by the Standards for Blood Banking and Transfusion Services, AABB.
- Travel to an area where malaria is endemic as defined by the CDC (www.cdc.gov/travel).
- At risk for the possible transmission of Creutzfeldt-Jakob Disease (CJD) and Variant Creutzfeldt-Jakob Disease (vCJD) as described in the FDA Guidance for Industry, January 9, 2002, Revised Preventive Measures to Reduce the Possible Risk of Transfusion of Creutzfeldt-Jakob Disease (CJD) and Variant Creutzfeldt-Jakob Disease (vCJD) by Blood and Blood Products
- Febr le (temperature >38 (Infinite)C)
- Hemoglobin level
- African American women <11.5 grams/dL
- Other women <12.0 grams/dL
- Men <12.5 grams/dL
- HCT
- African American women <34%
- Other women <36%
- Men <38%
- Platelets <150 times 103/microL
- Absolute neutrophil count <1.0 times 103/microL
- Positive tests for blood borne pathogens (as required by the Standards for Blood Banks and Transfusion Services, AABB. The currently required tests include anti-HIV1/2, anti-HCV, anti-HBc, Anti-HTLV I/II, anti-T. Cruzi, HBsAg, syphilis, and molecular testing for West Nile virus, HCV, HBV, and HIV-1).
SUBJECT EXCLUSION CRITERIA AUTOLOGOUS DONORS
Subjects who meet ANY of the following criteria will be excluded from participation in this study:
- Positive tests for anti-HIV1/2, anti-HCV, or HBsAg
- Does not meet criteria established by the NIH IRB-approved treatment protocol for administration of iPSCs, as created by the protocol PI.
About this study
Official title: Collection of Blood From Healthy Volunteers and Clinical Research Subjects for the Production of Clinical Grade Induced Pluripotent Stem Cell (iPSC) Products
Background:
- Pluripotent stem cells are cells that can be converted to any type of cell (muscle, nerve, liver, etc.). Researchers are collecting blood samples to develop way to make a special cell product called induced pluripotent stem cells (iPSCs). iPSCs may be used to repair or replace organs and tissues, produce blood and immune cells for transfusion, or correct inherited immune and blood diseases. iPSCs can be made from many kinds samples, such as skin, blood, or hair. This is different from embryonic stem cells, which can only be derived from embryos.
Read more
Objectives:
- To develop new methods to make iPSCs; to identify better ways to collect, produce, and grow them; and to make an iPSC bank.
Eligibility:
- Healthy adults and adults already in a treatment protocol who have a medical condition that could be treated with iPSCs.
Design:
- All participants will be screened with questionnaire, physical exam, and blood and HIV tests.
- All participants:
- Will donate 4 tablespoons of blood. It will be taken by needle from a vein in their arm.
- Participants already in another protocol:
- Will have their blood collected, separated in a lab, and iPSCs grown in large numbers. Some may have their sample given back to them as a treatment.
- Samples will be kept in the study up to 5 years or until a participant withdraws from the study or becomes ineligible to participate, or the study is closed. If participants have not withdrawn their consent, they may be contacted in the future to donate again.
Read the detailed description
Induced pluripotent stem cells (iPSCs) can be produced from small quantities of blood. Clinical protocols to treat several diseases using iPSCs or cells derived from iPSCs are being developed by intramural investigators, i.e., investigators in National Institute of Allergy and Infectious Diseases (NIAID) and National Eye Institute (NEI). This clinical protocol will allow personnel in the Cell Processing Service (CPS) of the Department of Transfusion Medicine (DTM) to collect blood from healthy donors or patients with diseases targeted in IRB-approved treatment protocols to manufacture iPSCs or iPSC derived products to support these intramural investigators. CPS DTM will submit a Drug Master File (DMF) to the US Food and Drug Administration (FDA) for the manufacture and testing of these products. When iPSCs are used clinically in some cases the donors will be the recipients of the iPSCs, or the ex vivo expanded and differentiated iPSCs (autologous therapy). In other cases, healthy subjects will serve as donors of the blood used as starting material for manufacturing iPSCs (allogeneic therapy) where no matching of HLA or other antigens is required between the healthy blood donor and the iPSC recipient. While methods to produce and expand iPSCs have been described, they must be modified periodically to meet changing Good Manufacturing Practices (GMP) requirements for manufacturing clinical cell and gene therapies, therefore peripheral blood for iPSCs from normal donors is needed for research and development.
- Conditions
- Induced Pluripotent Stem Cells
- Keywords
- Blood Donation, Tissue Procurement, Cellular Therapies
What is being measured
The primary outcome is the main question the study is designed to answer.
collection of blood samples for production of iPSCs or iPSV-derived therapy products
Measured: Ongoing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2014 (estimated)
- Study start5 Feb 2014
- Primary completion1 Nov 2018
- Study completion1 Nov 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Induced Pluripotent Stem CellsStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- David F Stroncek, M.D., Principal investigator, National Institutes of Health Clinical Center (CC)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02056613
- Sponsor's protocol ID
- 140057
- Other IDs
- 14-CC-0057
- Status last verified
- 1 Nov 2018
- First submitted
- 5 Feb 2014
- Last updated
- 5 Nov 2018
- Results posted
- No
Publications
Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell. 2006 Aug 25;126(4):663-76. doi: 10.1016/j.cell.2006.07.024. Epub 2006 Aug 10.
PubMed 16904174Mostoslavsky G. Concise review: The magic act of generating induced pluripotent stem cells: many rabbits in the hat. Stem Cells. 2012 Jan;30(1):28-32. doi: 10.1002/stem.742.
PubMed 21948613Somers A, Jean JC, Sommer CA, Omari A, Ford CC, Mills JA, Ying L, Sommer AG, Jean JM, Smith BW, Lafyatis R, Demierre MF, Weiss DJ, French DL, Gadue P, Murphy GJ, Mostoslavsky G, Kotton DN. Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette. Stem Cells. 2010 Oct;28(10):1728-40. doi: 10.1002/stem.495.
PubMed 20715179
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Collection of Blood From Healthy Volunteers and Clinical Research Subjects for the Production of Clinical Grade Induced Pluripotent Stem Cell (iPSC) Products
- ClinicalTrials.gov ID
- NCT02056613
- Acronym / sponsor ID
- 140057
- Conditions
- Induced Pluripotent Stem Cells
- Keywords
- Blood Donation, Tissue Procurement, Cellular Therapies
- Study type
- Observational
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- National Institutes of Health Clinical Center (CC)US National Institutes of Health
- Study start
- 5 Feb 2014
- Primary completion
- 1 Nov 2018 (actual)
- Study completion
- 1 Nov 2018 (actual)
- First posted
- 6 Feb 2014 (estimated)
- 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.
Background:
- Pluripotent stem cells are cells that can be converted to any type of cell (muscle, nerve, liver, etc.). Researchers are collecting blood samples to develop way to make a special cell product called induced pluripotent stem cells (iPSCs). iPSCs may be used to repair or replace organs and tissues, produce blood and immune cells for transfusion, or correct inherited immune and blood diseases. iPSCs can be made from many kinds samples, such as skin, blood, or hair. This is different from embryonic stem cells, which can only be derived from embryos.
Objectives:
- To develop new methods to make iPSCs; to identify better ways to collect, produce, and grow them; and to make an iPSC bank.
Eligibility:
- Healthy adults and adults already in a treatment protocol who have a medical condition that could be treated with iPSCs.
Design:
- All participants will be screened with questionnaire, physical exam, and blood and HIV tests.
- All participants:
- Will donate 4 tablespoons of blood. It will be taken by needle from a vein in their arm.
- Participants already in another protocol:
- Will have their blood collected, separated in a lab, and iPSCs grown in large numbers. Some may have their sample given back to them as a treatment.
- Samples will be kept in the study up to 5 years or until a participant withdraws from the study or becomes ineligible to participate, or the study is closed. If participants have not withdrawn their consent, they may be contacted in the future to donate again.
Detailed description
Induced pluripotent stem cells (iPSCs) can be produced from small quantities of blood. Clinical protocols to treat several diseases using iPSCs or cells derived from iPSCs are being developed by intramural investigators, i.e., investigators in National Institute of Allergy and Infectious Diseases (NIAID) and National Eye Institute (NEI). This clinical protocol will allow personnel in the Cell Processing Service (CPS) of the Department of Transfusion Medicine (DTM) to collect blood from healthy donors or patients with diseases targeted in IRB-approved treatment protocols to manufacture iPSCs or iPSC derived products to support these intramural investigators. CPS DTM will submit a Drug Master File (DMF) to the US Food and Drug Administration (FDA) for the manufacture and testing of these products. When iPSCs are used clinically in some cases the donors will be the recipients of the iPSCs, or the ex vivo expanded and differentiated iPSCs (autologous therapy). In other cases, healthy subjects will serve as donors of the blood used as starting material for manufacturing iPSCs (allogeneic therapy) where no matching of HLA or other antigens is required between the healthy blood donor and the iPSC recipient. While methods to produce and expand iPSCs have been described, they must be modified periodically to meet changing Good Manufacturing Practices (GMP) requirements for manufacturing clinical cell and gene therapies, therefore peripheral blood for iPSCs from normal donors is needed for research and development.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
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
collection of blood samples for production of iPSCs or iPSV-derived therapy products
Time frame: Ongoing
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2018.
- Background: PubMed 16904174Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell. 2006 Aug 25;126(4):663-76. doi: 10.1016/j.cell.2006.07.024. Epub 2006 Aug 10.
- Background: PubMed 21948613Mostoslavsky G. Concise review: The magic act of generating induced pluripotent stem cells: many rabbits in the hat. Stem Cells. 2012 Jan;30(1):28-32. doi: 10.1002/stem.742.
- Background: PubMed 20715179Somers A, Jean JC, Sommer CA, Omari A, Ford CC, Mills JA, Ying L, Sommer AG, Jean JM, Smith BW, Lafyatis R, Demierre MF, Weiss DJ, French DL, Gadue P, Murphy GJ, Mostoslavsky G, Kotton DN. Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette. Stem Cells. 2010 Oct;28(10):1728-40. doi: 10.1002/stem.495.
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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.
David F Stroncek, M.D. principal investigator
National Institutes of Health Clinical Center (CC)
Questions about this trial
What is this clinical trial studying?
An observational study of Induced Pluripotent Stem Cells, run by National Institutes of Health Clinical Center (CC).
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: withdrawn, as of the sponsor's last update on 5 Nov 2018. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
collection of blood samples for production of iPSCs or iPSV-derived therapy products (time frame: Ongoing). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02056613, which also lists 3 linked publications.
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 5 Nov 2018.