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Peripheral Blood Stem Cells Obtained From Normal Volunteers for Studying Retroviral Vector Mediated Gene Transfer Into Primitive Hematopoietic Cells and Vector Mediated Transgene Expression in Mature Hematopoietic Lineages

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00758992
First Posted: September 25, 2008
Last Update Posted: March 14, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
National Heart, Lung, and Blood Institute (NHLBI)
Doris Duke Charitable Foundation
Information provided by (Responsible Party):
St. Jude Children's Research Hospital
  Purpose
These studies are designed to evaluate the relative efficiency of gene transfer into primitive human hematopoietic cells by comparing lentiviral and foamy virus vectors as vehicles for transfer and expression of globin genes. Normal volunteers will serve as research participants. Each will receive a 4 day course of Granulocyte-Colony Stimulating Factor (G-CSF) after which a peripheral blood apheresis will be performed to recover a mononuclear cell population enriched in primitive hematopoietic cells. The stem and progenitor cells will be purified by selection based on expression of the CD34 antigen. The CD34+ population will be cultured in vitro with various cytokines and transduced with vector particles. The efficiency of gene transfer will be evaluated in the transduced CD34+ population, in progenitors contained within that population by culture in semisolid media and in cells capable of establishing human hematopoiesis in immunodeficient mice. The level of transgene expression will be evaluated in mature hematopoietic lineages that develop in vitro or in immunodeficient mice.

Condition
Immunodeficiency

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Peripheral Blood Stem Cells Obtained From Normal Volunteers for Studying Retroviral Vector Mediated Gene Transfer Into Primitive Hematopoietic Cells and Vector Mediated Transgene Expression in Mature Hematopoietic Lineages

Resource links provided by NLM:


Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
  • To compare the relative efficiencies of lentiviral and foamy virus vectors in transducing primitive hematopoietic cells mobilized into peripheral blood by cytokine administration. [ Time Frame: Up to 3 years after apheresis collection of donor cells. ]

Biospecimen Retention:   Samples With DNA
Blood

Enrollment: 14
Study Start Date: October 2006
Study Completion Date: July 2015
Primary Completion Date: July 2015 (Final data collection date for primary outcome measure)
Groups/Cohorts
Immunodeficient mice
Please see the Study Description for complete information.

  Show Detailed Description

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 40 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Healthy Adult Research Participants
Criteria

Inclusion Criteria:

  • Age greater than or equal to 18 years and less than or equal to 40.
  • Meets donation criteria for autologous blood donors per SJCRH Blood Donor Center standard operating procedure.
  • Adequate venous access for apheresis on examination as per the judgment of the Blood Donor Center nursing staff, physician staff or physician investigators.

Exclusion Criteria:

  • Females - lactating.
  • Concurrent use of systemic medications that in the judgment of the physician investigators adversely affect platelet function, such as aspirin or non-steroidal antiinflammatory agents.
  • Any of the following diagnoses (prior or current):

    • pulmonary disease.
    • inflammatory disorder.
    • coronary artery disease.
    • stroke (cerebral vascular accident).
    • hypertension.
    • cardiac arrhythmias.
    • venous thrombosis.
    • pulmonary embolus
    • hematological disease.
    • eczema or psoriasis.

Additional eligibility criteria (to be obtained after signing informed consent)

  • Body mass index less than 30 kg/m2.
  • No hepatomegaly or splenomegaly.
  • Hemoglobin greater than or equal to 12.5 g/dL.
  • Platelet count greater than or equal to 150,000/mm3.
  • Total WBC > 4200/ul and neutrophil count >1800/ul.
  • Females - not pregnant (negative serum or urine; to be obtained after signing informed consent).
  Contacts and Locations
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): NCT00758992


Locations
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)
Doris Duke Charitable Foundation
Investigators
Principal Investigator: Arthur W Nienhuis, MD St. Jude Children's Research Hospital
  More Information

Additional Information:
Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00758992     History of Changes
Other Study ID Numbers: PBSCRV
P01HL053749 ( U.S. NIH Grant/Contract )
2010037 ( Other Identifier: Doris Duke Foundation )
First Submitted: September 18, 2008
First Posted: September 25, 2008
Last Update Posted: March 14, 2016
Last Verified: March 2016

Keywords provided by St. Jude Children's Research Hospital:
gene transfer

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Immune System Diseases