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 is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by St. Jude Children's Research Hospital
Sponsor:
Collaborators:
Doris Duke Charitable Foundation
Information provided by (Responsible Party):
St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:
NCT00758992
First received: September 18, 2008
Last updated: July 10, 2014
Last verified: July 2014
  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 Intervention
Healthy Volunteers
Drug: G-CSF
Procedure: Stem Cell Apheresis

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
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: January 2015 ] [ Designated as safety issue: No ]

Estimated Enrollment: 24
Study Start Date: October 2006
Estimated Study Completion Date: January 2017
Estimated Primary Completion Date: May 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
1 Drug: G-CSF
Growth Factor, Granulocyte- Colony Stimulating Factor (G-CSF) will be given for 4 days.
Procedure: Stem Cell Apheresis
Stem Cell Apheresis is the procedure for collecting stem cells from the blood. The process uses a machine to draw blood from a vein in one arm and pass it through a machine that separates the blood into parts ( plasma, platelets, white blood cells, and red blood cells). One or more of these parts will be removed from the blood. The remaining parts will be mixed together and returned through a vein in the opposite arm from which the blood was initially drawn.

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years to 40 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00758992

Contacts
Contact: Arthur W Nienhuis, MD 1-866-278-5833 info@stjude.org

Locations
United States, Tennessee
St. Jude Children's Research Hospital Recruiting
Memphis, Tennessee, United States, 38105
Contact: Arthur W Nienhuis, MD    866-278-5833    info@stjude.org   
Principal Investigator: Arthur W Nienhuis, MD         
Sponsors and Collaborators
St. Jude Children's Research Hospital
Doris Duke Charitable Foundation
Investigators
Principal Investigator: Arthur W Nienhuis, MD St. Jude Children's Research Hospital
  More Information

Additional Information:
No publications provided

Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT00758992     History of Changes
Other Study ID Numbers: PBSCRV, P01HL053749, 2010037
Study First Received: September 18, 2008
Last Updated: July 10, 2014
Health Authority: United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on October 02, 2014