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

NCT00758992 | Completed

• 3 other identifiers: PBSCRVP01HL0537492010037
• Study Type: observational
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Immunodeficiency

Keywords

gene transfer

Outcome Measures

Primary Outcomes (1)

• To compare the relative efficiencies of lentiviral and foamy virus vectors in transducing primitive hematopoietic cells mobilized into peripheral blood by cytokine administration.

  Up to 3 years after apheresis collection of donor cells.

Study Arms (1)

Immunodeficient mice

Please see the Study Description for complete information.

Eligibility Criteria

• Age: 18 Years - 40 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Healthy Adult Research Participants

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator
• Doris Duke Charitable Foundation: collaborator

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

Biospecimen

Retention: SAMPLES WITH DNA

Blood

MeSH Terms

Conditions

Immunologic Deficiency Syndromes

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Officials

• Arthur W Nienhuis, MD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 18, 2008
• First Posted: September 25, 2008
• Study Start: October 1, 2006
• Primary Completion: July 1, 2015
• Study Completion: July 1, 2015
• Last Updated: March 14, 2016

Record last verified: 2016-03

Locations

US (1)