Phase I Study of Retrovirus-Mediated Transfer of cDNA for Human CD18 Into Peripheral Blood Repopulating Cells of Patients With Leukocyte Adherence Deficiency

2014-08-27 03:58:14 | BioPortfolio



I. Investigate the efficiency and safety of transducing the human CD18 DNA into filgrastim (G-CSF) mobilized, CD34 enriched peripheral blood repopulating cells from patients with a severe or moderate deficiency form of leukocyte adherence deficiency (LAD) by retrovirus-mediated gene transfer.

II. Investigate whether the human CD18 cDNA is expressed sufficiently to benefit patients with this disease.

III. Determine whether repeated infusions of transduced peripheral blood repopulating cells increase the number of surface CD11/CD18 positive cells in these patients.

IV. Determine the extent of long-term persistence of transduced peripheral blood repopulating cells in LAD patients not receiving prior myeloablation.



Patients receive filgrastim (G-CSF) subcutaneously daily on days 1-5 with peripheral blood cell collections on days 4 and 5. Patients receive infusions of transduced CD34+ peripheral blood cells on day 8. Patients may be offered a second course of transduced peripheral blood cells for a total of 4 infusions.

Patients are followed monthly for one year and then annually for 4 years.

Study Design

Primary Purpose: Treatment


Leukocyte Adhesion Deficiency Syndrome


filgrastim, Retrovirus vector LgCD18


Active, not recruiting


Office of Rare Diseases (ORD)

Results (where available)

View Results


Published on BioPortfolio: 2014-08-27T03:58:14-0400

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Medical and Biotech [MESH] Definitions

Rare, autosomal recessive disorder caused by deficiency of the beta 2 integrin receptors (RECEPTORS, LEUKOCYTE-ADHESION) comprising the CD11/CD18 family of glycoproteins. The syndrome is characterized by abnormal adhesion-dependent functions, especially defective tissue emigration of neutrophils, leading to recurrent infection.

Cell-surface glycoprotein beta-chains that are non-covalently linked to specific alpha-chains of the CD11 family of leukocyte-adhesion molecules (RECEPTORS, LEUKOCYTE-ADHESION). A defect in the gene encoding CD18 causes LEUKOCYTE-ADHESION DEFICIENCY SYNDROME.

Family of proteins associated with the capacity of LEUKOCYTES to adhere to each other and to certain substrata, e.g., the C3bi component of complement. Members of this family are the LYMPHOCYTE FUNCTION-ASSOCIATED ANTIGEN-1; (LFA-1), the MACROPHAGE-1 ANTIGEN; (Mac-1), and the INTEGRIN ALPHAXBETA2 or p150,95 leukocyte adhesion protein. They all share a common beta-subunit which is the CD18 antigen. All three of the above antigens are absent in inherited LEUKOCYTE-ADHESION DEFICIENCY SYNDROME, which is characterized by recurrent bacterial infections, impaired pus formation, and wound healing as well as abnormalities in a wide spectrum of adherence-dependent functions of granulocytes, monocytes, and lymphoid cells.

A group of three different alpha chains (CD11a, CD11b, CD11c) that are associated with an invariant CD18 beta chain (ANTIGENS, CD18). The three resulting leukocyte-adhesion molecules (RECEPTORS, LEUKOCYTE ADHESION) are LYMPHOCYTE FUNCTION-ASSOCIATED ANTIGEN-1; MACROPHAGE-1 ANTIGEN; and ANTIGEN, P150,95.

Movement of tethered, spherical LEUKOCYTES along the endothelial surface of the microvasculature. The tethering and rolling involves interaction with SELECTINS and other adhesion molecules in both the ENDOTHELIUM and leukocyte. The rolling leukocyte then becomes activated by CHEMOKINES, flattens out, and firmly adheres to the endothelial surface in preparation for transmigration through the interendothelial cell junction. (From Abbas, Cellular and Molecular Immunology, 3rd ed)

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