Page 1193 - Clinical Immunology_ Principles and Practice ( PDFDrive )

1158 ParT TEN Prevention and Therapy of Immunological Diseases

TABLE 85.2 Clinical Trials of Gene Therapy for Primary Immune Deficiencies
PID Investigator(s) Year Vector/Target Conditioning NCT #
ADA SCID Blaese, Anderson, Culver 1990 LASN (MLV LTR) None —-
PBMC
Bordignon, Mavilio 1992 G1ADA None —-
(MLV LTR)
PBMC and BM
Hoogerbrugge, Valerio 1993 MLV-ADA None —-
BM CD34 +
Kohn, Parkman 1993 G1NA-ADA (MLV LTR) None —-
UCB CD34 +
Onodera/Sakiyama 1995 LASN None —-
(MLV LTR)
PBMC
Aiuti/Roncarolo 1998 G1ADA Busulfan 4 mg/kg 00599781
(MLV-LTR)
BM CD34 +
Gaspar/Thrasher 1999 SFFV-ADA-wpre Melphalan; busulfan —-
BM CD34 +
Otsu/Ariga 2003 GCsap-M-ADA BM CD34 + None —-
Kohn/Candotti 2001 MND-ADA and GCsap-M-ADA None (4). —-
BM CD34 + Busulfan (6)
GlaxoSmithKlein 2008 G1ADA Busulfan 00598481
BM CD34 +
Kohn/Candotti 2009 MND-ADA Busulfan 90 mg/m2 00794508
BM CD34 +
Gaspar 2012 EFS-ADA Busulfan 01380990
+
CD34 BM/PBSC
Kohn 2013 EFS-ADA Busulfan 01852071
BM CD34 +
XSCID Cavazzana-Calvo/ Fischer 1998 MFG-IL2Rg None —-
BM CD34 +
Thrasher/Gaspar 1999 MLV-IL2Rg None —-
BM CD34 +
Thrasher/Fischer/ 2011 SIN γRV EFS-IL2Rg None Fr: 01410019
Cavazzana/Pai/ Williams BM CD34 + UK: 01175239
US: 01129544
Sorrentino 2012 EFS-IL2Rg-Ins None/ Busulfan 01512888
BM CD34 +
Malech/DeRavin 2011 EFS-IL2Rg-Ins Busulfan 01306019
CD34 PBSC
+
LAD Hickstein, Bauer 1999 MLV-CD18 None —-
BM
CGD Malech 1995 MLV-p47 None —-
+
CD34 PBSC
Malech 1998 MLV-gp91phox None —-
Ott/Grez 2009 SFFV-gp91phox 8 mg/kg 00927134
CD34 PBSC
+
Kang/Malech 2010 MT-gp91phox 10 mg/kg —-
+
CD34 PBSC
Thrasher/ Serve/ 2013 pChim-gp91phox (G1XCGD) pK adjusted, ~12 mg/kg 01855685
Reichenbach CD34 PBSC
+
Kohn/Williams/Kang 2015 pChim-gp91phox (G1XCGD) pK adjusted, ~12 mg/kg 02234934
+
CD34 PBSC
WAS Klein 2008 SFFV-WAS gRV Busulfan —-
Aiuti 2011 1.6hWASP-WPRE LV Bu/Flu/Rituximab 01515462
Hacein-Bey Abina… 2011 Bu/flu (rituximab or alemtuzimab 02333760
Cavazzana for auto-immunity)
Thrasher 2011 Bu/Flu 01347242
Pai 2011 Bu/Flu 01410825
CGD, chronic granulomatous disease; LAD, leukocyte adhesion defect; PID, primary immune deficiencies; SCID, severe combined immune deficiency; WAS, Wiskott-Aldrich
syndrome; XSCID, X-linked SCID.