Biomedical Engineering Reference
In-Depth Information
Table 5.1
The most relevant clinical trials in gene therapy
Trial
Phase
N
(Treated/
Control)
Agent
Vector
Delivery
method
Patients
Outcomes
AGENT-1[
30
]
Grines et al. 2002
I-II
60/19
FGF4
AdV
IC
Angina II-III patients
Improvement in exercise time
AGENT-2[
31
]
Grines et al. 2003
II
35/17
FGF4
AdV
IC
Angina II-IV patients (no-option for
revascularization)
; Infarct size
AGENT-4[
32
]
Henry et al. 2007
III
78 (2CD)/
38
FGF4
AdV
IC
Angina pectoris (no-option for
revascularization)
No significant effect in exercise
tolerance
AWARE[
32
]
Henry et al. 2007
III
300
FGF4
AdV5
IC
Angina pectoris (no-option for
revascularization)
Non provided
KAT301 Trial[
33
,
34
]
Hedman et al. 2003 &
2009
II-III
28/37
VEGF165
AdV vs
plasmid
liposomes
IC
Angina II-III patients undergoing
coronary angioplasty and stenting
procedure on restenosis and
myocardial perfusion
Improvement in LV function
(No adverse effects 8 years later)
Euroinject One Study[
35
]
Kastrup et al. 2005 [
35
]
II-III
40/40
VEGF165
Naked plasmid
IM
Angina III-IV patients (no-option for
revascularization)
Improvement in wall motion and LV
function.
NORTHERN[
36
]
Stewart et al. 2009
II-III
48/45
VEGF121
AdV
PC
Angina III-IV patients (no-option for
revascularization)
: Revascularization
REVASC[
37
]
Stewart et al. 2006
II
32/35
VEGF121
AdV
EP
Patients with severe angina due to
coronary artery disease (no-
option for revascularization)
Non provided
CUPID
Jaski et al. 2009[
38
];
Jessup et al, 2011[
39
]
II
25 (3CD)/
14
SERCA2a
AAV1
PC
III-IV Angina patients
Improvements in symptomatic and
LV function/remodeling
parameters
(No adverse effects)
NCT00787059[
30
]
Grines et al. 2002
I-II
Ongoing
(*72)
AC6
AdV5
IC
Congestive Heart Failure
Non provided
NCT01082094[
31
]
Grines et al. 2003
I
Ongoing
(*16)
ACRX-100
(SDF-
1)
Naked plasmid
IC
Heart Failure
Non provided
