December 1-2, 1994, Data Management Report -- Page 
Attachrant IV - I^ge 6 
Protocol 
Status 
Pending 
NIN and 
FDA 
approval 
Open* 
(* triable 
to obtain 
purging 
antibodies 
and 
vectors In 
combina- 
tion) 
Open 
Open 
* Deaths 
VN 
< 
Z 
8 
PD 
O ! 
Adverse 
Effects 
VN 
< 
z 
Tea 
Fever, chills, 
diarrhea, skin 
irritation 
Yes 
(Published) 
1 patient 
mi Id to 
moderate 
fatigue, 
fever, 
hypoxemia, 
pulmonary 
infiltrate, 
lung function 
abnormalities 
(all symptoms 
transient) 
Problems 
Associated 
With Gene 
Transfer 
< 
z 
NA 
No 
No 
Evidence Of 
Biological 
Activity 
< 
z 
< 
z 
a. 
Yes 
(Publ Ished) 
In Vivo Evidence Of 
Gene Transfer And 
Expression In 
Target Cells 
VN 
< 
z 
VN-A 
T-YesG/YesP 
(Published) 
•Vector-mediated 
CFTR SANA and CFTR 
protein in the 
respiratory 
epithelium 
Patients 
Entered On 
Study 
o 
o 
16 
(5 melanoma) 
(1 renal cell) 
(2 colon) 
(5 edeno) 
(1 clear cell) 
(2 Infiltrat- 
ing duct) 
K> 
5 
O — ' 
Maximus 
Patient 
Accrual 
14 
<H/F) 
12 
(M/F) 
20 
(M/F) 
24 
(M/F, >15 yr) 
10 Lung 
14 Nasal 
Divided 
between N1H, 
Cornell, and 
Rockefeller 
U. 
fl 
HA 
119 
GTI 
Cornel l 
N I H 
Vector Construct 
Packaging Cell Line 
(Delivery Vehicle) 
a> 
SI 
z 
•Ti eg 3 
i i I 
< ♦ « 
z a. ec 
J O w 
o 
%> 
z 
a 
z 
5 i 
s ■; 
z o 
V. h* u 
— 1 K1 K 
z < oe 
— < a. w 
G1!L4SvNa 
PA317 
(Retrovirus) 
AdCFTR 
293 
(Adenovirus) 
Dates of 
T reatment : 
(1) First Patient 
(2) Most Recent 
Patient 
< 
< 
z 
(1) 9-9-93 
(2) 10-17-94 
K> 
o* o* 
N- *0 
»- n j 
•jr O* 
*- rg 
Category 
T/C 
(NSCLC) 
5 CD 
^ Z 
X W 
3/A/l 
I/IG0/CF 
t 
** 
9 
a 
[ J s > 
5 s > e 
3*> ? ? 
U ! 1 
C PO O. Cl. 
" , N V N. 
k <_> Z < 
M < — O 
c3 Of z u. 
RAC/9-15-92 
NIH/2-5-93 
FDA/ 
RAC/9-15-92 
NIH/2-5-93 
F0A/9-8-93 
FDA/1-13-94 
rg po ki 
0*0*0* i 
pn *o *o 
<\l a . 
*- 4# *1 
V X 
U X < 
< — o 
OC Z u- 
pi/ 
Institution 
Roth/ 
MD Anderson 
- « 
? 
k ** 
CD l/> 
J •& 
* i 
4» • 
• +* 
• — 
n a. 
o 
-i 3 
Crystal/ 
Cornell, 
Rockefeller U., 
NIH 
NIH/RAC f 
(FDA ISO •) 
9403-031/ 
9209-031 
(Pending) 
9209-032 
n 
IO 
o 
s 1 
o w 
1 
o 
eg 
5 * 
[ 513 ] 
