Gene Therapy for CF using Cationic Liposome Mediated Gene Transfer: Phase I Trial 
3. Because I do not have a normal CF gene, it is possible that if cells within my 
nose begin to use a normal CF gene, my body will recognize the CF protein 
which results as new and foreign, and attempt to destroy cells making the protein. 
I understand that animal studies have suggested that too much normal CF protein 
does not appear to cause any serious side effects, and although the risk is likely 
to be very small the effect in humans is unknown. 
4. Other issues: 
a) I am aware that participation in Phase I studies such as this one sometimes 
may make it more difficult to gain entry into future experimental trials, 
although this is not intended by the physicians responsible for the study. 
I am aware that participation in this study could exclude me from later 
gene therapy or other experimental trials, until gene transfer has become 
a proven form of beneficial, and commercially available, therapy. 
b) Throughout the study I will continue to be monitored for complications 
which are normally associated with CF. Should any of these complications 
develop I will be given appropriate therapy. If the complications do not 
appear to be related to the gene transfer protocol, the usual means of 
payment (for example, insurance) should be arranged. UAB and The 
Children’s Hospital of Alabama have made no provision for monetary 
compensation in the event of physical injury resulting from the research 
and in the event of such injury, medical treatment is provided, but is not 
provided free of charge. 
c) I have been informed that my participation in this study requires the 
practice of birth control because of the very small risk of the CF gene 
reaching the sperm or egg cell. If I conceive an infant unexpectedly during 
this study I will notify the medical researchers immediately. 
d) Despite the best efforts of the medical research team to maintain my 
confidentiality as part of this study, the public interest in gene therapy 
makes it possible that the news media or others could attempt to discover 
my identity. Although everything possible will be done to protect my 
privacy, if my identity is discovered through some other means I could be 
asked for interviews and it is possible that my privacy might be reduced 
in other ways due to my participation in this study. 
e) Because gene administration is a new form of therapy, close follow up 
with the medical researchers in this study will be required. Even after the 
five-month study is completed, I will continue to be followed for my 
cystic fibrosis, and will have a blood test and chest x-ray performed as 
part of my regular care approximately one year following gene transfer. 
I also understand that although the medical research team does not expect 
any substantial harmful effects to result from this study, the effects of 
gene transfer administration which I experience may be very important in 
terms of helping other patients with CF and possibly other diseases as 
well. This means that in the case of my death (whether this is due to a 
CF-related problem or to some other cause, such as an accident which has 
nothing to do with CF) an autopsy will be requested so that the effects of 
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