Gene Therapy for CF using Cationic Liposome Mediated Gene Transfer: Phase I Trial 
III.E. APPENDICES 
TABLE 3 
Inclusion Criteria 
a. Diagnosis of CF based upon sweat chloride level greater than 60 mEq/1 together 
with clinical manifestations of CF. 
b. Male and/or female patients greater than 18 years of age. Males entered into the 
study must have documented azoospermia. More than 95% of CF males are 
infertile and have azoospermia associated with congenital vas deferens atrophy. 
c. Mild to moderate pulmonary disease (FEV, greater than 40% predicted) and no 
pulmonary exacerbation within the four weeks prior to entry into the study. 
d. Genotype: Because of likely heterogeneity in the molecular pathogenesis of CF, 
homozygous Delta F508 patients will be preferred. Other CF patients will be 
eligible if they both 1) are compound heterozygotes for the F 508 mutation and 
2) have pancreatic insufficiency. 
Exclusion Criteria: 
a. Patients demonstrating evidence of sequelae of CF which might make participa- 
tion in this study difficult or of higher risk will be excluded. 
1) . Severe cardiac, hepatic or other organ system disease, including juvenile- 
type (insulin-dependent) diabetes mellitus. 
2) Acute or recurrent sinusitis or history of obstructive nasal polyposis. 
3) P0 2 less than 60 mm mercury assessed by room air blood gas. 
4) Women not practicing birth control for at least one month prior to 
admission into the study, or with a positive pregnancy test during the pre- 
study evaluation. 
5) Unstable lung disease, with worsening pulmonary symptoms, arterial 
blood gases, or pulmonary function tests within the two weeks prior to 
entry into the study. 
6) Corticosteroid therapy within the previous four months. 
7) Pneumothorax or marked hemoptysis (e.g., greater than 250 cc of blood 
within a 24-hour period) within the past year. 
8) Inability in the opinion of the investigators to comply with or comprehend 
the protocol. 
b. DNase use or participation in another experimental clinical study within the past 
two months. 
[844] 
Recombinant DNA Research, Volume 18 
