Gene Therapy for CF using Canonic Liposome Mediated Gene Transfer: Phase I Trial 
2. EKG (12 lead), urinalysis. 
3. Chest x-ray (PA and lateral), arterial blood gases obtained on room air, oximetry, sinus 
films, pulmonary function tests (including spirometry, lung capacity measurements, and 
diffusion capacity), and sputum culture. 
4. Nasal endoscopy, nasal washes for culture, nasal brushings and biopsy and for 
histopathology. 
5. Sperm count for males not already known to be azoospermic; pregnancy test for females 
prior to entry into the study. 
6. Nasal transepithelial potential difference measurements. 
7. Serum sample to measure baseline antibody titers against lipid/DNA conjugates. 
ULE. Clinical Protocol 
1. Study Design. This is a non-randomized, non-blinded Phase I trial of Lipid/DNA 
conjugates for use in gene transfer of CFTR to respiratory nasal epithelium. 
2. Pre- treatment evaluation. Patients will enter the study and written, informed consent will 
be obtained prior to participation in the screening evaluation. Pre-treatment evaluation 
will be performed 4 weeks prior to gene transfer and at the time of admission to the 
Clinical Research Center (Day 1, see below) and will include: 
a. Complete history, chart review. 
b. Physical examination. 
c. Serum chemistry evaluation (including serum alkaline phosphatase, bilirubin, 
LDH, BUN, creatinine, albumin, calcium, phosphate, sodium, potassium, 
chloride, C0 2 , glucose measurements) and complete blood count (including 
platelets and differential), urinalysis, as well as CF genotype analysis. 
d. Sperm count on males not previously known to be azoospermic; pregnancy test 
on females. 
e. Nasal endoscopy. 
f. Serum for analysis of antibody to DNA/lipid conjugates (baseline studies). 
g. Pulmonary function tests, including diffusion capacity. 
h. Arterial blood gases and oximetry. 
i. 12-lead EKG. 
j. PA and lateral chest x-ray. Nasal/sinus x-rays. 
k. Sputum culture and antibiotic sensitivity. 
l. Measurement of nasal potential difference. 
m. Nasal brushings/washings. 
In addition, at 14d and Id prior to gene transfer, patients will undergo history and physical 
examinations, oximetry, nasal potential difference measurements, and serum sampling for 
antibody to DNA/lipid conjugates (baseline studies). 
3. Gene transfer procedures. 
Patients will be admitted to a private room in the Clinical Research Center at The 
Children’s Hospital of Alabama on the day of gene transfer. The patient will be placed 
in a left lateral decubitus position and given mild sedation (Midazolam, 2-3 mgs IM). 
Nasal endoscopy (i.e., flexible or short rigid [Hopkins rod] examination) will be used to 
Recombinant DNA Research, Volume 18 
[829] 
