Genzyme Corporation Ad2/CFTR-2 Gene Transfer Protocol: Single 
One Kendall Square, Cambridge, MA 02139 Aerosol Administration to the Lung of CF Patients 
radiological appearance of infiltrates, or other evidence of an inflammatory or other adverse effect, a CT scan and/or 
bronchoscopy with BAL may be performed earlier than what is described in the study procedures section of this 
protocol. At all visits the patient will be queried for the presence of any adverse events and concomitant medication 
information will be collected. 
Bronchoscopy : Bronchoscopy will be performed on Day 7. Bronchoscopy will be performed under local anesthesia of 
the upper and lower airways with topical application of Lidocaine and I.V. sedation, according to the policy and 
procedures of each of the participating medical centers. The patient will be NPO for 4-8 hours prior to the 
bronchoscopy. The patients will be premedicated with a combination of Demerol, Midazolam, Atropine, and/or Chloral 
Hydrate prior to the procedure. Oxygen will be administered throughout the procedure. Topical anesthesia with 
Lidocaine 1-2 % will be applied directly to the nose, pharynx and larynx. Anesthesia of the lower airways with Lidocaine 
1-2 % will be applied directly through the suction channel. Sedation will be maintained with Midazolam 0.1 -0.2 mg/kg, 
slow I.V. infusion. 
O 2 saturation, heart rate and respiratory rate will be monitored at all times during bronchoscopy using a pulse oximeter. 
Each parameter will be recorded every 5 minutes during the procedure. Any abnormalities that occur will be recorded. 
The procedure will be stopped at any time at which there is a sustained O 2 desaturation below 85% and/or decrease in 
heart rate below 60 and/or apnea. Once the procedure is completed, the patient will be observed for a minimum of 1-2 
hours. 
Brushing : The brush is inserted through the suction channel to the desired position. The brush is moved back and forth 
through the examined area. The brush alone is removed after brushing the initial lobe(s). The brush should be 
withdrawn from the final brushing procedure within the bronchoscope. These specimens from each brushing will be 
tested to determine the presence of Ad2/CFTR-2. It will involve 4 segments of the lower lobe(s) identified by HRCT and 
chest x-ray as having a high probability of receiving delivery of Ad2/CFTR-2. 
STUDY VARIABLES 
Medical History, Physical Examination & Vital Signs 
Purpose : To assess: 1) The patient's medical history, 2) The patient's general medical condition, 3) Evidence of 
patient discomfort, systemic responses, or inflammation. 
Methods : Study patients will be queried as to their medical history of various body system specific medical conditions. 
At each visit of the study, patients will undergo a complete or limited general physical examination, as designated in the 
protocol, and be questioned about discomfort and/or local or systemic manifestations of inflammation. Vital signs will 
also be recorded. The presence of clinical symptoms such as headache, local pain and/or bleeding, purulent 
rhinorrhea, pharyngitis, conjunctivitis, pneumonia, transient diarrhea and gastroenteritis will be closely monitored. 
Systemic symptoms such as fever and/or leucocytosis will be also be closely followed. 
CF Symptom Assessment 
Purpose : To assess: 1) The patient's CF history, 2) The patient's symptoms of CF, 3) Evidence of patient respiratory 
discomfort or inflammation. 
Methods : Study patients will be queried as to their CF history including when they were diagnosed, genotype, family 
history, most recent hospitalization(s) related to CF and current CF therapy. At each visit of the study, patients will 
undergo an evaluation of respiratory, nasal and gastrointestinal: 1) symptoms of CF, 2) symptoms of discomfort and 3) 
manifestations of inflammation or other airway obstruction. 
Specific attention will be placed on respiratory symptoms such as inflammation, edema, erythema, discharge/exudate, 
denudation, chronic cough and/or wheezing, dyspnea, hemoptysis, hypoxia, and/or airway obstruction. 
General Safety Laboratory Analysis: Blood and Urine 
Purpose : To assess the patient's systemic response to Ad2/CFTR-2 application. 
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