11.0 STATISTICAL CONSIDERATIONS 
This is a Phase I study of an AAV-CFTR vector administered to subjects with cystic 
fibrosis. Demographic and background characteristics obtained at enrollment will be listed 
and summarized. 
The MTD of tgAAVCF vector administered as a single instillation to the nose and/or lung 
lobe will be identified, or the highest dose level will be declared less than the MTD, as 
described in Section 7.4.1. The type and grade of toxicides noted during therapy will be 
summarized for each dose level. All adverse events noted by the investigator will be 
tabulated according to the affected body system. 
Descriptive statisdcs will be used to summarize changes from baseline in the measured 
parameters (Section 8) for each dose cohort. 
12.0 ADMINISTRATIVE CONSIDERATIONS 
12.1 Institutional Review Board 
In accordance with federal regulations (21 CFR 312.66), an Institutional Review Board 
(IRB) that complies with regulations in 21 CFR 56 must review and approve this protocol 
and the informed consent form prior to initiation of the study. The Investigator will submit 
to the Sponsor a list of the names, occupations, and affiliations of the members of the IRB. 
No supplies will be shipped until the IRB has given its approval and the Sponsor has been 
notified of this fact in writing. 
12.2 Consent 
The Principal Investigator or his associate must explain verbally and in writing the nature, 
duration, and purpose of the study and possible consequences of treatment. Patients must 
also be informed that they may withdraw from the study at any time and for any reason 
without jeopardizing their future treatment. In accordance with federal regulations (21 CFR 
3 12), all patients must sign the IRB-approved consent form in the presence of a witness. 
Prior to the start of the study, a copy of the IRB-approved consent form must be submitted 
to the Sponsor. 
12.3 Study Records 
The Sponsor will provide the Investigator with records of drug shipments, case report 
forms designed to collect the data specified for each individual, and other forms as 
necessary. 
The Investigator and/or institution is required: 
1. To prepare and maintain these forms in accordance with federal regulations (set 
forth in the statement of investigator form FDA 1572) and to sign, date, and return 
them to the Sponsor. 
2. To return all unused study drug supplies to the Sponsor at the conclusion of this 
study. 
3. To maintain a record of drug disposition and the case report forms for a period of 
two years following the date a Product License Application is approved for the drug 
indication described in this protocol. If the application is not approved, records 
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