MEDICAL RECORD 
CONSENT TO PARTICIPATE IN A CLINICAL RESEARCH STUDY 
• Adult Patient or • Parent, for Minor Patient 
INSTITUTE' National Heart, Lung, and Blood Institute 
STUDY NUMBER PRINCIPAL INVESTIGATOR: 
STUDY TITLE' Gene Therapy of the Respiratory Manifestations of 
Ronald G. Crystal, M.D. 
Cystic Fibrosis Using a 
Replication Deficient, Recombinant Adeno virus to Transfer the Normal Human Cyoti- Fibrosis 
Transmembrane Conductance Regulator cDNA to tne Airway Epithelium. 
INTRODUCTION 
We invite you (or your child) to take part in a research study at the National Institutes of Health. It is important that 
you read and understand several general principles that apply to all who take part in our studies: (a) taking part in | 
the study is entirely voluntary; (b) personal benefit may not result from taking part in the study, but knowledge may 
be gained that will benefit others; (c) you may withdraw from the study at any time without penalty or loss of any 
benefits to which you are otherwise entitled. The nature of the study, the risks, inconveniences, discomforts, and 
other pertinent information about the study are discussed below. You are urged to discuss any questions you have 
about this study with the staff members who explain it to you. 
Purpose and Background 
. 
The purpose of this consent document is to explain to you the possible risks 
and benefits of an experimental gene therapy protocol for the treatment of 
cystic fibrosis. You will receive a copy of the protocol itself, which is 
referred to throughout this consent document. We encourage you to read the 
protocol and to ask as many questions as you feel are necessary to help you 
understand and make an informed decision regarding participation in this 
clinical trial. 
It is important for you to understand that this is an initial trial designed 
to evaluate toxicity (possible side effects) and efficacy (possible positive 
results) . There is no guarantee that this experimental gene therapy will 
result in an improvement in your lung function, general health, or prognosis 
(future health). The definite benefits of participating in this initial 
toxicity/efficacy study are unknown. There is a small possibility, however, 
that the experimental treatment will slow or prevent the progression of the 
manifestations of cystic fibrosis in your lung. The trial will take a 
considerable amount of time (a minimum of 4 months) and it involves risks that 
are important for you to understand. 
Individuals with cystic fibrosis (CF) have abnormal lung function because they 
lack the presence of a normal functioning gene, called the "cystic 
transmembrane conductance regulator" (CFTR) gene, in the cells lining the 
lung. Instead, such individuals contain in their lung cells mutations 
(alterations in the normal DNA sequence) in the CFTR genes which they have 
inherited from both parents. These mutations cause CF because of the 
inability of the mutated gene to produce ah effective CFTR protein. This 
protocol attempts to compensate for this abnormality by introducing a normal 
CONSENT TO PARTICIPATE IN A CLINICAL 
RESEARCH STUDY 
Recombinant DNA Research, Volume 16 
PATIENT IDENTIFICATION 
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