Attachment II - Pg. 8 
Clinical and Public Health Considerations 
Describe the type of cells that will be used for gene transfer. Describe 
the exact nature of the mode of delivery to the patient. 
What volume of treated cells will be used?. Will there be a single or 
multiple treatments for gene transfer? If multiple treatments, over what 
period of time? 
Describe the number of patients that will be involved in the study. 
What are the clinical endpoints of the study? How will patients be 
monitored to assess specific effects of treatment on the disease. 
Describe clinical and laboratory follow-up studies. How frequently will 
such studies be done? 
N.B. Note that federal rules require periodic reporting to the local IRB. 
How often will this be done? In addition, it is requested that the 
investigators report to the working groups of the Recombinant DNA 
Advisory Committee regarding followup of the gene transfer experiment at 
six and twelve month intervals following gene therapy. 
Describe tests that will be done to assess possible toxic or side effects 
of gene therapy. 
Will there be long-term patient follow-up? If a patient should die, will 
an autopsy be requested and will special autopsy studies be done? 
Is there any conceivable danger to others than treated patients? 
What dangers are postulated and what precautions will be taken, if any, 
to protect others (i.e. patients sharing a room, health-care workers or 
family members) from risk such as the low probability of retroviral 
infection? 
If there are possible dangers from gene therapy to patients, present 
cost-benefit considerations. As an example, with a lethal disease a 
slight but definite hazard of neoplasm some years after gene therapy 
might be acceptable while such a hazard would be completely unacceptable 
in using a new vaccine on healthy people. 
What professional personnel (medical and non-medical) will be involved in 
the proposed study? What are their specific qualifications and their 
experience with respect to the disease to be treated? Provide curricula 
vitae. At what hospital or clinic will the treatment be given? Which 
facilities of the hospital or clinic will be expecially important for 
this study? Will patients occupy regular hospital beds or clinical 
research center beds? 
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