Recombinant DNA Advisory Committee - 6/9-10/94 
Dr. Smith pointed out one discrepancy regarding patient enrollment in the protocol by 
Dr. Galpin, et. al. (Protocol #9306-048). This protocol was submitted for RAC review 
on a voluntary basis since NIH funding was not involved. The protocol has accrued 16 
patients while the RAC approved a maximum of 15 subjects. No explanation has been 
provided for over-accrual. Drs. Parkman and Straus said that Viagene, Inc., should be 
notified since it is the sponsoring company and bears partial responsibility for data 
reporting. Ms. Buc said that if the protocol is not obligated to have RAC review under 
the NIH Guidelines', therefore, data reporting should be collected on a voluntary basis. 
Dr. Post concurred with Ms. Buc's statement. Dr. Zallen said that the investigators have 
benefitted from the RAC review and approval process; therefore, data reporting should 
be required. Dr. Chase said that future studies submitted on a voluntary submission 
should be approved with the stipulation that data reporting is mandatory. Ms. Wilson 
stated that the results obtained from this trial, i.e., immunological assays, may have 
bearing on future protocols submitted by the same sponsoring company. Dr. Noguchi 
agreed with the statement made by Ms. Wilson. Dr. Noguchi stated that the FDA 
encourages companies to voluntarily submit their protocols for RAC review, otherwise, 
public review by FDA committees would be required. 
Ms. Sheryl Osborne of Viagene, Inc., responded that the immunological data from Dr. 
Galpin's trial is contained in a coded format; therefore, the results are not yet available. 
Ms. Osborne stated that some of the subjects enrolled in this trial are on the placebo 
arm; perhaps, accounting for the discrepancy in patient number. 
Dr. Smith pointed out an adverse event report dated March 31, 1994, submitted by Dr. 
OldGeld for a subject entered on the HSV-TK/GCV glioblastoma study (Protocol 
#9206-019). A hemorrhagic complication occurred as a result of multiple intratumoral 
injections (44). The adverse event was not directly related to gene transfer. Dr. Walters 
said the RAC will follow up on this event. The other significant adverse effect was 
observed in Dr. Crystal's cystic fibrosis (CF) study (Protocol #9212-034). Dr. Wivel said 
that one subject developed pneumonitis related to the high dose of adenovirus vector. 
The investigators have reduced the vector dose. 
Summarized below are the categories of human gene transfer protocols that have been 
approved by the RAC to date: 
ilPii 
T 
Total CT+ M) 
RAC Approved 
50 
22 
72 
NDl Director Approved 
41 
22 
63 
Categories of RACrApproved Protocols 
f ' 
Cancer 
32 
4 
36 
Cystic Fibrosis 
7 
0 
7 
SOD/ADA 
1 
0 
1 
Recombinant DNA Research, Volume 19 
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