Recombinant DNA Advisory Committee - 3/34/94 
The RAC-approved version of the Cover Sheet for Expedited Review of a Single Patient 
Human Gene Transfer Experiment (Expedited Review) cover sheet reads: 
A. 
COVER SHEET FOR EXPEDITED REVIEW 
OF A SINGLE PATIENT HUMAN GENE TRANSFER EXPERIMENT 
(Expedited Review) 
BACKGROUND 
1. 
Provide the following information about the proposed study, title, principal investigators, and participating 
institutions. 
a 
VECTOR, TARGET CRIJ^ AND TRANSDUCTION PROCEDURES 
1. 
What arc the proposed gene and vector for this protocol? 
2. 
Has the proposed vector previously been approved by the RAC? If so, provide the title and principal 
investigator(s) of at least one RAC-approved protocol utilizing this vector. If not, attach the complete vector 
sequence (hard copy and a 3Vi inch diskette in ASQI format). 
3. 
Who is the vector supplier? 
4. 
What is the target cell to be transduced by the proposed vector? 
5. 
What is the rate of transduction of the proposed vector and target cells in the proposed setting (i.e., your 
laboratory)? 
6. 
What is the level of gene expression demonstrated in the target cell? 
7. 
How was gene expression determined? 
8. 
What assay was used to detect replication-competent virus (RCR)? 
9. 
Was RCR detected? 
10. 
What is the level of sensitivity of the RCR assay? (Attach documentation). 
11. 
Has the vector been reviewed and accepted for clinical investigation by the Food and Drug Administration? 
What was the date of investigational new drug (IND) submission? Is there a sufficient supply of the clinical 
grade material available to complete the proposed study? If clinical grade material is unavailable, on what 
date will such material become available? 
c 
CLINICAL PROTOCOL 
1. 
What in vitro or in vivo svstemfs^ were used to determine nreclinical efficacv? 
2. 
What is the end point of the protocol? 
3. 
Is the orotxjsed study identical to another RAC-aoDroved protocol except that the study will be oerformed at 
a satellite institution? If so, provide a letter of cross-reference from the sponsoring institution. 
4. 
Is the oroixised study similar to another RAC-aoDroved nrotocol? Identify the maior differences. 
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Recombinant DNA Research, Volume 19 
