I 
PROTOCOL SYNOPSIS 
TITLE: 
A Phase I/II Pilot Study of the Safety of the Adoptive Transfer of Syngeneic 
Gene-Modified Cytotoxic T-Lymphocytes in HIV-Infected Identical Twins 
SPONSOR: 
National Institute of Allergy and Infectious Diseases 
CO-SPONSOR: 
Cell Genesys Inc. 
PHASE: 
I/II 
INDICATION: 
HIV infection 
STUDY DESIGN: 
Open-label, comparative, sequentially randomized treatment with genetically 
unmodified or modified ex v/vo-expanded T-lymphocytes in patients with HIV 
infection who possess a seronegative syngeneic twin. Genetic modification 
consists of introduction of a gene for HLA-unrestricted "universal" receptors 
specific for the gpl20 HIV envelope protein. Treatment is divided into 
Periods I and II as outlined below under "Procedures". 
PRIMARY 
ENDPOINT: 
Safety of administering syngeneic genetically modified CD8+ cytotoxic ' 
T-Iymphocytes. 1 1 
SECONDARY 
ENDPOINTS: 
Effects on primary disease status (CD4 count, HIV burden, p24 antigen), ' ) 
longevity of genetically modified transplanted T-lymphocytes. ii 
l|; 
SAMPLE SIZE: 
Treatment Treatment * ! 
Cohort Period I Period II | 
0 6 -> 6 -1- 4 new 
1 6 -> 
2 6 -> } 18-1-12 new at MTD j 
3 6 -> i 1 
A maximum of 40 evaluable patients will be treated on this protocol: 10 given I 
unmodified T-lymphocytes and 30 given modified T-lymphocytes. l| 
> .1 
MAJOR 
ELIGIBILITY 
CRITERIA 
Patients >18 years of age with HIV infection and a seronegative identical . I 
twin, Karnofsky >60%, life expectancy >6 months, adequate organ I 
function,signed informed consent. 1 
TEST ARTICLE: 
Genetically modified, ex v/vo-expanded syngeneic T lymphocytes. 1 
1 
COMPARISON: 
Ex v/v6>-expanded, unmodified syngeneic T-lymphocytes. 1 
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Recombinant DNA Research, Volume 19 :| 
