Six to eight patients per year will be treated on this pilot trial. A 
maximum of 16 patients will be treated. Consideration will be given 
to treating fewer than 16 patients if either no long-term gene 
marking of the marrow occurs in the first 6-8 patients or if the 
marking procedure is successful and the clinical mdrl retroviral 
vector is ready for study . The data obtained will be analyzed and 
descriptively reported. Because feasibility and trafficking patterns 
are the main end points of this study, no formal statistical analysis 
will be carried out. 
Because the probability of transducing a human stem cell in a 
patient recovering from chemotherapy is unknown and because the 
number of pluripotent stem cells contributing to graftment is 
unknown, it is not possible to assess the number of patients 
required to undertake this aim. 
13.0 Reporting of Data and Adverse Drug Reactions 
This study will be CTMS monitored. Data will be submitted to CTMS 
at least every two weeks. The NCI/DCT Case Report or ACES will be 
used to report to CTMS. 
Reporting of adverse drug reactions (ADR) will be made using the 
Division of Cancer Treatment Common Toxicity Criteria (Appendix I), 
and Toxicity Criteria for ABMT Studies Supplementary Toxicity 
Criteria (Appendix II) for reference according to the guidelines 
published by the DCT, NCI. Report by telephone to IDB within 24 
hours (301-496-7957, available 24 hours per day) the toxicities 
below. A written report should follow within ten working days to: 
Investigation Drug Branch, P.o. Box 30012, Bethesda, Maryland 
20824. Adverse Drug Reactions must also be reported to the 
Chairman, ICRS at the same time as IDB. Reporting guidelines are 
summarized as follows: 
All life-threatening events (Grade 4, except for Grade 4 
myelosuppression) which may be due to administration of 
investigations drug(s). 
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