unknown death rate is higher than the figures above, the design provides even higher 
chances of stopping the trial early for reevaluation. The probability for terminating 
accrual early is 0.59, 0.89 and 0.98 if the true death rate is 0.20, 0.30 or 0.40, 
respectively. 
Toxicity 
If six episodes of unacceptable (Grade IV) toxicity or death during BMT are observed 
in the first 20 patients treated, then accrual to the study will be terminated and all data 
evaluated to determine whether continuation is warranted. This decision rule provides 
for terminating the study early with probabilities of 0.01, 0.07 and 0.20 if the true 
unknown toxic rate is 0.10, 0.15 or 0.20, respectively. If the true unknown toxic rate 
is unacceptably large we will have an even greater chance of stopping the trial early for 
reevaluation. The probability for terminating accrual is 0.38, 0.58 and 0.87 if the true 
toxic rate is 0.20, 0.30 or 0.40, respectively. 
11.0 RECORDS TO BE KEPT 
Flow sheets documenting dates and doses of therapy as well as clinical chemistries, 
hematologic parameters, the clinical status and occurrence of any adverse events and 
subsequent interventions are to be kept on all patients. 
Imaging reports 
Surgical summaries 
Autopsy summaries, where appropriate 
Date of entry on to other appended protocols 
Informed consent documents 
All required clinical evaluation records will be the responsibility of Drs Helen Heslop 
or Robert Krance who will also be responsible for analysis of the clinical outcome and 
toxicity. 
The laboratory evaluation of retroviral insertion will be the responsibility of Drs. Brenner 
and Heslop. 
12.0. REPORTING REQUIREMENTS 
12.1 Register all patients with Bone Marrow Transplant Data Manager at Ext . 
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