13.4 Patient Confidentiality 
All reports and patient samples will be identified only 
by a coded number to maintain patient confidentiality. 
All records will be kept confidential to the extent 
permitted by law. The investigator should keep a 
separate log of patients' codes, names, and addresses. 
Documents which identify the patient by name (informed 
consent) should be kept in strict confidence. 
14.0 CRITERIA AND PROCEDURES FOR PROTOCOL MODIFICATION AND STUDY 
TERMINATION 
Modifications which may affect the safety of the study 
patient, or which may alter the scope of the investigation, 
the scientific quality of the study, the study design, 
dosages, duration of therapy, patient assessments (added 
evaluation that poses potential risk or inconvenience to the 
patient) , number of patients, and patient eligibility 
criteria, may be made only after appropriate consultation 
between the sponsor and the investigator. 
If the consensus is to revise the current protocol, a 
formal List of Changes will accompany the amended protocol 
and these will be submitted to the FDA, the site's IRB, and 
other committees as required. 
Both the sponsor and the investigator reserve the right 
to terminate the study at any time. If this becomes 
necessary, appropriate procedures for continuing the long- 
term follow-up requested by the regulatory agencies will be 
arranged after review and approval by both parties. 
15.0 PUBLICATION OF DATA /PROTECT I ON OF COMPANY SECRETS 
The investigator will agree to inform the sponsor of all 
study results. Any manuscript, abstract, or presentation 
will be made available to the sponsor and all participating 
study investigators for review prior to submission of 
manuscripts, abstracts, or scientific meetings. This will 
allow the sponsor to protect proprietary information and to 
provide comment based on information that may not yet be 
available to the investigator. 
16.0 BIOHAZARD WARNINGS AND PRECAUTIONS 
Patients will be treated in standard hospital and clinic 
rooms. No special patient precautions will be taken. 
17.0 REFERENCES 
1. Alter, B.P., Young, N.S. in Nathan, D.G., Oski, F.A. , eds . : 
Hematology of Infancy and Childhood . 4th edition, p.216, 
1993 . 
2. Strathdee, C.A., Duncan, A.M.V. , Buchwald, M. Nature 
Genetics 1:196, 1992. 
3. Strathdee, C.A., Gavish, H. , Shannon, W.R., Buchwald, M. 
Nature 356:763, 1992. 
4. Whitney, M.A. , Saito, H. , Jakobs, P.M., Gibson, R.A. , Moses, 
R.E., Grompe, M. Nature Genetics 4:202, 1993. 
5. Schroeder-Kurth, T.M. , Auerbach, A.D., Obe, G. eds . : Fanconi 
anemia: clinical, cytogenetic and experimental aspects . 
1989 . 
6. Williams, D.A. Human Gene Therapy 1:229, 1990. 
7. Karlsson, S. Blood 78:2481, 1991. 
8. Nienhuis, A.W. , Walsh, C.E., Liu, J.M. in Young, N.S. , ed. : 
Viruses and bone marrow , p. 353, 1993. 
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