local regulations permit, the study monitor should have 
access to original laboratory test reports and other 
patient documents needed to verify data entered on the 
CRFs . The investigator should cooperate with the 
assigned study monitor to ensure that all problems are 
resolved. 
12.0 BIOSTATISTICAL CONSIDERATIONS 
Summaries for the following parameters will be compiled at 
six months and one year: 
-percentage of cells containing the FACC vector 
-sensitivity of cells to MMC; progenitor growth in MMC 
-blood counts, bone marrow examination 
-safety, adverse effects 
13.0 ETHICAL CONSIDERATIONS 
The investigator will ensure that the study is conducted in 
full conformance with DHHS and FDA standards for human 
research. 
13.1 Informed Consent 
All study participants must sign an informed consent 
(see Appendix VII) . The investigators will inform all 
subjects as to the nature, aims, duration, potential 
hazards, and procedures to be performed during the 
study and that his or her medical records may be 
reviewed by the sponsors or the FDA. This protocol 
must receive approval by the Institutional Review Board 
of all participating sites prior to implementation. 
The investigator must also explain that the patients 
are completely free to refuse to enter the study or to 
withdraw from it at any time. The protocol will be 
discussed in detail with all potentially eligible 
patients. All revisions of the protocol must be 
reflected in the consent form and reviewed by the IRB. 
13.2 Alternative Therapies 
Marrow transplantation from allogeneic donors (matched 
histocompatible siblings) and from alternative donors 
(matched unrelated normal volunteers or partially 
mismatched family members) have been successfully 
employed in some FA patients. All potential 
participants will be fully informed of the availability 
and suitability of these procedures, as well as their 
associated success rates and hazards. Participation in 
this protocol should not affect marrow transplantation 
as a future therapeutic option. 
13.3 Assent process 
For minors, the consent document will be provided to 
parents or guardians. The consent form will be the 
basis for the description of the procedures and 
research therapies to the child and parents. Because 
of the wide age range of potential participants, 
discussion will be tailored to the age and 
sophistication of the child or adolescent. The assent 
process will take place with several separated visits 
to the patient, discussions with the patient alone and 
with the patient in company of his parents, and 
sufficient time will be allowed for full consideration 
of the protocol by the patient and family. 
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Recombinant DNA Research, Volume 19 
