MEDICAL RECORD 
C&NSENTTO PA^t7cIPATE IN~A CLlWcAL RESEARCH STUDY 
* Adult Patient or • Parent, for Minor Patient 
INSTITUTE: NATIONAL HEART. LUNG AND BLOOD INSTITUTE 
STUDY NUMBER: PRINCIPAL INVESTIGATOR: Cvnthia Dunbar. M.D. 
STUDY TITLE: Informed Consent for Retroviral-Mediated Gene Transfer of Bone Marrow 
and Peripheral Blood Stem Cells During Autologous Bone Marrow Transplantation for Chronic 
Myelogenous Leukemia 
INTRODUCTION 
We invite you to take pan in a research study at the National Institutes of Health. It is 
important that you read and understand several general principles that apply to all that take pan 
in our studies: (a) taking pan in the study is entirely voluntary; (b) personal benefit will not 
result from taking pan in this study, but knowledge may be gained that will benefit others; (c) 
you may withdraw from the study at any time without penalty or loss of any benefits to which 
you are otherwise entitled. The nature of the study, the risks, inconveniences, discomforts, and 
other pertinent information about the study are discussed below. You are urged to discuss any 
questions you may have about this study with the staff members who explain it to you. 
NATURE OF STUDY 
Autologous bone marrow transplantation is a treatment with potential benefit for patients 
with chronic myelogenous leukemia (CML). Autologous bone marrow transplantation and its 
risks have been explained to you in a separate Informed Consent document and you have agreed 
to be enrolled in our transplantation protocol. You are now also being asked to take part in a 
research study of "retroviral-mediated gene transfer" of bone marrow cells and peripheral blood 
stem cells used during autologous bone marrow transplantation. The intent of this protocol is to 
treat you with high dose chemotherapy and to then give back the bone marrow and circulating 
blood cells that have been removed from you. Part of the bone marrow and circulating cells will 
have been “marked” with a special gene as described below. The goal of the study is to 
determine whether it is possible to put a new gene into bone marrow or circulating blood cells 
that will stay in your body for several months or more. It is not yet known whether it is possible 
to successfully place new genes into patients' bone marrow or circulating blood cells and to have 
the genes stay in these cells over several months to years. If this could be accomplished, it 
would represent an important potential advance in the treatment of many diseases. We may also 
learn important information that could improve the safety and usefulness of autologous 
transplantation in CML. 
You are being asked to take part in a research study that will try and accomplish the 
R 
placement of a bacterial gene called neo — into your bone marrow and circulating blood cells. An 
important part of the study involves testing bone marrow and circulating blood cells periodically 
to see whether the neo-^ gene is in these cells and how long the gene stays in the cells. The new 
technology to be used in this research study is called "retroviral-mediated gene transfer". It will 
be used to put a (neo^) gene into some of your bone marrow cells and peripheral blood 
PATIENT IDENTIFICATION 
CONSENT TO PARTICIPATE IN A CLINICAL 
RESEARCH STUDY 
• Adult Patient or 'Parent, for Minor Patient 
[194] 
Recombinant DNA Research, Volume 16 
