This protocol (GENEFU) provides a mechanism for the 15-year followup period that the FDA
requires for all participants in gene transfer protocols and assures that adequate followup
can be maintained for a wide variety of participants on different individual gene therapy
protocols at St. Jude Children's Research Hospital.
GENEFU serves as an umbrella protocol for long-term follow-up (LTFU) for recipients of gene
therapy/gene marked (GT/GM) products at St. Jude Children's Research Hospital. The FDA has
recommended methods to assess the risk of delayed adverse events after GT/GM and has provided
specific requirements regarding the duration and design of LTFU observations. This protocol
is intended to provide LTFU in accordance with the FDA guidelines for those who received a
GT/GM product as part of a St. Jude-sponsored clinical trial or compassionate use treatment
plan. The protocol calls for a physical examination or general health evaluation and
collection of required blood samples annually for up to 15 years after the last receipt of a
Goals will be to obtain clinical histories in order to detect late clinical outcomes
suggestive of retroviral or lentiviral disease, including but not limited to cancer/second
malignancies, neurologic disorders, autoimmune disorders, and hematologic disorders. Blood
samples will be archived and tested when clinically or scientifically indicated, as in the
event of development of a second malignancy. This prospective cohort study will utilize
descriptive statistics in the analysis of long-term late effects outcomes. It offers a
uniform approach to long-term safety monitoring in research participants who have received a
gene-transduced product as part of St. Jude-sponsored GT or GM protocols and compassionate
use treatment plans.
Secondary objectives include the following: to obtain and archive blood samples to 1) detect
frequency of vector marking in peripheral blood mononuclear cells when clinically or
scientifically indicated, such as in the event of participant development of second
malignancy, autoimmune disease, hematologic abnormality, or neurologic event, and; 2) To
obtain and archive blood samples to detect and characterize clonal proliferation events
occurring in gene transfer trials when clinically or scientifically indicated.
- Has received an integrating retroviral or lentiviral vector based GT/GM product at St.
Jude Children's Research Hospital within the past 15 year time period. Patient is no
later than 15 years post receipt of GT/GM product.