GENEFU: Long-Term Follow-Up of Patients who have Received Gene Therapy/Gene Marked Products

Description

Gene transfer is a relatively new area of science and medicine. Physicians want to make sure the use of gene transfer is safe when given as part of research or treatment for disease. Because gene transfer is a newly developing area of medicine, little is known about the effects that these treatments might have long after their use. 

Government agencies recommend that all recipients of gene transfer be monitored for 15 years after receiving a gene transfer product. This study will follow children who have received a gene transfer product at St. Jude Children’s Research Hospital within the past 15 years. The study will include physical evaluations and blood tests that will be performed at least once a year, for the 15 year period.  The information gathered in this research study will serve as case history data for the detection of late clinical effects associated with the use of gene transfer therapies.

Objectives

• To find out if infants, children, and young adults develop any side-effects months or years after receiving a gene transfer product as part of a research study or related treatment at St. Jude Children's Research Hospital.

Eligibility

• Only current St. Jude patients are eligible.
• Participant received an integrating retroviral or lentiviral vector based gene transfer or gene marked product at St. Jude Children’s Research Hospital within the past 15 year time period.

Principal Investigator

Stephen Gottschalk, MD