Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT05713214 |
| Other study ID # |
LONGENE |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
February 8, 2023 |
| Est. completion date |
December 1, 2052 |
Study information
| Verified date |
May 2024 |
| Source |
St. Jude Children's Research Hospital |
| Contact |
Aimee Talleur, MD |
| Phone |
866-278-5833 |
| Email |
referralinfo[@]stjude.org |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
Human gene therapy products are designed to achieve therapeutic effect through genetic
modifications of human cells using retroviral or lentiviral vectors, resulting in permanent
or long-acting changes in the human body. With this genetic modification comes risk of
undesirable adverse events. Due to this risk, the Food and Drug Administration (FDA) and the
Center for Biologics Evaluation and research (CBER) require long-term follow-up (15 years) of
participants that receive investigational gene therapy products that meet defined criteria.
This protocol will provide a mechanism by which to appropriately monitor participants that
have received a genetically modified cellular product on a St. Jude initiated study.
Description:
This is a prospective, longitudinal, non-therapeutic study which includes routine assessment
for long-term effects, as per FDA guidelines, after receipt of a gene therapy product on a
St. Jude investigator-initiated clinical trial. There will be no group assignment, no use of
placebo groups and no use of control subjects. Participants will enroll on this long-term
follow-up study after completion of necessary follow-ups on the interventional clinical trial
on which they received treatment with a genetically modified product, to complete a total of
15 years of follow-up post infusion.
Assessments will include routine history, physical exam and blood sample procurement, in
accordance with FDA guidance, for 15 years post infusion of the gene therapy product. Study
evaluations may be performed by a local provider in conjunction with the St. Jude research
team. In addition, standard clinical data related to the subject's prior gene therapy
treatment will be reviewed. The data may include, but is not limited to: patient
demographics, disease history, on-going disease status updates, gene therapy product and
treatment characteristics, patient clinical status, post gene therapy disease directed
therapies, relapse and death
