Clinical Trials Logo

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.


Clinical Trial 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 ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05713214
Study type Observational
Source St. Jude Children's Research Hospital
Contact Aimee Talleur, MD
Phone 866-278-5833
Email referralinfo@stjude.org
Status Recruiting
Phase
Start date February 8, 2023
Completion date December 1, 2052

See also
  Status Clinical Trial Phase
Completed NCT03096821 - Extended Analysis for Leukemia/Lymphoma Treatment
Recruiting NCT05956457 - PRO and Wearable Data Insights From Individuals With R/R Multiple Myeloma N/A
Active, not recruiting NCT03560882 - A Pilot Trial of Atorvastatin in Tumor Protein 53 (p53) -Mutant and p53 Wild-Type Malignancies Phase 1
Recruiting NCT03821519 - Infusion of Donor Derived Cytokine Induced Killer (CIK) Cells in Hematological Patients Relapsed After Haploidentical Stem Cell Transplant Phase 1/Phase 2
Active, not recruiting NCT03413800 - Len/Dex/DLI in Relapsed Multiple Myeloma After Allogeneic Stem Cell Transplant Phase 2
Recruiting NCT05270096 - International Leukemia Target Board
Recruiting NCT03328078 - A Study of CA-4948 in Patients With Relapsed or Refractory Primary Central Nervous System Lymphoma Phase 1/Phase 2
Withdrawn NCT05205252 - A Study of Tazemetostat in Combination With Various Treatments in Participants With Blood Cancer. Phase 1/Phase 2
Recruiting NCT04327583 - Impact of the Coordinated Intervention of the Healthcare Facilities Pharmacist and the Dispensary Pharmacist on the Care Pathway for Cancer Patients Treated With Oral Therapy N/A