Long-term Follow-up After Adoptive Transfer of Genetically Modified Cell Products

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.

Study Overview

• Status: Recruiting
• Conditions: Refractory Hematologic Malignancy; Relapsed Hematologic Malignancy

Detailed 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 Type: Observational
• Enrollment (Estimated): 1000

Contacts and Locations

• Study Contact: Name: Aimee Talleur, MD; Phone Number: 866-278-5833; Email: referralinfo@stjude.org
• Study Contact Backup: Name: Swati Naik, MBBS; Email: swati.naik@stjude.org

Study Locations

• United States
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Recruiting
      • St. Jude Children's Research Hospital
      • Contact: Aimee Talleur, MD; Phone Number: 866-278-5833; Email: referralinfo@stjude.org
      • Principal Investigator: Aimee Talleur, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All participants who have received genetically modified cell therapy products on St. Jude investigator-initiated clinical studies.

Description

Inclusion Criteria:

• Receipt of a genetically modified cell product on a St. Jude investigator-initiated study within the prior 15 years.

Exclusion Criteria:

• Inability or unwillingness of research participant and/or legal guardian/ representative to give written informed consent.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Obtain histories for detection of significant delayed medical events after receipt of a gene therapy product on a St. Jude investigator-initiated clinical trial.	Obtain histories for detection of significant delayed medical events after receipt of a gene therapy product on a St. Jude investigator-initiated clinical trial.	30 years

Collaborators and Investigators

• Sponsor: St. Jude Children's Research Hospital
• Investigators: Principal Investigator: Aimee Talleur, MD, St. Jude Children's Research Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 8, 2023
• Primary Completion (Estimated): December 1, 2052
• Study Completion (Estimated): December 1, 2052

Study Registration Dates

• First Submitted: January 17, 2023
• First Submitted That Met QC Criteria: January 31, 2023
• First Posted (Actual): February 6, 2023

Study Record Updates

• Last Update Posted (Actual): May 26, 2026
• Last Update Submitted That Met QC Criteria: May 20, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Hematologic Neoplasms
• Other Study ID Numbers: LONGENE

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No