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

NCT ID: NCT05713214

Last Updated: 2025-07-01

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 1000 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-02-08
• Study Completion Date: 2052-12-01

Brief 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.

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

Conditions

Relapsed Hematologic Malignancy; Refractory Hematologic Malignancy

Study Design

• Observational Model Type: OTHER
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

St. Jude Children's Research Hospital

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Aimee Talleur, MD

Role: PRINCIPAL_INVESTIGATOR

St. Jude Children's Research Hospital

Locations

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Aimee Talleur, MD

Role: CONTACT

referralinfo@stjude.org

866-278-5833

Swati Naik, MBBS

Role: CONTACT

swati.naik@stjude.org

Facility Contacts

Aimee Talleur, MD

Role: primary

referralinfo@stjude.org

866-278-5833

Other Identifiers

LONGENE

Identifier Type: -

Identifier Source: org_study_id