A Safety and Efficacy Long-Term Follow-up Study of Adult Participants Treated With Gene Modified T Cells

NCT06798298 | Recruiting FDA Drug

• 1 other identifier: LTF-01
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective study for the Long-Term Follow-Up (LTFU) of safety and efficacy of all participants exposed to gene modified (GM) T cell therapy in accordance with Health Authorities' guidance for participants treated with gene therapy products. Participants who received at least one infusion of gene modified T cells in a 2seventy bio (prior to April 2024) or Regeneron Pharmaceuticals sponsored study will be asked to participate in this LTFU protocol, upon either premature discontinuation from, or completion of the parent treatment protocol. Participants enrolled in this LTFU protocol will have safety assessments, laboratory evaluations, and complete patient-reported outcome (PRO) questionnaires at scheduled intervals as applicable.

Conditions

Long Term Safety Following GM T Cell Therapy

Keywords

GM T cell therapy; Replication Competent Lentivirus (RCL)

Outcome Measures

Primary Outcomes (7)

• Incidence of delayed adverse events considered at least possibly related to prior gene modified T cell therapy

  Up to 15 years

• Persistence of gene modified T cells

  Up to 15 years

• Analysis of vector integration sites

  Up to 15 years

• Incidence of RCL

  Up to 15 years

• Disease progression status

  To assess long-term efficacy following treatment with gene modified T cells

  Up to 15 years

• Date of disease progression

  Up to 15 years

• Survival status

  Up to 15 years

Secondary Outcomes (1)

• Health-Related Quality of Life (HRQoL) Changes

  Up to 5 years

Study Arms (1)

GM T cell population

Participants exposed to gene modified T cell therapy on a 2seventy bio (prior to April 2024) or Regeneron Pharmaceuticals sponsored study

Genetic: Non-interventional

Interventions

Non-interventional GENETIC

No study drug administered. Long-term follow-up only.

GM T cell population

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Enrolled participants will be determined by the number of participants participating in GM T cell 2seventy bio (prior to April 2024) and Regeneron Pharmaceuticals sponsored studies consenting to participate in the LTFU study.

You may qualify if:

• All participants who received at least one GM T cell infusion in a previous 2seventy bio (prior to April 2024) or Regeneron Pharmaceuticals sponsored study, and have completed the post-treatment follow-up period on the parent treatment protocol, or discontinued follow-up on the parent protocol after completing at least 6 months of safety monitoring, as applicable.
• Participant (and legal representative, when applicable) must understand and voluntarily sign an Informed Consent Form (ICF)/Informed Assent Form (IAF) prior to any study related assessments/procedures being conducted.

You may not qualify if:

• Not applicable.

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (1)

Tennessee Oncology

Nashville, Tennessee, 37203, United States

RECRUITING

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Clinical Trials Administrator

CONTACT

844-734-6643; clinicaltrials@regeneron.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 9, 2025
• First Posted: January 29, 2025
• Study Start: November 10, 2025
• Primary Completion (Estimated): June 17, 2040
• Study Completion (Estimated): June 17, 2040
• Last Updated: November 24, 2025

Record last verified: 2025-11

Locations

US (1)