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

Not yet recruiting

• Conditions: Long Term Safety Following GM T Cell Therapy

• Registration Number: NCT06798298
• Lead Sponsor: Regeneron Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-01-09
• Study First Submitted QC: 2025-01-28
• Study First Posted: 2025-01-29
• Status Verified: 2025-04
• Last Update Submitted: 2025-05-09
• Last Update Posted: 2025-05-11
• Study Start: 2025-05-30
• Primary Completion: 2039-12-31
• Study Completion: 2039-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

1. 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.
2. 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.

Exclusion Criteria

Not applicable.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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	Up to 15 years	To assess long-term efficacy following treatment with gene modified T cells
Date of disease progression	Up to 15 years
Survival status	Up to 15 years

Secondary Outcome Measures

Name	Time	Method
Health-Related Quality of Life (HRQoL) Changes	Up to 5 years	Assessed using instruments administered in the parent treatment protocol