Long Term Safety Following GM T Cell Therapy
Conditions
Keywords
GM T cell therapy, Replication Competent Lentivirus (RCL)
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.
Interventions
GENETICNon-interventional
No study drug administered. Long-term follow-up only.
Sponsors
Regeneron Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Health-Related Quality of Life (HRQoL) Changes | Up to 5 years | Assessed using instruments administered in the parent treatment protocol |
Countries
United States
Contacts
CONTACTClinical Trials Administrator
STUDY_DIRECTORClinical Trial Management
Regeneron Pharmaceuticals
Outcome results
None listed