Long-Term Follow-up Protocol for Participants Treated With Gene-Modified T Cells

Study Overview
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Brief Summary
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This is a prospective study for the long-term follow-up (LTFU) of safety and efficacy for all pediatric and adult participants exposed to Gene-modified (GM) T cell therapy participating in a previous Celgene sponsored or Celgene alliance partner sponsored study. Participants who received at least one GM T cell infusion will be asked to enroll in this LTFU protocol upon either premature discontinuation from, or completion of the prior parent treatment protocol.

Official Title
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Long-Term Follow-up Protocol for Subjects Treated With Gene-Modified T Cells

Conditions
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Neoplasms

Intervention / Treatment
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* Genetic: Gene-modified (GM) T cell therapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Received at least one gene-modified (GM) T-cell infusion in a previous Celgene sponsored or Celgene alliance partner-sponsored study, and have discontinued, or completed the post-treatment follow-up period in the parent treatment protocol, as applicable. Must understand and voluntarily sign an Informed Consent Form/Informed Assent Form prior to any study-related assessments/procedures being conducted. Exclusion Criteria: Not Applicable Other protocol-defined inclusion/exclusion criteria apply
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Participants exposed to Gene-modified (GM) T cell therapy<br> | Genetic: Gene-modified (GM) T cell therapy<br>* No investigational product will be administered<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of delayed Adverse Events (AEs) |  | Up to 15 years from last gene-modified (GM) T cell infusion | 
| Persistence of GM T cells |  | Up to 15 years from last GM T cell infusion | 
| Analysis of vector integration sites |  | Up to 15 years from last GM T cell infusion | 
| Incidence of replication-competent lentiviruses |  | Up to 15 years from last GM T cell infusion | 
| Physical growth as assessed by physical examination (pediatric participants only) |  | Up to 15 years from last GM T cells infusion or until Tanner Stage 5 is reached | 
| Incidence of sexual maturation as assessed by the Tanner staging system (pediatric participants only) |  | Up to 15 years from last GM T cells infusion or until Tanner Stage 5 | 
| Disease status of the primary malignancy |  | Up to 15 years from last GM T cell infusion | 
| Disease Progression: Number of participants who continue to be responders, who have progressed, and who have relapsed will be reported. |  | Up to 15 years from last GM T cells infusion | 
| Disease Relapse: Number of participants who continue to be responders, who have progressed, and who have relapsed will be reported. |  | Up to 15 years from last GM T cells infusion | 
| Overall Survival defined as the time from the first dose of investigational product or from the randomization date to the date of death or the date the participant is last known to be alive. |  | Up to 15 years from last GM T cells infusion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Lymphocyte count (B-cell) | If B-cell recovery criteria are not met at Month 60 (Year 5), then annual monitoring will continue until B-cell recovery is confirmed on 2 consecutive analyses or up to 15 years, until subject withdrawal of consent, or death, whichever occurs first. | Up to 15 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Long-term follow up, Gene-Modified T Cells, CAR T Cell