Brief Summary:
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.
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
Primary outcome:
1. Incidence of delayed Adverse Events (AEs) (Time Frame - Up to 15 years from last gene-modified (GM) T cell infusion)
2. Persistence of GM T cells (Time Frame - Up to 15 years from last GM T cell infusion)
3. Analysis of vector integration sites (Time Frame - Up to 15 years from last GM T cell infusion)
4. Incidence of replication-competent lentiviruses (Time Frame - Up to 15 years from last GM T cell infusion)
5. Physical growth as assessed by physical examination (pediatric participants only) (Time Frame - Up to 15 years from last GM T cells infusion or until Tanner Stage 5 is reached)
6. Incidence of sexual maturation as assessed by the Tanner staging system (pediatric participants only) (Time Frame - Up to 15 years from last GM T cells infusion or until Tanner Stage 5)
7. Disease status of the primary malignancy (Time Frame - Up to 15 years from last GM T cell infusion)
8. Disease Progression: Number of participants who continue to be responders, who have progressed, and who have relapsed will be reported. (Time Frame - Up to 15 years from last GM T cells infusion)
9. Disease Relapse: Number of participants who continue to be responders, who have progressed, and who have relapsed will be reported. (Time Frame - Up to 15 years from last GM T cells infusion)
10. 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. (Time Frame - Up to 15 years from last GM T cells infusion)
Secondary outcome:
1. Lymphocyte count (B-cell) (Time Frame - Up to 15 years):
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.
- Gene-modified (GM) T cell therapy:
No investigational product will be administered
Quelle: ClinicalTrials.gov
