Title
Long-Term Follow-Up Protocol for Subjects Treated with Gene-Modified T Cells

Description
Subjects participating in this study will be followed from time of roll-over from the parent GM T protocol until 15 years after the last GMT infusion, withdrawal of consent, lost to follow-up, or death, whichever occurs first. Both local and central laboratory evaluations and safety assessments will be conducted during this trial. In addition, pediatric subjects will be monitored for growth, development and sexual maturity.
Stage 5 per Tanner Staging Criteria must be reached prior to study discontinuation.

Objective
Primary Outcome Measures:

Adverse Events (AEs) [ Time Frame: Up to 15 years from last GM T cell infusion ]


Incidence of delayed Adverse Events suspected to be related to prior gene-modified (GM) T cell therapy



Tumor Response Status [ Time Frame: At month 12 from last GM T cells infusion then yearly until date of disease relapse or progression, assessed up to year 15 ]


Number of subjects who continue to be responders, who have progressed, and who have relapsed will be reported. When reporting progression/relapse the appropriate date will also be reported.



Disease Progression [ Time Frame: Up to 15 years from last GM T cells infusion ]


Number of subjects who continue to be responders, who have progressed, and who have relapsed will be reported. When reporting progression/relapse the appropriate date will also be reported.



Disease Relapse [ Time Frame: Up to 15 years from last GM T cells infusion ]


Number of subjects who continue to be responders, who have progressed, and who have relapsed will be reported. When reporting progression/relapse the appropriate date will also be reported.



Overall Survival [ Time Frame: Up to 15 years from last GM T cells infusion ]


Overall survival is 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 subject is last known to be alive.



Health-related quality of life (HRQoL) [ Time Frame: Up to approximately 5 years ]


Health-related quality of life (HRQoL) analyses will be performed by combining data collected in this protocol with the data from each respective parent study as appropriate per parent study specified analysis plan, and per LTFU study plan after the parent study is closed out.



Height of pediatric subjects treated with GM T cells [ Time Frame: At month 12 from last GM T cells infusion then yearly until subject reaches Stage 5 per Tanner staging criteria or for 15 years from last GM T cell infusion, whichever occurs later ]


Height (inches or centimeters) will be collected for all pediatric subjects and descriptively summarized



Weight of pediatric subjects treated with GM T cells [ Time Frame: At month 12 from last GM T cells infusion then yearly until subject reaches Stage 5 per Tanner staging criteria or for 15 years from last GM T cell infusion, whichever occurs later ]


Weight (pounds or kilograms) will be collected for all pediatric subjects and descriptively summarized



Sexual maturation of pediatric subjects treated with GM T cells [ Time Frame: At month 12 from last GM T cells infusion then yearly until subject reaches Stage 5 per Tanner staging criteria or for 15 years from last GM T cell infusion, whichever occurs later ]


Sexual maturation, assessed by Tanner staging system, will be conducted for all pediatric subjects and descriptively summarized

Treatment Subjects exposed to Gene-modified (GM) T cell therapy
Subjects will be followed for 15 years from the last GM T cells infusion until withdrawal of consent, lost to follow-up, or death, whichever occurs first. Annual safety assessments will be conducted every 6 months during the first 5 years from the date of last GM T cell infusion. Laboratory evaluations and safety assessments will be conducted during this period. After 5 years, subjects will continue to be followed yearly. During the trial, pediatric subjects will be monitored for growth, development and sexual maturity. Stage 5 per Tanner Staging Criteria must be reached prior to study discontinuation.

Key Eligibility Inclusion Criteria:

Subjects who meet the following criteria will be eligible to participate in the Long-Term Follow-Up study:

All adult and pediatric subjects who received at least one GM T cells 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.

Subject (and, parental/legal representative, when applicable) must understand and voluntarily sign an Informed Consent Form/Informed Assent Form prior to any study-related assessments/procedures being conducted.

Subject is willing and able to adhere to the study visit schedule and other protocol requirements.

Applicable Disease Sites
Multiple Myeloma

Participating Institutions
UW Health University Hospital