Date trial registered
Apr 22, 2018
Incorporation of the first participant
Apr 2, 2019
Recruitment status
Not Recruiting
Academic title
(Data source: WHO)
Long-Term Follow-up Protocol for Subjects Treated with Gene-Modified T cells.
Type of trial
(Data source: WHO)
Interventional clinical trial of medicinal product
Design of the trial
(Data source: WHO)
Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: Long-Term Follow-up Protocol If controlled, specify comparator, Other Medicinial Product: Placebo: Other: Number of treatment arms in the trial: 0
Phase
(Data source: WHO)
Human pharmacology (Phase I): noTherapeutic exploratory (Phase II): yesTherapeutic confirmatory - (Phase III): yesTherapeutic use (Phase IV): no
Primary end point
(Data source: WHO)
Main Objective: - To assess the risk of delayed adverse events (AEs) following exposure to gene-modified (GM) T cells
- To monitor for long-term persistence of GM T cells, including analysis of vector integration sites, as appropriate.
- To monitor for generation of replication competent retroviruses (RCR)
- To assess long-term efficacy following treatment with GM T cells
- Describe growth, developmental outcome, and sexual maturity status for subjects who were aged < 18 years at time of GM T cell treatment
- To assess long term health-related quality of life following treatment with GM T cells;Secondary Objective: None;Primary end point(s): Safety:
- Incidence of delayed Adverse Events suspected to be related to prior gene-modified (GM) T cell therapy, including:
- New malignancies (hematologic or solid)
- New neurologic disorder, or exacerbation of a pre-existing neurologic disorder
- New rheumatologic or autoimmune disorder, or exacerbation of a prior rheumatologic or other autoimmune disorder
- New hematologic disorder
- Other new clinical conditions considered related to the prior GM T cell therapy by the investigator.
Hospitalizations, regardless of relationship to prior treatment, including reasons and dates
- Persistence of GM T cells
- Analysis of vector integration sites
- Incidence of replication-competent retroviruses (RCR)
- Height, weight, growth, and organ development will be assessed for all pediatric subjects
- Sexual maturity status for pediatric subjects
Efficacy
Where applicable:
- Tumor Response Status
- Date of Disease Progression
- Date of Relapse
- Survival Status
HRQoL
Measurement of health-related quality of life (HRQoL) changes as assessed using instruments administered in the parent treatment protocol;Timepoint(s) of evaluation of this end point: HRQoL: Up to 5 years from last GM T cells infusion
Other endpoints: Up to 15 years from last GM T cells infusion
Secundary end point
(Data source: WHO)
Secondary end point(s): Not Applicable.;Timepoint(s) of evaluation of this end point: Not Applicable.
Contact information
(Data source: WHO)
Celgene Corporation
