Clinical trial NCT05024175: Long-term Follow-up of Subjects Treated with CAR T Cells
Official title: Long-term Follow-up of Subjects Treated with CAR T Cells
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Long Term Adverse Effects
CAR-T
Duty to Follow Up
Adult
Progression-Free Survival
Disease-Free Survival
Overall Survival
Keywords: Long-Term Follow-up to CAR-T Cells
Follow-up Studies
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
First posted: 2021-08-27
Enrollment: 45
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects will be asked to participate leading up to the last DF/HCC corresponding main study visit

Brief summary: This is a single site, non-randomized, open-label, long-term safety and efficacy follow-up study for Phase 1 studies that evaluate the safety and efficacy of CAR T cells: NCT05660369 (DF/HCC# 22-175) and NCT06026319 (DF/HCC# 23-474).

Detailed description: This is a long-term safety and efficacy follow-up study for subjects who have been treated with CAR T cells in corresponding Phase I main studies that evaluated the safety and efficacy of CAR T cells in subjects.

No investigational treatment will be administered in this study.

The FDA (2020) recommends long-term follow up for subjects treated with gene therapy drug products to monitor for delayed adverse events (AEs), as well as durability of clinical response.

Therefore, after monitoring of subjects in the main studies has been completed (24 months after CAR T cells infusion, or \<24 months after CAR T cells infusion if subject terminated early due to disease progression or due to discontinuing corresponding main study for any reason), subjects will be asked to participate in a long-term follow-up study (LTFU).

Subjects will be followed for up to 15 years following their last CAR T cells infusion in the corresponding main study.

Eligibility criteria: Inclusion Criteria:

Subjects will be asked to participate leading up to the last DF/HCC corresponding main study visit.

Subjects meeting the following criteria are eligible for study participation:

* Provision of voluntary written informed consent by subject
* CAR T cells were administered in DF/HCC IRB corresponding main study

Exclusion Criteria:

Subjects meeting the following criterion are to be excluded from study participation:

- Subject unable to comply with study requirements

Primary outcomes:
measure: Overall Survival
timeFrame: 1 month
measure: Overall Survival
timeFrame: 6 months
measure: Overall Survival
timeFrame: 12 months
measure: Overall Survival
timeFrame: 24 months
measure: Progresison Free Survival
timeFrame: 1 month
measure: Progresison Free Survival
timeFrame: 6 months
measure: Progresison Free Survival
timeFrame: 12 months
measure: Progresison Free Survival
timeFrame: 24 months
measure: New incidence or exacerbation of a pre-existing neurologic disorder or prior rheumatologic or other autoimmune disorder
timeFrame: Up to 15 Years
measure: New incidence of a hematologic disorder
timeFrame: up to 15 years
measure: Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v 5.0
timeFrame: up to 15 years
measure: Vector-derived RCL
timeFrame: baseline and in 3 months, 6 months, and annually up to 15 years
measure: Assessment of CAR T cells persistence by VCN in peripheral blood
timeFrame: baseline and in 3 months, 6 months, every 6 months up to 5 years, and annually up to 15 years
measure: Progression-free Survival
timeFrame: From post-CAR T cells infusion date until date of first documented disease progression or date of death from any cause, assessed up to 15 years post treatment
measure: Overall Survival
timeFrame: From post-CAR T cells infusion date until documented date of death from any cause, assessed post treatment every 6 months through 5 years and then annually up to 15 years
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Matthew J Frigault, MD
role: PRINCIPAL_INVESTIGATOR
References: