Long-term Follow-up of Subjects Treated With CAR T Cells

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).

Study Overview

• Status: Recruiting
• Conditions: Adult; CAR-T; Overall Survival; Long Term Adverse Effects; Progression-Free Survival; Disease-Free Survival; Duty to Follow Up
• Intervention / Treatment: Diagnostic test: Disease assessments; Procedure: Tumor Biopsy; Diagnostic test: Blood test

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.

• Study Type: Observational
• Enrollment (Estimated): 45

Contacts and Locations

• Study Contact: Name: Matthew J Frigault, MD; Phone Number: 617-643-6175; Email: mfrigault@mgb.org

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Massachusetts General Hospital
      • Principal Investigator: Matthew J Frigault, MD
      • Contact: Matthew J Frigault, MD; Phone Number: (617) 724-4000; Email: mfrigault@mgb.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

CAR T cells; Eligibility to participate on this study if enrolled on a DF/HCC IRB corresponding main study and received infusion of CAR T cells; The research study procedures include evaluations and follow up visits: Timepoints of each evaluation and follow up visit- per protocol; Medical History/Physical Exam; Blood Test; Assessment of Disease: CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans.; Tumor biopsy.; Data Collection; Biobanking

Diagnostic test: Disease assessments; CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans as per protocol; Procedure: Tumor Biopsy; Tumor Biopsy per protocol; Diagnostic test: Blood test; Blood Test per protocol

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive	1 month
Overall Survival	defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive	6 months
Overall Survival	defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive	12 months
Overall Survival	defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive	24 months
New incidence or exacerbation of a pre-existing neurologic disorder or prior rheumatologic or other autoimmune disorder	An annual physical exam will be conducted for surveillance of manifestations indicative of oncoretroviral diseases.	Up to 15 Years
New incidence of a hematologic disorder	An annual physical exam will be conducted for surveillance of manifestations indicative of oncoretroviral diseases.	up to 15 years
Vector-derived RCL	Archiving of samples for potential detection of vector-derived RCL. RCL testing will be monitored at the central RCL lab (Indiana University) by a suitable qPCR assay for detection of the lentiviral vector (VSV-g DNA).	baseline and in 3 months, 6 months, and annually up to 15 years
Assessment of CAR T cells persistence by VCN in peripheral blood	VCN will be performed in DNA from whole blood to monitor for persistence of vector sequence	baseline and in 3 months, 6 months, every 6 months up to 5 years, and annually up to 15 years
Progresison Free Survival	PFS is defined as the number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.	1 month
Progresison Free Survival	PFS is defined as the number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.	6 months
Progresison Free Survival	PFS is defined as the number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.	12 months
Progresison Free Survival	PFS is defined as the number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.	24 months
Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v 5.0	NCI Common Terminology Criteria for Adverse Events (CTCAE)	up to 15 years
Progression-free Survival	The number of days from the day of CAR T cells treatment to the first documented disease progression or date of death, whichever occurs first. Patients who are lost to follow up without a known date of progression or death due to any cause will be censored in the analysis at the date of their last available tumor assessment.	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
Overall Survival	The time from post-CAR T cells infusion to the date of death.	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

Collaborators and Investigators

• Sponsor: Marcela V. Maus, M.D.,Ph.D.
• Investigators: Principal Investigator: Matthew J Frigault, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): August 7, 2023
• Primary Completion (Estimated): August 1, 2038
• Study Completion (Estimated): August 1, 2039

Study Registration Dates

• First Submitted: August 23, 2021
• First Submitted That Met QC Criteria: August 23, 2021
• First Posted (Actual): August 27, 2021

Study Record Updates

• Last Update Posted (Actual): March 16, 2026
• Last Update Submitted That Met QC Criteria: March 13, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Follow-up Studies; Long-Term Follow-up to CAR-T Cells
• Additional Relevant MeSH Terms: Pathologic Processes; Pathological Conditions, Signs and Symptoms; Long Term Adverse Effects; Investigative Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Hematologic Tests
• Other Study ID Numbers: 20-721

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to Sponsor Investigator or designee. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
• IPD Sharing Time Frame: Data can be shared no earlier than 1 year following the date of publication
• IPD Sharing Access Criteria: Contact the Partners Innovations team at http://www.partners.org/innovation
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No