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

NCT05024175 | Recruiting

• 1 other identifier: 20-721
• Study Type: observational
• Target: 45
• Locations: 1 country
• Sites: 1

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).

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

Outcome Measures

Primary Outcomes (15)

• 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

• 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

• 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

• 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

• 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

• 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

Study Arms (1)

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; Procedure: Tumor Biopsy; Diagnostic Test: Blood test

Interventions

Disease assessments DIAGNOSTIC_TEST

CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans as per protocol

CAR T cells

Tumor Biopsy PROCEDURE

Tumor Biopsy per protocol

CAR T cells

Blood test DIAGNOSTIC_TEST

Blood Test per protocol

CAR T cells

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• Subjects meeting the following criterion are to be excluded from study participation:
• \- Subject unable to comply with study requirements

Sponsors & Collaborators

• Marcela V. Maus, M.D.,Ph.D.: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

MeSH Terms

Conditions

Long Term Adverse Effects

Interventions

Hematologic Tests

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Study Officials

• Matthew J Frigault, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Matthew J Frigault, MD

CONTACT

617-643-6175; mfrigault@mgb.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor Investigator

Study Record Dates

• First Submitted: August 23, 2021
• First Posted: August 27, 2021
• Study Start: August 7, 2023
• Primary Completion (Estimated): August 1, 2038
• Study Completion (Estimated): August 1, 2039
• Last Updated: March 16, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data can be shared no earlier than 1 year following the date of publication
• Access Criteria: Contact the Partners Innovations team at http://www.partners.org/innovation

Locations

US (1)