Long Term Adverse EffectsNCT05024175
Long-term Follow-up of Subjects Treated With CAR T Cells
An observational study of Long Term Adverse Effects and CAR-T, run by Marcela V. Maus, M.D.,Ph.D. at 1 site in United States.
Trial 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 (HCC# 22-175), NCT06026319 (HCC# 23-474) and NCT NCT07388277 (MGB# 2025P003179. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Long Term Adverse Effects, CAR-T, Duty to Follow Up
- What is being tested?
- Disease assessments, Tumor Biopsy and Blood test
- Type of treatment
- Injection or infusion, Surgery or procedure
- How many people
- 45 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Aug 2023to 1 Aug 2039
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Subjects will be asked to participate leading up to the last HCC or MGB corresponding main study visit
Who may be able to join
- Subjects will be asked to participate leading up to the last HCC or MGB 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 HCC IRB or MGB IRB corresponding main study
Who cannot join
- Subjects meeting the following criterion are to be excluded from study participation:
- Subject unable to comply with study requirements
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Massachusetts General Hospital
Boston, Massachusetts, 02115
Matthew J Frigault, MD, (617) 724-4000, mfrigault@mgb.org
Matthew J Frigault, MD
About this study
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 (HCC# 22-175), NCT06026319 (HCC# 23-474) and NCT NCT07388277 (MGB# 2025P003179.
Read the 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.
- Conditions
- Long Term Adverse Effects, CAR-T, Duty to Follow Up, Adult, Progression-Free Survival, Disease-Free Survival
- Being tested
- Disease assessments, Tumor Biopsy, Blood test
- Keywords
- Long-Term Follow-up to CAR-T Cells, Follow-up Studies
- Medical subject headings
- Long Term Adverse Effects
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Disease assessmentsDiagnostic test
CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans as per protocol
Tumor BiopsyProcedure or surgery
Tumor Biopsy per protocol
Blood testDiagnostic test
Blood Test per protocol
| Study group | Role | What they receive |
|---|---|---|
| CAR T cells | * Eligibility to participate on this study if enrolled on an HCC IRB or MGB 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 Receives: Diagnostic Test: Disease assessments; Procedure: Tumor Biopsy; Diagnostic Test: Blood test |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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)
Measured: 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).
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Aug 2021
- Study start7 Aug 2023
- Primary completion1 Aug 2038 (estimated)
- Study completion1 Aug 2039 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Long Term Adverse EffectsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 45 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Marcela V. Maus, M.D.,Ph.D.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Marcela V. Maus, M.D.,Ph.D., Massachusetts General Hospital
- Investigators
- Matthew J Frigault, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05024175
- Sponsor's protocol ID
- 20-721
- Status last verified
- Sep 2026
- First submitted
- 23 Aug 2021
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Harvard Cancer Consortium (HCC) 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.
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up of Subjects Treated With CAR T Cells
- ClinicalTrials.gov ID
- NCT05024175
- Acronym / sponsor ID
- 20-721
- Conditions
- Long Term Adverse Effects, CAR-T, Duty to Follow Up, Adult, Progression-Free Survival, Disease-Free Survival
- Keywords
- Long-Term Follow-up to CAR-T Cells, Follow-up Studies
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 45 participants (estimated)
- Lead sponsor
- Marcela V. Maus, M.D.,Ph.D.University, hospital or other
- Study start
- 7 Aug 2023 (actual)
- Primary completion
- 1 Aug 2038 (estimated)
- Study completion
- 1 Aug 2039 (estimated)
- First posted
- 27 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 (HCC# 22-175), NCT06026319 (HCC# 23-474) and NCT NCT07388277 (MGB# 2025P003179.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CAR T cells | – | Diagnostic Test: Disease assessments, Procedure: Tumor Biopsy, Diagnostic Test: Blood test* Eligibility to participate on this study if enrolled on an HCC IRB or MGB 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Subjects will be asked to participate leading up to the last HCC or MGB corresponding main study visit
Key inclusion criteria (4 listed)
- Subjects will be asked to participate leading up to the last HCC or MGB corresponding main study visit.
- Provision of voluntary written informed consent by subject
- CAR T cells were administered in HCC IRB or MGB IRB corresponding main study
Key exclusion criteria (2 listed)
- Subject unable to comply with study requirements
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Disease assessmentsCT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans as per protocol | Diagnostic test | CAR T cells |
| Tumor BiopsyTumor Biopsy per protocol | Procedure or surgery | CAR T cells |
| Blood testBlood Test per protocol | Diagnostic test | CAR T cells |
Primary outcome
Overall Survival
Time frame: 1 month
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Overall Survival
Time frame: 6 months
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Overall Survival
Time frame: 12 months
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Overall Survival
Time frame: 24 months
defined as the time from randomization (or registration) to death due to any cause, or censored at date last known alive
Progresison Free Survival
Time frame: 1 month
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.
Progresison Free Survival
Time frame: 6 months
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.
Progresison Free Survival
Time frame: 12 months
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.
Progresison Free Survival
Time frame: 24 months
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.
New incidence or exacerbation of a pre-existing neurologic disorder or prior rheumatologic or other autoimmune disorder
Time frame: Up to 15 Years
An annual physical exam will be conducted for surveillance of manifestations indicative of oncoretroviral diseases.
New incidence of a hematologic disorder
Time frame: up to 15 years
An annual physical exam will be conducted for surveillance of manifestations indicative of oncoretroviral diseases.
Number of Participants with Treatment-Related Adverse Events as Assessed by CTCAE v 5.0
Time frame: up to 15 years
NCI Common Terminology Criteria for Adverse Events (CTCAE)
Vector-derived RCL
Time frame: baseline and in 3 months, 6 months, and annually up to 15 years
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).
Assessment of CAR T cells persistence by VCN in peripheral blood
Time frame: baseline and in 3 months, 6 months, every 6 months up to 5 years, and annually up to 15 years
VCN will be performed in DNA from whole blood to monitor for persistence of vector sequence
Progression-free Survival
Time frame: 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
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.
Overall Survival
Time frame: 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
The time from post-CAR T cells infusion to the date of death.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
- Individual participant dataSharing planned: Yes. The Harvard Cancer Consortium (HCC) 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Matthew J Frigault, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Long Term Adverse Effects and CAR-T, run by Marcela V. Maus, M.D.,Ph.D. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Matthew J Frigault, MD (617-643-6175, mfrigault@mgb.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (time frame: 1 month). The study lists 15 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2026.