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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CAR T cells |
|
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Disease assessments | Diagnostic Test | CT (Computerized Tomography) scan or PET-CT (Positron Emission Tomography-Computerized Tomography) scans as per protocol |
|
| 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 |
| 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 |
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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:
Exclusion Criteria:
Subjects meeting the following criterion are to be excluded from study participation:
- Subject unable to comply with study requirements
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Subjects will be asked to participate leading up to the last DF/HCC corresponding main study visit
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Matthew J Frigault, MD | Contact | 617-643-6175 | mfrigault@mgb.org |
| Name | Affiliation | Role |
|---|---|---|
| Matthew J Frigault, MD | Massachusetts General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Massachusetts General Hospital | Recruiting | Boston | Massachusetts | 02115 | United States |
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.
Data can be shared no earlier than 1 year following the date of publication
Contact the Partners Innovations team at http://www.partners.org/innovation
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| ID | Term |
|---|---|
| D000069451 | Long Term Adverse Effects |
| ID | Term |
|---|---|
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D006403 | Hematologic Tests |
| ID | Term |
|---|---|
| D019411 | Clinical Laboratory Techniques |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D008919 | Investigative Techniques |
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| Tumor Biopsy | Procedure | Tumor Biopsy per protocol |
|
| Blood test | Diagnostic Test | Blood Test per protocol |
|
| 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 |