CAR-T Long Term Follow Up (LTFU) Study
- Conditions
- Long Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access Program
- Interventions
- Genetic: Previously treated CAR-T patients
- Registration Number
- NCT02445222
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow up of treated patients is required. The purpose of this study is to monitor all patients exposed to CAR-T therapied for 15 years following their last CAR-T (e.g. CTL019) infusion to assess the risk of delayed adverse events (AEs), monitor for replication competent lentivirus (RCL) and assess long-term efficacy, including vector persistence.
- Detailed Description
Patients are enrolled following completion or early discontinuation from a Novartis sponsored or supported study of CAR T-Cell treatment. Patients will be followed for 15 years post treatment from the last treatment. They will be monitored for safety and efficacy within the primary treatment protocols for the protocol defined duration. Patients can drop off treatment protocols at any time to enter this long term Follow up study. Patients discontinuing from the primary treatment protocols for any reason will be enrolled in this long term follow up (LTFU). This will allow collecting data on long term safety and efficacy (as applicable) as mandated by the health authorities of all patients treated with CAR-T therapy within the concept of a single protocol.
Collection of such long term effects of CAR-T cell therapy will help to further define the risk-benefit profile of CAR-T Therapies.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 1400
- All patients who have received a CAR-T therapy and completed or discontinued early from a Novartis sponsored treatment protocol that utilized CAR-T cells or from any CAR-T trial sponsored by the University of Pennsylvania with which Novartis has a contractual agreement to co-develop the CAR technology.
- Patients who have provided informed consent for the long term follow up study prior to their study participation .
- There are no specific exclusion criteria for this study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Previously treated CAR-T patients Previously treated CAR-T patients Patients who previously were exposed to lentiviral-based CART cell therapy
- Primary Outcome Measures
Name Time Method Percentage of patients with certain events (see description) at M3 post treatment, M6, M9, M12 and then, every 6M up to year 5, yearly until year 15. The percentage of pts with listed categories: New secondary malignancies, new serious infection, new incidence of serious neurologic disorder, New incidence or exacerbation of a prior rheumatologic or other autoimmune disorder, New incidence of a hematologic disorder
- Secondary Outcome Measures
Name Time Method Percentage of patients who relapse or progress among patients who had not relapsed or progressed at study entry/re-entry;Incidence of death at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15. B- and T- lymphocyte count at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15. Percentage of patients with detectable CAR transgene levels in peripheral blood by q-PCR at pre- specified time points at M3 post treatment, M6, M9, M12 and every 6M up to year 5, yearly until year 15. Percentage of patients with detectable RCL by VSV-G at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15 Height and weight, Tanner staging, menstruation status at M3 post treatment then M6, M12 and every year until year 15.
Trial Locations
- Locations (27)
Mayo Clinic Arizona
🇺🇸Phoenix, Arizona, United States
Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States
UCSF Medical Center
🇺🇸San Francisco, California, United States
Stanford University Medical Center
🇺🇸Stanford, California, United States
Emory University School of Medicine/Winship Cancer Institute
🇺🇸Atlanta, Georgia, United States
Childrens Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
University of Chicago Medical Center, Hematology & Oncology
🇺🇸Chicago, Illinois, United States
University of Kansas Cancer Center SC
🇺🇸Westwood, Kansas, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Beth Israel Deaconess Medical Cente
🇺🇸Boston, Massachusetts, United States
Dana Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
University of Michigan Health System SC CTL019
🇺🇸Ann Arbor, Michigan, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Children's Mercy Hospital SC
🇺🇸Kansas City, Missouri, United States
Weill Cornell Medical College
🇺🇸New York, New York, United States
Duke Unversity Medical Center SC - CTL019B2205J
🇺🇸Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Ohio ST Compr Cancer Ctr James Hosp
🇺🇸Columbus, Ohio, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
The Childrens Hosp of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
UTSW/Children's Medical Center
🇺🇸Dallas, Texas, United States
MD Anderson Cancer Center SC
🇺🇸Houston, Texas, United States
University of Utah Clinical Trials Office
🇺🇸Salt Lake City, Utah, United States
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
Novartis Investigative Site
🇬🇧London, United Kingdom