Long Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access Program
Conditions
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.
Interventions
Lentiviral-based CAR-T cell therapy
Sponsors
University of Pennsylvania
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
0 Years to 100 Years
Healthy volunteers
No
Inclusion criteria
* 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 .
Exclusion criteria
* There are no specific
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
| 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. |
| Height and weight, Tanner staging, menstruation status | at M3 post treatment then M6, M12 and every year until year 15. |
Countries
Australia, Austria, Belgium, Canada, China, Denmark, Finland, France, Germany, Greece, Hong Kong, Israel, Italy, Japan, Netherlands, Norway, Saudi Arabia, Singapore, South Korea, Spain, Taiwan, United Kingdom, United States
Contacts
Primary ContactNovartis Pharmaceuticals
Backup ContactNovartis Pharmaceuticals
Outcome results
None listed