CAR-T Long Term Follow-Up Study
Research type
Research Study
Full title
Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy
IRAS ID
264926
Contact name
Piers Patten
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2014-001673-14
Clinicaltrials.gov Identifier
Duration of Study in the UK
14 years, 10 months, 28 days
Research summary
This is a long-term follow up study to look at the safety and how effective the treatment of patients who have been exposed to an investigational product known as tisagenlecleucel (also known as CAR-T).
Novartis have a number of trials that test tisagenlecleucel in a variety of blood cancers. Tisagenlecleucel is manufactured in a laboratory using a patient's own white blood cells (specifically T cells) which fight off infections in the human body. The T cells are collected from a patient using a technique known as 'Leukapheresis' and then they are genetically modified in a laboratory by using a weakened form of a virus. The virus does not cause infection itself but it is an excellent transporter of genetic material and allows the integration of this material into the T cell, causing a specific protein to be displayed on it's cell surface which recognises and hopefully kills specific cancer cells in the patient's body that also display a matching protein. These modified T cells are now known as 'tisagenlecleucel' and they are given back to the patient via an intravenous infusion in the arm. This type of intervention is known as 'gene therapy'.
Health Authorities have guidance on gene therapy that utilize viruses to genetically modify cells and they require long term safety and efficacy follow up of all treated patients.
The purpose of this study is to monitor all patients who have been exposed to tisagenlecleucel for 15 years following their last infusion to assess the risk of any delayed side effects and long term efficacy, including checking the presence of the virus in the blood.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
20/SC/0245
Date of REC Opinion
23 Sep 2020
REC opinion
Further Information Favourable Opinion