CAR-T Long Term Follow Up (LTFU) Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Previously treated CAR-T patients.
- Who it may be relevant to
- Registry conditions: Long Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access Program. Basic parameters: 0 years — 100 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Austria, Belgium, Canada +18
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy
Overview
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
- Genetic Previously treated CAR-T patients
Lentiviral-based CAR-T cell therapy
Primary outcome measures
- Percentage of patients with certain events (see description) [Time frame: at M3 post treatment, M6, M9, M12 and then, every 6M up to year 5, yearly until year 15.]
Secondary outcome measures (5)
- Percentage of patients with detectable CAR transgene levels in peripheral blood by q-PCR at pre- specified time points [Time frame: 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 [Time frame: 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 [Time frame: at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15.]
- B- and T- lymphocyte count [Time frame: 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 [Time frame: at M3 post treatment then M6, M12 and every year until year 15.]
Eligibility criteria
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 exclusion criteria for this study.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Other
Study locations
United States · 44 centers
- Mayo Clinic Arizona — Phoenix
- City of Hope National Medical Center — Duarte
- Childrens Hospital Los Angeles — Los Angeles
- UCLA Medical Center — Los Angeles
- UCSF Medical Center — San Francisco
- Stanford University Medical Center — Stanford
- Emory University School of Medicine-Winship Cancer Institute — Atlanta
- Emory University School of Medicine/Winship Cancer Institute — Atlanta
- … and 36 more centers
Spain · 11 centers
Center list to be confirmed — check the primary protocol.
Germany · 9 centers
- Novartis Investigative Site — Munich
- Novartis Investigative Site — Regensburg
- Novartis Investigative Site — Würzburg
- Novartis Investigative Site — Frankfurt am Main
- Novartis Investigative Site — Cologne
- Novartis Investigative Site — Leipzig
- Novartis Investigative Site — Berlin
- Novartis Investigative Site — Hamburg
- … and 1 more center
Italy · 7 centers
Center list to be confirmed — check the primary protocol.
Australia · 6 centers
- Novartis Investigative Site — Camperdown
- Novartis Investigative Site — Melbourne
- Novartis Investigative Site — Melbourne
- Novartis Investigative Site — Parkville
- Novartis Investigative Site — Murdoch
- Novartis Investigative Site — Herston
France · 5 centers
- Novartis Investigative Site — Nantes
- Novartis Investigative Site — Paris
- Novartis Investigative Site — Paris
- Novartis Investigative Site — Pierre-Bénite
- Novartis Investigative Site — Villejuif
Japan · 5 centers
Center list to be confirmed — check the primary protocol.
Austria · 4 centers
- Novartis Investigative Site — Linz
- Novartis Investigative Site — Salzburg
- Novartis Investigative Site — Vienna
- Novartis Investigative Site — Vienna
Canada · 4 centers
- Novartis Investigative Site — Hamilton
- Novartis Investigative Site — Toronto
- Novartis Investigative Site — Montreal
- Novartis Investigative Site — Montreal
South Korea · 4 centers
Center list to be confirmed — check the primary protocol.
Netherlands · 3 centers
Center list to be confirmed — check the primary protocol.
Israel · 2 centers
- Novartis Investigative Site — Ramat Gan
- Novartis Investigative Site — Tel Aviv
Norway · 2 centers
Center list to be confirmed — check the primary protocol.
Singapore · 2 centers
Center list to be confirmed — check the primary protocol.
United Kingdom · 2 centers
Center list to be confirmed — check the primary protocol.
Belgium · 1 center
- Novartis Investigative Site — Ghent
China · 1 center
- Novartis Investigative Site — Beijing
Denmark · 1 center
- Novartis Investigative Site — Copenhagen
Finland · 1 center
- Novartis Investigative Site — Helsinki
Greece · 1 center
- Novartis Investigative Site — Chaïdári
Hong Kong · 1 center
- Novartis Investigative Site — Hong Kong
Saudi Arabia · 1 center
Center list to be confirmed — check the primary protocol.
Taiwan · 1 center
Center list to be confirmed — check the primary protocol.
Publications
- Maziarz RT, Bishop MR, Tam CS, Borchmann P, Worel N, McGuirk JP, Holte H, Waller EK, Jaglowski S, Andreadis C, Foley SR, Westin JR, Fleury I, Ho PJ, Mielke S, Teshima T, Janakiram M, Hsu J, Izutsu K, Kersten MJ, Ghosh M, Wagner-Johnston N, Kato K, Corradini P, Ma W, Han X, Nuortti M, Awasthi R, Mundt KE, Majdan M, Maier HJ, Jegerlehner A, Salles G, Schuster SJ. Five-Year Analysis of the JULIET Tri PMID 41252666
Identifiers
NCT: NCT02445222 · CCTL019A2205B · 2014-001673-14