Long term follow-up study for patients who have been treated with lentivirus-based chimeric antigen receptor (CAR) T-Cell Therapy

• Register: EUCTR
• Last refreshed on: 13 October 2020
• Main ID: EUCTR2014-001673-14-AT
• Date of registration: 07/07/2015
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Long term follow-up study for patients who have been treated with lentivirus-based chimeric antigen receptor (CAR) T-Cell Therapy
• Scientific title: Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy - CD19 CART Long Term Follow Up (LTFU) study
• Date of first enrolment: 06/10/2015
• Target sample size: 700
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-001673-14
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Austria, Belgium, Brazil, Canada, China, Denmark, Finland, France, Germany, Hong Kong, Italy, Japan, Korea, Republic of, Netherlands, Norway, Singapore, Spain, Sweden, Switzerland, Taiwan, United Kingdom, United States

Contacts

• Name: Drug Regulatory Affairs
• Affiliation: Novartis Pharma GmbH

• Name: Drug Regulatory Affairs
• Affiliation: Novartis Pharma GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
1.Patients must have received CAR-T therapy within one of the
following:
• Novartis or Penn sponsored CAR-T treatment trials where CAR-T was given as monotherapy or as combination therapy.
• Novartis managed access programs outside of the commercial setting,
i.e. where CAR-T therapy was intended to be given in the setting of a
Novartis or Penn sponsored CAR-T treatment trial
2.Patients must provide informed consent prior to their entry into this
study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 255
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 340
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 105

Exclusion criteria:
- There are no specific exclusion criteria for this study.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Patients who have received CAR T-cell therapy in the context of a Novartis and/or Penn treatment trial.
MedDRA version: 21.0 Level: PT Classification code 10003917 Term: B-cell type acute leukaemia System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA version: 20.0 Level: PT Classification code 10003899 Term: B-cell lymphoma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

Therapeutic area: Diseases [C] - Cancer [C04]

Intervention(s)

Product Name: CTL019
Product Code: CTL019
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: tisagenlecleucel-T (USAN)
Current Sponsor code: CTL019
Other descriptive name: AUTOLOGOUS T CELLS TRANSDUCED WITH LENTIVIRAL VECTOR CONTAINING A CHIMERIC ANTIGEN RECEPTOR DIRECTED AGAINST CD19
Concentration unit: Other
Concentration type: range
Concentration number: 100000000-500000000

Primary Outcome(s)

Primary end point(s): Proportion of patients with events in each of the following categories:
• New secondary malignancies
• New serious infections,
• New incidence of serious neurologic disorder,
• New incidence or exacerbation of a prior rheumatologic or other
autoimmune disorder,
• New incidence of a hematologic disorder
• Other adverse events considered related to CAR T cell therapy

Secondary Objective: 1 • Monitor the persistence of modified T-cells in peripheral blood
2 • Monitor for RCL (Replication Competent Lentivirus)
3 • Assess the long-term efficacy of CAR-T
4 • Monitor lymphocyte levels
5 • Describe the growth, development, and female reproductive status
for patients who were aged < 18 years at the time of the initial CAR-T
infusion

Timepoint(s) of evaluation of this end point: as defined per protocol

Main Objective: The purpose of this study is to monitor all patients exposed to CAR Tcells (CAR-T) for 15 years following last CAR-T (e.g.CTL019) infusion to
assess the risk of delayed adverse events (AEs) and assess long term
efficacy, including vector persistence.
Primary objective is:
to describe selected, delayed AEs that are suspected to be related to
previous CAR T-cell therapy as outlined in current Health Authority
guidelines

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: as defined per protocol

Secondary end point(s): 1- Proportion of patients with detectable CAR transgene levels in
peripheral blood by q-PCR at pre-specified time points
2- Proportion of patients with detectable RCL by VSV-G q-PCR in
peripheral blood at pre-specified time points
3- Proportion of patients who relapse or progress among patients who
had not relapsed or progressed at study entry/re-entry
and Incidence of death
4- B and T lymphocyte count
5-Height and weight, Tanner staging, menstruation status

Secondary ID(s)

CCTL019A2205B

NCT02445222

Source(s) of Monetary Support

Novartis Pharma Services AG

Ethics review

Status: Approved
Approval date: 12/07/2015
Contact: