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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02445222




Registration number
NCT02445222
Ethics application status
Date submitted
1/05/2015
Date registered
15/05/2015
Date last updated
15/04/2020

Titles & IDs
Public title
CAR-T Long Term Follow Up (LTFU) Study
Scientific title
Long Term Follow-Up of Patients Exposed to Lentiviral-Based CAR T-Cell Therapy
Secondary ID [1] 0 0
2014-001673-14
Secondary ID [2] 0 0
CCTL019A2205B
Universal Trial Number (UTN)
Trial acronym
PAVO
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Long Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access Program 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Previously treated CAR-T patients

Other: Previously treated CAR-T patients - Patients who previously were exposed to lentiviral-based CART cell therapy


Other interventions: Previously treated CAR-T patients
Lentiviral-based CAR-T cell therapy

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of pts with certain events (see description) - Proportion 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
Timepoint [1] 0 0
at M3 post treatment, M6, M9, M12 and then, every 6M up to year 5, yearly until year 15.
Secondary outcome [1] 0 0
Proportion of patients with detectable CAR transgene levels in peripheral blood by q-PCR at pre- specified time points
Timepoint [1] 0 0
at M3 post treatment, M6, M9, M12 and every 6M up to year 5, yearly until year 15.
Secondary outcome [2] 0 0
Proportion of patients with detectable RCL by VSV-G
Timepoint [2] 0 0
at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15
Secondary outcome [3] 0 0
Proportion of patients who relapse or progress among patients who had not relapsed or progressed at study entry/re-entry;Incidence of death
Timepoint [3] 0 0
at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15.
Secondary outcome [4] 0 0
B- and T- lymphocyte count
Timepoint [4] 0 0
at M3 post treatment then M6, M9, M12 and every 6M up to year 5, yearly until year 15.
Secondary outcome [5] 0 0
Height and weight, Tanner staging, menstruation status
Timepoint [5] 0 0
at M3 post treatment then M6, M12 and every year until year 15.

Eligibility
Key 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 .
Minimum age
No limit
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- There are no specific exclusion criteria for this study.

Study design
Purpose of the study
Other
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Not Applicable
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Melbourne
Recruitment hospital [2] 0 0
Novartis Investigative Site - Parkville
Recruitment hospital [3] 0 0
Novartis Investigative Site - Camperdown
Recruitment postcode(s) [1] 0 0
3000 - Melbourne
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment postcode(s) [3] 0 0
NSW - Camperdown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
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United States of America
State/province [5] 0 0
Kansas
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United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
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Missouri
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United States of America
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New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
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Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
United States of America
State/province [16] 0 0
Wisconsin
Country [17] 0 0
Austria
State/province [17] 0 0
Linz
Country [18] 0 0
Austria
State/province [18] 0 0
Vienna
Country [19] 0 0
Austria
State/province [19] 0 0
Wien
Country [20] 0 0
Belgium
State/province [20] 0 0
Gent
Country [21] 0 0
Canada
State/province [21] 0 0
Ontario
Country [22] 0 0
Canada
State/province [22] 0 0
Quebec
Country [23] 0 0
France
State/province [23] 0 0
Paris Cedex 10
Country [24] 0 0
France
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Paris Cedex 19
Country [25] 0 0
France
State/province [25] 0 0
Paris
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France
State/province [26] 0 0
Pierre Benite
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Germany
State/province [27] 0 0
Frankfurt
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Germany
State/province [28] 0 0
Koeln
Country [29] 0 0
Germany
State/province [29] 0 0
Wuerzburg
Country [30] 0 0
Italy
State/province [30] 0 0
MB
Country [31] 0 0
Japan
State/province [31] 0 0
Fukuoka
Country [32] 0 0
Japan
State/province [32] 0 0
Hokkaido
Country [33] 0 0
Japan
State/province [33] 0 0
Kyoto
Country [34] 0 0
Japan
State/province [34] 0 0
Miyagi
Country [35] 0 0
Japan
State/province [35] 0 0
Tokyo
Country [36] 0 0
Netherlands
State/province [36] 0 0
CS
Country [37] 0 0
Netherlands
State/province [37] 0 0
Amsterdam
Country [38] 0 0
Norway
State/province [38] 0 0
Oslo
Country [39] 0 0
Spain
State/province [39] 0 0
Andalucia
Country [40] 0 0
Spain
State/province [40] 0 0
Barcelona/ Cataluny/Espanya
Country [41] 0 0
Spain
State/province [41] 0 0
Barcelona
Country [42] 0 0
Spain
State/province [42] 0 0
Catalunya
Country [43] 0 0
Spain
State/province [43] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
University of Pennsylvania
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT02445222
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
1-888-669-6682
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02445222