Efficacy and Safety of Tisagenlecleucel in Adult Patients With Refractory or Relapsed Follicular Lymphoma (ELARA)
Primary Purpose
Follicular Lymphoma
Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
tisagenlecleucel
Sponsored by
About this trial
This is an interventional treatment trial for Follicular Lymphoma focused on measuring Refractory or relapsed Follicular Lymphoma, Refractory, Relapsed, Follicular lymphoma, CTL019, Tisagenlecleucel, Chimeric antigen receptor, CAR19, CAR-T
Eligibility Criteria
Inclusion Criteria:
- Refractory or relapsed Follicular Lymphoma (Grade 1, 2, 3A)
- Radiographically measurable disease at screening
Exclusion Criteria:
- Evidence of histologic transformation
- Follicular Lymphoma Grade 3B
- Prior anti-CD19 therapy
- Prior gene therapy
- Prior adoptive T cell therapy
- Prior allogeneic hematopoietic stem cell transplant
- Active CNS involvement by malignancy
Other protocol-defined Inclusion/Exclusion criteria may apply.
Sites / Locations
- City of Hope National Medical Center Dept.ofCityofHopeMedicalCtr(1)
- UCSF Medical Center .
- H Lee Moffitt Cancer Center and Research Institute
- University of Chicago Medical Center Hematology and Oncology
- University of Kansas Hospital and Medical Center DeptofUofKansas CancerCenter-2
- Michigan Med University of Michigan
- Oregon Health and Science Univ
- University of Pennsylvania Clinical Studies Unit Perelman Center for Adv Med
- MD Anderson Cancer Center SC
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Amsterdam UMC, locatie AMC
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CTL019
Arm Description
All patients who received tisagenlecleucel infusion.
Outcomes
Primary Outcome Measures
Complete Response Rate (CRR) Per Independent Review Committee (IRC) Assessment
Complete response rate was defined as the percentage of participants with a best overall response (BOR) of complete response (CR) recorded from tisagenlecleucel infusion until progressive disease or start of new anticancer therapy, whichever came first. CRR was determined by an independent review committee (IRC) and was based on Lugano 2014 classification response criteria. The radiological response is first obtained from CT and PET studies according to the Lugano 2014 criteria. CT response is based on anatomical measurements of index/non-index/new lesions and spleen length. The possible response outcomes are complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD). PET response based on a 5-point scale (5PS) or Deauville score. The possible outcomes for PET response are complete metabolic response (CMR), partial metabolic response (PMR), no metabolic response (NMR), or progressive metabolic disease (PMD).
Secondary Outcome Measures
Overall Response Rate (ORR) Per IRC Assessment
Overall response rate is defined as the percentage of participants with a best overall disease response of complete response (CR) or partial response (PR). Response was evaluated per Lugano 2014 classification response criteria. The radiological response is first obtained from CT and PET studies according to the Lugano 2014 criteria. CT response is based on anatomical measurements of index/non-index/new lesions and spleen length. The possible response outcomes are complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD). PET response based on a 5-point scale (5PS) or Deauville score. The possible outcomes for PET response are complete metabolic response (CMR), partial metabolic response (PMR), no metabolic response (NMR), or progressive metabolic disease (PMD).
Duration of Response (DOR) Per IRC
Duration of response (DOR) applied only to participants whose best overall disease response was CR or PR. It is defined as the time from the date of first documented disease response (CR or PR) to the date of first documented progression or death due to follicular lymphoma (FL).
Progression Free Survival (PFS)
Time from tisagenlecleucel infusion to first documented disease progression or death due to any cause
Overall Survival (OS)
Time from tisagenlecleucel infusion to death due to any cause
Tisagenlecleucel Transgene Concentration
Transgene concentration as detected by qPCR in target tissue
Cmax; Cellular Kinetic Parameter of Tisagenlecleucel
The maximum (peak) observed in peripheral blood or other body fluid drug concentration after single dose administration (% or copies/ µg)
Tmax; Cellular Kinetic Parameter of Tisagenlecleucel
The time to reach maximum (peak) peripheral blood or other body fluid drug concentration after single dose administration (days)
AUC0-28; Cellular Kinetic Parameter of Tisagenlecleucel
The AUC from time zero to day 28, in peripheral blood (%*days or days*copies/ µg)
AUC0-84d; Cellular Kinetic Parameter of Tisagenlecleucel
The AUC from time zero to day 84, in peripheral blood (%*days or days*copies/ µg)
T1/2; Cellular Kinetic Parameter of Tisagenlecleucel
The half-life associated with the elimination phase slope of a semi logarithmic concentration-time curve (days) in peripheral blood
Tlast; Cellular Kinetic Parameter of Tisagenlecleucel
The last observed measureable timepoint after dose administration
Summary of Exposure of CD3+ Tisagenlecleucel Cells in Peripheral Blood
In vivo cellular kinetics of CD3+ tisagenlecleucel cells detected by flow cytometry
Humoral Immunogenicity
Antibody titers specific to the tisagenlecleucel molecule prior to and following infusion.
Cellular Immunogenicity
Presence of T lymphocytes activated by the tisagenlecleucel protein
Summary Scores of PRO Measured by SF-36v2 Quality of Life Questionnaire
Effect of tisagenlecleucel therapy on Patient reported outcomes
Summary Scores of PRO Measured by EQ-5D-3L Quality of Life Questionnaire
Effect of tisagenlecleucel therapy on Patient reported outcomes
Summary Scores of PRO Measured by FACT-Lym Quality of Life Questionnaire
Effect of tisagenlecleucel therapy on Patient reported outcomes
Full Information
NCT ID
NCT03568461
First Posted
May 24, 2018
Last Updated
September 5, 2023
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT03568461
Brief Title
Efficacy and Safety of Tisagenlecleucel in Adult Patients With Refractory or Relapsed Follicular Lymphoma
Acronym
ELARA
Official Title
A Phase II, Single Arm, Multicenter Open Label Trial to Determine the Efficacy and Safety of Tisagenlecleucel (CTL019) in Adult Patients With Refractory or Relapsed Follicular Lymphoma
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 12, 2018 (Actual)
Primary Completion Date
November 24, 2020 (Actual)
Study Completion Date
May 22, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This is a multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in adult patients with relapsed or refractory FL.
Detailed Description
This single-arm, open label study had the following sequential phases: Screening, Pretreatment, Treatment and Follow-up. In the Pre-treatment phase, the patient could undergo bridging therapy (optional) and lymphodepleting (LD) chemotherapy. Treatment and Follow-up Phase included tisagenlecleucel infusion, and safety and efficacy follow-up for at least 24 months. For all the patients who received tisagenlecleucel infusion, additional survival follow-up was to be performed to determine survival status every 3 months.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Follicular Lymphoma
Keywords
Refractory or relapsed Follicular Lymphoma, Refractory, Relapsed, Follicular lymphoma, CTL019, Tisagenlecleucel, Chimeric antigen receptor, CAR19, CAR-T
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
98 (Actual)
8. Arms, Groups, and Interventions
Arm Title
CTL019
Arm Type
Experimental
Arm Description
All patients who received tisagenlecleucel infusion.
Intervention Type
Biological
Intervention Name(s)
tisagenlecleucel
Other Intervention Name(s)
CTL019
Intervention Description
Tisagenlecleucel is single infusion.
Primary Outcome Measure Information:
Title
Complete Response Rate (CRR) Per Independent Review Committee (IRC) Assessment
Description
Complete response rate was defined as the percentage of participants with a best overall response (BOR) of complete response (CR) recorded from tisagenlecleucel infusion until progressive disease or start of new anticancer therapy, whichever came first. CRR was determined by an independent review committee (IRC) and was based on Lugano 2014 classification response criteria. The radiological response is first obtained from CT and PET studies according to the Lugano 2014 criteria. CT response is based on anatomical measurements of index/non-index/new lesions and spleen length. The possible response outcomes are complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD). PET response based on a 5-point scale (5PS) or Deauville score. The possible outcomes for PET response are complete metabolic response (CMR), partial metabolic response (PMR), no metabolic response (NMR), or progressive metabolic disease (PMD).
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Overall Response Rate (ORR) Per IRC Assessment
Description
Overall response rate is defined as the percentage of participants with a best overall disease response of complete response (CR) or partial response (PR). Response was evaluated per Lugano 2014 classification response criteria. The radiological response is first obtained from CT and PET studies according to the Lugano 2014 criteria. CT response is based on anatomical measurements of index/non-index/new lesions and spleen length. The possible response outcomes are complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD). PET response based on a 5-point scale (5PS) or Deauville score. The possible outcomes for PET response are complete metabolic response (CMR), partial metabolic response (PMR), no metabolic response (NMR), or progressive metabolic disease (PMD).
Time Frame
1 year
Title
Duration of Response (DOR) Per IRC
Description
Duration of response (DOR) applied only to participants whose best overall disease response was CR or PR. It is defined as the time from the date of first documented disease response (CR or PR) to the date of first documented progression or death due to follicular lymphoma (FL).
Time Frame
1 year
Title
Progression Free Survival (PFS)
Description
Time from tisagenlecleucel infusion to first documented disease progression or death due to any cause
Time Frame
2 years
Title
Overall Survival (OS)
Description
Time from tisagenlecleucel infusion to death due to any cause
Time Frame
2 years
Title
Tisagenlecleucel Transgene Concentration
Description
Transgene concentration as detected by qPCR in target tissue
Time Frame
2 years
Title
Cmax; Cellular Kinetic Parameter of Tisagenlecleucel
Description
The maximum (peak) observed in peripheral blood or other body fluid drug concentration after single dose administration (% or copies/ µg)
Time Frame
2 years
Title
Tmax; Cellular Kinetic Parameter of Tisagenlecleucel
Description
The time to reach maximum (peak) peripheral blood or other body fluid drug concentration after single dose administration (days)
Time Frame
2 years
Title
AUC0-28; Cellular Kinetic Parameter of Tisagenlecleucel
Description
The AUC from time zero to day 28, in peripheral blood (%*days or days*copies/ µg)
Time Frame
2 years
Title
AUC0-84d; Cellular Kinetic Parameter of Tisagenlecleucel
Description
The AUC from time zero to day 84, in peripheral blood (%*days or days*copies/ µg)
Time Frame
2 years
Title
T1/2; Cellular Kinetic Parameter of Tisagenlecleucel
Description
The half-life associated with the elimination phase slope of a semi logarithmic concentration-time curve (days) in peripheral blood
Time Frame
2 years
Title
Tlast; Cellular Kinetic Parameter of Tisagenlecleucel
Description
The last observed measureable timepoint after dose administration
Time Frame
2 years
Title
Summary of Exposure of CD3+ Tisagenlecleucel Cells in Peripheral Blood
Description
In vivo cellular kinetics of CD3+ tisagenlecleucel cells detected by flow cytometry
Time Frame
2 years
Title
Humoral Immunogenicity
Description
Antibody titers specific to the tisagenlecleucel molecule prior to and following infusion.
Time Frame
2 years
Title
Cellular Immunogenicity
Description
Presence of T lymphocytes activated by the tisagenlecleucel protein
Time Frame
2 years
Title
Summary Scores of PRO Measured by SF-36v2 Quality of Life Questionnaire
Description
Effect of tisagenlecleucel therapy on Patient reported outcomes
Time Frame
2 years
Title
Summary Scores of PRO Measured by EQ-5D-3L Quality of Life Questionnaire
Description
Effect of tisagenlecleucel therapy on Patient reported outcomes
Time Frame
2 years
Title
Summary Scores of PRO Measured by FACT-Lym Quality of Life Questionnaire
Description
Effect of tisagenlecleucel therapy on Patient reported outcomes
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Refractory or relapsed Follicular Lymphoma (Grade 1, 2, 3A)
Radiographically measurable disease at screening
Exclusion Criteria:
Evidence of histologic transformation
Follicular Lymphoma Grade 3B
Prior anti-CD19 therapy
Prior gene therapy
Prior adoptive T cell therapy
Prior allogeneic hematopoietic stem cell transplant
Active CNS involvement by malignancy
Other protocol-defined Inclusion/Exclusion criteria may apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
City of Hope National Medical Center Dept.ofCityofHopeMedicalCtr(1)
City
Duarte
State/Province
California
ZIP/Postal Code
91010 3000
Country
United States
Facility Name
UCSF Medical Center .
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
H Lee Moffitt Cancer Center and Research Institute
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
University of Chicago Medical Center Hematology and Oncology
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
University of Kansas Hospital and Medical Center DeptofUofKansas CancerCenter-2
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Michigan Med University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109 5271
Country
United States
Facility Name
Oregon Health and Science Univ
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
University of Pennsylvania Clinical Studies Unit Perelman Center for Adv Med
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
MD Anderson Cancer Center SC
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Novartis Investigative Site
City
Herston
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
Novartis Investigative Site
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3000
Country
Australia
Facility Name
Novartis Investigative Site
City
Camperdown
ZIP/Postal Code
NSW
Country
Australia
Facility Name
Novartis Investigative Site
City
Linz
ZIP/Postal Code
4020
Country
Austria
Facility Name
Novartis Investigative Site
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
Novartis Investigative Site
City
Paris 10
ZIP/Postal Code
75475
Country
France
Facility Name
Novartis Investigative Site
City
Pierre Benite
ZIP/Postal Code
69495
Country
France
Facility Name
Novartis Investigative Site
City
Koeln
ZIP/Postal Code
50937
Country
Germany
Facility Name
Novartis Investigative Site
City
Muenchen
ZIP/Postal Code
81377
Country
Germany
Facility Name
Novartis Investigative Site
City
Ulm
ZIP/Postal Code
89081
Country
Germany
Facility Name
Novartis Investigative Site
City
Bologna
State/Province
BO
ZIP/Postal Code
40138
Country
Italy
Facility Name
Novartis Investigative Site
City
Milano
State/Province
MI
ZIP/Postal Code
20132
Country
Italy
Facility Name
Novartis Investigative Site
City
Fukuoka city
State/Province
Fukuoka
ZIP/Postal Code
812-8582
Country
Japan
Facility Name
Novartis Investigative Site
City
Sapporo city
State/Province
Hokkaido
ZIP/Postal Code
060 8648
Country
Japan
Facility Name
Novartis Investigative Site
City
Sendai city
State/Province
Miyagi
ZIP/Postal Code
980 8574
Country
Japan
Facility Name
Amsterdam UMC, locatie AMC
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
Novartis Investigative Site
City
Oslo
ZIP/Postal Code
NO 0424
Country
Norway
Facility Name
Novartis Investigative Site
City
Sevilla
State/Province
Andalucia
ZIP/Postal Code
41013
Country
Spain
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
Novartis Investigative Site
City
Birmingham
ZIP/Postal Code
B15 2TH
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.
IPD Sharing URL
https://www.clinicalstudydatarequest.com
Citations:
PubMed Identifier
35973192
Citation
Salles G, Schuster SJ, Dreyling M, Fischer L, Kuruvilla J, Patten PEM, von Tresckow B, Smith SM, Jimenez-Ubieto A, Davis KL, Anjos C, Chu J, Zhang J, Lobetti Bodoni C, Thieblemont C, Fowler NH, Dickinson M, Martinez-Lopez J, Wang Y, Link BK. Efficacy comparison of tisagenlecleucel vs usual care in patients with relapsed or refractory follicular lymphoma. Blood Adv. 2022 Nov 22;6(22):5835-5843. doi: 10.1182/bloodadvances.2022008150.
Results Reference
derived
PubMed Identifier
34921238
Citation
Fowler NH, Dickinson M, Dreyling M, Martinez-Lopez J, Kolstad A, Butler J, Ghosh M, Popplewell L, Chavez JC, Bachy E, Kato K, Harigae H, Kersten MJ, Andreadis C, Riedell PA, Ho PJ, Perez-Simon JA, Chen AI, Nastoupil LJ, von Tresckow B, Ferreri AJM, Teshima T, Patten PEM, McGuirk JP, Petzer AL, Offner F, Viardot A, Zinzani PL, Malladi R, Zia A, Awasthi R, Masood A, Anak O, Schuster SJ, Thieblemont C. Tisagenlecleucel in adult relapsed or refractory follicular lymphoma: the phase 2 ELARA trial. Nat Med. 2022 Feb;28(2):325-332. doi: 10.1038/s41591-021-01622-0. Epub 2021 Dec 17.
Results Reference
derived
Learn more about this trial
Efficacy and Safety of Tisagenlecleucel in Adult Patients With Refractory or Relapsed Follicular Lymphoma
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