Diffuse Large B-cell LymphomaNCT05541341
Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies
An observational study of Diffuse Large B-cell Lymphoma and Acute Lymphoblastic Leukemia, run by Novartis Pharmaceuticals at 7 sites in Brazil.
Trial summary
This will be a multicenter, national, non-interventional, prospective cohort study Read more about this study
- Who is it for?
- Ages 0 to 100 years
- Conditions the study is for
- Diffuse Large B-cell Lymphoma, Acute Lymphoblastic Leukemia, Follicular Lymphoma
- What is being tested?
- tisagenlecleucel
- Type of treatment
- Injection or infusion
- How many people
- 200 participants
- Where
- 7 sitesBrazil
- Study dates
- 23 Nov 2023to 23 Nov 2043
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study population will include pediatric/ adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia aged 0-25 years and adult patients aged ≥ 18 years with relapsed/refractory Diffuse Large B-cell Lymphoma and relapsed/refractory follicular lymphoma, who received tisagenlecleucel infusion in the commercial setting or out-of-specification.
Who may be able to join
- Patients eligible for inclusion in this study must meet the following criteria:
- Patients who receive tisagenlecleucel infusion in the commercial setting or out-of-specification (OOS) use, AND
- Signed informed consent must be obtained prior to participation in study, AND
- For ALL participants:
- Patients of any gender aged 0-17 years (named as pediatric) with relapsed/ refractory B-cell ALL diagnosis that received tisagenlecleucel infusion, OR
- Patients of any gender, aged 18-25 years (named as adults) - with relapsed/ refractory B-cell ALL diagnosis that received tisagenlecleucel infusion, OR
- For DBLCL and FL participants:
- Patients of any gender aged 18 years or older, who have been diagnosed with relapsed/ refractory Diffuse Large B-cell Lymphoma and received tisagenlecleucel infusion.
Who cannot join
- Patients who did not consent to data collection.
- Patients who received tisagenlecleucel infusion as part of any interventional clinical trial.
Trial locations and contacts
7 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
7 sites in 1 country
Brazil7 sites, 7 recruiting
- Recruiting
Novartis Investigative Site
Curitiba, Paraná, 81520-060
- Recruiting
Novartis Investigative Site
Minas Gerais, Belo Horizonte, 34006-059
- Recruiting
Novartis Investigative Site
Salvador, Estado de Bahia, 41253-190
- Recruiting
Novartis Investigative Site
São Paulo, São Paulo, 01323-900
- Recruiting
Novartis Investigative Site
São Paulo, São Paulo, 04544-000
- Recruiting
Novartis Investigative Site
São Paulo, 01409-902
- Recruiting
Novartis Investigative Site
São Paulo, 01509-010
About this study
Official title: Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies: a 15-year Prospective Registry Study on Three Cohorts.
This will be a multicenter, national, non-interventional, prospective cohort study
Read the detailed description
Eligible participants will be pediatric (<18 years) and adult patients (aged 18 years or older) with B-cell malignancies who have received tisagenlecleucel through the commercial setting or out-of-specification (OOS) use in Brazil. We will collect data prospectively and complement missing information with retrospective data collection, when necessary. It is anticipated that approximately 200 patients will be enrolled in the cohort over 5 years divided among the study indications.
Since this is a non-interventional study, no administration of study drug or application of questionnaires will be mandated by this protocol. The study will consist of a "Pre-infusion" and a "Post infusion follow-up period" for up to 15 years post tisagenlecleucel infusion. All patients will be followed until death or last scheduled visit, whichever comes first.
For the study, "pre-infusion" and "follow-up post infusion" phases are defined as:
- "Pre-infusion" will consist of the patient's information from the time of diagnosis untiljust prior to infusion with tisagenlecleucel.
- "Follow-up Post infusion" information will comprise any information from the infusionof tisagenlecleucel onwards.
- Being tested
- tisagenlecleucel
- Keywords
- Diffuse Large B-cell Lymphoma, DLBCL, Acute Lymphoblastic Leukemia, ALL, Tisagenlecleucel, Brazil, NIS, Follicular Lymphoma
- Medical subject headings
- Lymphoma, Large B-Cell, Diffuse, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Lymphoma, Follicular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
tisagenlecleucelOther
Prospective observational study. There is no treatment allocation. Patients prescribed with tisagenlecleucel in the commercial setting or out-of-specification (OOS) are eligible to enroll into this study
| Study group | Role | What they receive |
|---|---|---|
| Acute Lymphoblastic Leukemia (ALL) | Children/young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia who received tisagenlecleucel infusion Receives: Other: tisagenlecleucel | |
| Diffuse Large B-cell Lymphoma (DLBCL) | Adult patients with relapsed/refractory Diffuse Large B-cell Lymphoma who received tisagenlecleucel infusion Receives: Other: tisagenlecleucel | |
| Follicular Lymphoma (FL) | Patients of any gender aged 18 year or older, with relapsed/refractory Follicular Lymphoma who received tisagenlecleucel infusion. Receives: Other: tisagenlecleucel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR)
The overall response rate will be defined as the total proportion of participants exhibiting either the best overall response (BOR) of complete or partial responses and the proportion of patient with BOR of CR/PR or CR/CRi for ALL patients will be reported along with its 95% CI.
For ALL participants, the BOR will be defined as a CR or a CRi in accordance with National Comprehensive Cancer Network (NCCN) guidelines and previous guidelines (Appelbaum et al 2007)(Cheson et al 2003).
For lymphomas, the BOR will be defined as a CR or PR in accordance with the Cheson response criteria (Cheson et al 2007) and the Lugano classification (Cheson et al 2016).
Measured: Up to 15 years
MRD negative overall response rate
The percentage of B-cell ALL patients who achieve a Best Overall Response (BOR) of CR or CRi with a Minimal residual disease (MRD) negative bone marrow will be provided with 95% CI.
Measured: Up to 15 years
Duration of overall response (DOR)
Duration of overall response (DOR) applies only to patients whose best overall disease response was either:
- CR or PR for patients with lymphomas, or
- CR or a CRi for patients with ALL.
DOR will be defined as the time from the date of first documented disease response (Complete Response (CR) or PR for patients with lymphomas, and Complete Remission (CR) or CRi for patients with ALL), whichever occurs first, to the date of first documented progression or first documented relapse according to indication, or to the date of death due to the underlying disease.
In case a patient does not have progression/relapse or death due to underlying disease (defined as the event for this outcome) prior to data cut-off, DOR will be censored at the date of the last assessment on or prior to the earliest censoring event.
Measured: Up to 15 years
Relapse-free survival (RFS)
RFS is measured by the time from date of first documented disease response as CR or CRi to relapse or death due to any cause in ALL patients.
In case a patient does not have relapse or death due to any cause prior to data cutoff, RFS will be censored at the date of the last adequate assessment on or prior to the earliest censoring event.
Measured: Up to 15 years
Event-free survival (EFS) for ALL patients
EFS is the time from date of first tisagenlecleucel infusion to treatment failure, relapse or death from any cause, whichever occurred first, for B-cell ALL patients.
Measured: Up to 15 years
Progression free survival (PFS) for DLBCL patients
PFS is defined as the time from the date of first infusion to the date of event defined as the first documented progression of lymphoma or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of the last adequate assessment.
In case a patient does not have progression or death prior to data cutoff, PFS will be censored at the date of the last adequate assessment on or prior to the earliest censoring event.
Measured: Up to 15 years
Overall survival (OS)
Overall survival is the time from date of first tisagenlecleucel infusion to the date of death due to any reason, In case a patient is alive at the date of last contact on or before data cutoff, OS is censored at the date of last contact.
Measured: Up to 15 years
Number of ALL patients with hematologic recovery
Dates of hematological recovery (i.e., dates of Absolute Neutrophil Count (ANC) and platelet recovery) will be collected.
ANC recovery is defined as an ANC of ≥ 0.5 × 109/L (500/mm^3) for 3 consecutive laboratory values obtained on different days. Date of ANC recovery is the date of the first of 3 consecutive laboratory values where the ANC is ≥ 0.5 × 109/L (CIBMTR).
The first date of the 3 consecutive laboratory values obtained on different days where the platelet count was ≥ 20 × 109/L should be recorded. It should be ensured that no platelet transfusions were administered for 7 days immediately preceding this date (CIBMTR).
Measured: Up to 15 years
Secondary outcomes (4)
The type and frequency of SAEs and AE of special interest
The type and frequency of SAEs and AE of special interest (including secondary malignancies) will be collected
Measured: Up to 15 years
Incidence and severity of CRS and ICANS among HTLV 1 and 2 positive versus HTLV 1 and 2 negative patients
Incidence and severity of Cytokine release syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) among Human T-cell Lymphotropic Virus (HTLV) 1 and 2 positive versus HTLV 1 and 2 negative patients.
For CRS AE the protocol will follow the American Society of Transplant and Cellular Therapy (ASTCT) CRS Consensus Grading.
For ICANS AE the protocol will follow ASTCT consensus as well. Which establishes the Immune effector Cell-associated Encephalopathy (ICE Score) for adults/ adolescents and Cornell Assessment of Pediatric Delirium (CAPD) for pediatric patients under 12 years
Measured: Up to 15 years
Pregnancy rates
Pregnancy rates will be collected
Measured: Up to 15 years
Number of patients with confirmed secondary malignancies diagnosis
Number of patients with confirmed secondary malignancies diagnosis will be collected
Measured: Up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2022
- Study start23 Nov 2023
- Primary completion23 Nov 2043 (estimated)
- Study completion23 Nov 2043 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Diffuse Large B-cell LymphomaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05541341
- Sponsor's protocol ID
- CCTL019BBR02
- Status last verified
- Jun 2025
- First submitted
- 13 Sep 2022
- Last updated
- 6 Jun 2025
- Results posted
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness and Safety of Tisagenlecleucel Therapy in Brazilian Patients With B-lymphocyte Malignancies: a 15-year Prospective Registry Study on Three Cohorts.
- ClinicalTrials.gov ID
- NCT05541341
- Acronym / sponsor ID
- CCTL019BBR02
- Keywords
- Diffuse Large B-cell Lymphoma, DLBCL, Acute Lymphoblastic Leukemia, ALL, Tisagenlecleucel, Brazil, NIS, Follicular Lymphoma
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 23 Nov 2023 (actual)
- Primary completion
- 23 Nov 2043 (estimated)
- Study completion
- 23 Nov 2043 (estimated)
- First posted
- 15 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This will be a multicenter, national, non-interventional, prospective cohort study
Detailed description
Eligible participants will be pediatric (<18 years) and adult patients (aged 18 years or older) with B-cell malignancies who have received tisagenlecleucel through the commercial setting or out-of-specification (OOS) use in Brazil. We will collect data prospectively and complement missing information with retrospective data collection, when necessary. It is anticipated that approximately 200 patients will be enrolled in the cohort over 5 years divided among the study indications.
Since this is a non-interventional study, no administration of study drug or application of questionnaires will be mandated by this protocol. The study will consist of a "Pre-infusion" and a "Post infusion follow-up period" for up to 15 years post tisagenlecleucel infusion. All patients will be followed until death or last scheduled visit, whichever comes first.
For the study, "pre-infusion" and "follow-up post infusion" phases are defined as:
- "Pre-infusion" will consist of the patient's information from the time of diagnosis untiljust prior to infusion with tisagenlecleucel.
- "Follow-up Post infusion" information will comprise any information from the infusionof tisagenlecleucel onwards.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Acute Lymphoblastic Leukemia (ALL) | – | Other: tisagenlecleucelChildren/young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia who received tisagenlecleucel infusion |
| Diffuse Large B-cell Lymphoma (DLBCL) | – | Other: tisagenlecleucelAdult patients with relapsed/refractory Diffuse Large B-cell Lymphoma who received tisagenlecleucel infusion |
| Follicular Lymphoma (FL) | – | Other: tisagenlecleucelPatients of any gender aged 18 year or older, with relapsed/refractory Follicular Lymphoma who received tisagenlecleucel infusion. |
Who may qualify
- Ages
- 0 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study population will include pediatric/ adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia aged 0-25 years and adult patients aged ≥ 18 years with relapsed/refractory Diffuse Large B-cell Lymphoma and relapsed/refractory follicular lymphoma, who received tisagenlecleucel infusion in the commercial setting or out-of-specification.
Key inclusion criteria (8 listed)
- Patients who receive tisagenlecleucel infusion in the commercial setting or out-of-specification (OOS) use, AND
- Signed informed consent must be obtained prior to participation in study, AND
- Patients of any gender aged 0-17 years (named as pediatric) with relapsed/ refractory B-cell ALL diagnosis that received tisagenlecleucel infusion, OR
- Patients of any gender, aged 18-25 years (named as adults) - with relapsed/ refractory B-cell ALL diagnosis that received tisagenlecleucel infusion, OR
- Patients of any gender aged 18 years or older, who have been diagnosed with relapsed/ refractory Diffuse Large B-cell Lymphoma and received tisagenlecleucel infusion.
Key exclusion criteria (2 listed)
- Patients who did not consent to data collection.
- Patients who received tisagenlecleucel infusion as part of any interventional clinical trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| tisagenlecleucelProspective observational study. There is no treatment allocation. Patients prescribed with tisagenlecleucel in the commercial setting or out-of-specification (OOS) are eligible to enroll into this study | Other | Acute Lymphoblastic Leukemia (ALL), Diffuse Large B-cell Lymphoma (DLBCL), Follicular Lymphoma (FL) |
Primary outcome
Overall response rate (ORR)
Time frame: Up to 15 years
The overall response rate will be defined as the total proportion of participants exhibiting either the best overall response (BOR) of complete or partial responses and the proportion of patient with BOR of CR/PR or CR/CRi for ALL patients will be reported along with its 95% CI. For ALL participants, the BOR will be defined as a CR or a CRi in accordance with National Comprehensive Cancer Network (NCCN) guidelines and previous guidelines (Appelbaum et al 2007)(Cheson et al 2003). For lymphomas, the BOR will be defined as a CR or PR in accordance with the Cheson response criteria (Cheson et al 2007) and the Lugano classification (Cheson et al 2016).
MRD negative overall response rate
Time frame: Up to 15 years
The percentage of B-cell ALL patients who achieve a Best Overall Response (BOR) of CR or CRi with a Minimal residual disease (MRD) negative bone marrow will be provided with 95% CI.
Duration of overall response (DOR)
Time frame: Up to 15 years
Duration of overall response (DOR) applies only to patients whose best overall disease response was either: * CR or PR for patients with lymphomas, or * CR or a CRi for patients with ALL. DOR will be defined as the time from the date of first documented disease response (Complete Response (CR) or PR for patients with lymphomas, and Complete Remission (CR) or CRi for patients with ALL), whichever occurs first, to the date of first documented progression or first documented relapse according to indication, or to the date of death due to the underlying disease. In case a patient does not have progression/relapse or death due to underlying disease (defined as the event for this outcome) prior to data cut-off, DOR will be censored at the date of the last assessment on or prior to the earliest censoring event.
Relapse-free survival (RFS)
Time frame: Up to 15 years
RFS is measured by the time from date of first documented disease response as CR or CRi to relapse or death due to any cause in ALL patients. In case a patient does not have relapse or death due to any cause prior to data cutoff, RFS will be censored at the date of the last adequate assessment on or prior to the earliest censoring event.
Event-free survival (EFS) for ALL patients
Time frame: Up to 15 years
EFS is the time from date of first tisagenlecleucel infusion to treatment failure, relapse or death from any cause, whichever occurred first, for B-cell ALL patients.
Progression free survival (PFS) for DLBCL patients
Time frame: Up to 15 years
PFS is defined as the time from the date of first infusion to the date of event defined as the first documented progression of lymphoma or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of the last adequate assessment. In case a patient does not have progression or death prior to data cutoff, PFS will be censored at the date of the last adequate assessment on or prior to the earliest censoring event.
Overall survival (OS)
Time frame: Up to 15 years
Overall survival is the time from date of first tisagenlecleucel infusion to the date of death due to any reason, In case a patient is alive at the date of last contact on or before data cutoff, OS is censored at the date of last contact.
Number of ALL patients with hematologic recovery
Time frame: Up to 15 years
Dates of hematological recovery (i.e., dates of Absolute Neutrophil Count (ANC) and platelet recovery) will be collected. ANC recovery is defined as an ANC of ≥ 0.5 × 109/L (500/mm^3) for 3 consecutive laboratory values obtained on different days. Date of ANC recovery is the date of the first of 3 consecutive laboratory values where the ANC is ≥ 0.5 × 109/L (CIBMTR). The first date of the 3 consecutive laboratory values obtained on different days where the platelet count was ≥ 20 × 109/L should be recorded. It should be ensured that no platelet transfusions were administered for 7 days immediately preceding this date (CIBMTR).
4 secondary outcomes
The type and frequency of SAEs and AE of special interest
Time frame: Up to 15 years
Incidence and severity of CRS and ICANS among HTLV 1 and 2 positive versus HTLV 1 and 2 negative patients
Time frame: Up to 15 years
Pregnancy rates
Time frame: Up to 15 years
Number of patients with confirmed secondary malignancies diagnosis
Time frame: Up to 15 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jun 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Diffuse Large B-cell Lymphoma and Acute Lymphoblastic Leukemia, run by Novartis Pharmaceuticals at 7 sites in Brazil.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Brazil. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (+41613241111, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (ORR) (time frame: Up to 15 years). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jun 2025.