Trial · NCT03610724
Phase II Open Label Trial to Determine Safety & Efficacy of Tisagenlecleucel in Pediatric Non-Hodgkin Lymphoma Patients
NCT03610724, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2024-03-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Phase II Open Label Trial to Determine Safety & Efficacy of Tisagenlecleucel in Pediatric Non-Hodgkin Lymphoma Patients. Trials, record NCT03610724. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03610724
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase II Open Label Trial to Determine Safety & Efficacy of Tisagenlecleucel in Pediatric Non-Hodgkin Lymphoma Patients
- NCT number
- NCT03610724
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 34 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Non-Hodgkin Lymphoma
- Interventions
- Tisagenlecleucel, lymphodepleting chemotherapy, Bridging Therapy
- First posted
- 2018-08-01
- Last updated
- 2024-06-20
- Other registries' IDs
- eudract:2017-005019-15
- Results first posted
- 2024-03-19
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Response Rate (ORR) as Determined by Local Investigator | 6 months post-tisagenlecleucel infusion | The overall response rate (ORR) is defined as the percentage of subjects with a best overall disease response of complete response (CR) or partial response (PR), where the best overall disease response is defined as the best disease response recorded from tisagenlecleucel infusion until progressive disease or start of new anticancer therapy, whichever comes first. |
| Secondary | Duration of Response (DOR) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | Duration of response (DOR) is defined as the time from the date of first documented disease response (CR or PR) as determined by local investigator assessments to the date of first documented progression or death due to underlying cancer. |
| Secondary | Event Free Survival (EFS) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | Event free survival (EFS) is defined as the time from date of first tisagenlecleucel infusion to the earliest date of death from any cause, disease progression as determined by local investigator assessments, or starting new anticancer therapy for underlying cancer, excluding hematopoietic stem cell transplant (HSCT). |
| Secondary | Relapse Free Survival (RFS) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | Relapse free survival (RFS) is defined as the time from the date of first documented disease response (CR or PR) as determined by local investigator assessments to the date of first documented disease progression or death due to any cause. |
| Secondary | Progression Free Survival (PFS) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | Progression free survival (PFS) is defined as the time from the date of first tisagenlecleucel infusion to the date of first documented disease progression as determined by local investigator assessments or death due to any cause. Progression is defined using the International non-Hodgkin Lymphoma Response Criteria. For a PET-based response, progressive disease is defined as a 4 or 5 on the 5 point scale with increased uptake compared to the nadir or new FDG-avid foci consistent with lymphoma. For a CT/MRI based response progressive disease is defined as a 25% increase in the SPD (sum of the products of two largest perpendicular diameters) of index lesions, or unequivocal progression in either non-index lesions or the spleen. Any new disease attributable to lymphoma would also constitute progressive disease. |
| Secondary | Overall Survival (OS) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | Overall survival (OS) is defined as the time from date of first tisagenlecleucel infusion to the date of death due to any cause. |
| Secondary | Cellular Kinetics Parameter: Cmax | Post-infusion day 4, day 7, day 11, day 14, day 21, day 28, month 3, month 6, month 9, month 12, month 18, month 24, and then annually until Month 48 | The maximum (peak) transgene level (copies/μg) observed in peripheral blood or other body fluid after single dose administration as measured by qPCR. Pharmacokinetics of tisagenlecleucel were based on chimeric antigen receptor (CAR) transgene levels in peripheral blood as detected by qPCR, unless otherwise noted. Pharmacokinetics of tisagenlecleucel were based on chimeric antigen receptor (CAR) transgene levels in peripheral blood as detected by qPCR, unless otherwise noted. |
| Secondary | Cellular Kinetics Parameter: Tmax | Post-infusion day 4, day 7, day 11, day 14, day 21, day 28, month 3, month 6, month 9, month 12, month 18, month 24, and then annually until Month 48 | The time to reach maximum (peak) transgene level (days) in peripheral blood or other body fluid after single dose administration. Pharmacokinetics of tisagenlecleucel were based on chimeric antigen receptor (CAR) transgene levels in peripheral blood as detected by qPCR, unless otherwise noted. |
| Secondary | Cellular Kinetics Parameter: AUC0-28d | 0 to 28 days | Area Under the Concentration-time Curve (AUCs) from the time course of transgene levels in peripheral blood following tisagenlecleucel infusion (days\*copies/μg), from day of infusion to day 28. D28 refers to the timepoint for definition of responder populations. Pharmacokinetics of tisagenlecleucel were based on chimeric antigen receptor (CAR) transgene levels in peripheral blood as detected by qPCR, unless otherwise noted. |
| Secondary | Cellular Kinetics Parameter: Clast | Post-infusion day 4, day 7, day 11, day 14, day 21, day 28, month 3, month 6, month 9, month 12, month 18, month 24, and then annually until Month 48 | The last observed quantifiable transgene level in peripheral blood. Pharmacokinetics of tisagenlecleucel were based on chimeric antigen receptor (CAR) transgene levels in peripheral blood as detected by qPCR, unless otherwise noted. |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Response Rate (ORR) as Determined by Local Investigator | 6 months post-tisagenlecleucel infusion | Efficacy Analysis Set (EAS): all subjects with aggressive r/r B-cell NHL who received an infusion of tisagenlecleucel and had measurable disease at baseline, i.e participants with complete or unknown response after bridging therapy were excluded from the efficacy analysis set. | Percentage of participants | NUMBER |
| SECONDARY | Duration of Response (DOR) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | EAS: all subjects with aggressive r/r B-cell NHL who received an infusion of tisagenlecleucel and had measurable disease at baseline. Only participants with best response of CR/PR post-infusion were included in the DOR analysis. | months | MEDIAN |
| SECONDARY | Event Free Survival (EFS) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | EAS: all subjects with aggressive r/r B-cell NHL who received an infusion of tisagenlecleucel and had measurable disease at baseline, i.e participants with complete or unknown response after bridging therapy were excluded from the efficacy analysis set. | months | MEDIAN |
| SECONDARY | Relapse Free Survival (RFS) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | EAS: all subjects with aggressive r/r B-cell NHL who received an infusion of tisagenlecleucel and had measurable disease at baseline. Only participants with best response of CR/PR post-infusion were included in the RFS analysis. | months | MEDIAN |
| SECONDARY | Progression Free Survival (PFS) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | EAS: all subjects with aggressive r/r B-cell NHL who received an infusion of tisagenlecleucel and had measurable disease at baseline, i.e participants with complete or unknown response after bridging therapy were excluded from the efficacy analysis set. | months | MEDIAN |
| SECONDARY | Overall Survival (OS) | Post-infusion Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, Month 24, and then annually until Month 48 | EAS: all subjects with aggressive r/r B-cell NHL who received an infusion of tisagenlecleucel and had measurable disease at baseline, i.e participants with complete or unknown response after bridging therapy were excluded from the efficacy analysis set. | months | MEDIAN |
| SECONDARY | Cellular Kinetics Parameter: Cmax | Post-infusion day 4, day 7, day 11, day 14, day 21, day 28, month 3, month 6, month 9, month 12, month 18, month 24, and then annually until Month 48 | Cellular kinetic analysis set (CKAS): subjects in the FAS who provided at least 1 valid cellular kinetic concentration for tisagenlecleucel. Certain PK parameters were not estimable for all subjects due to insufficient samples. | copies/μg | GEOMETRIC_MEAN |
| SECONDARY | Cellular Kinetics Parameter: Tmax | Post-infusion day 4, day 7, day 11, day 14, day 21, day 28, month 3, month 6, month 9, month 12, month 18, month 24, and then annually until Month 48 | CKAS: subjects in the FAS who provided at least 1 valid cellular kinetic concentration for tisagenlecleucel. Certain PK parameters were not estimable for all subjects due to insufficient samples. | days | MEDIAN |
| SECONDARY | Cellular Kinetics Parameter: AUC0-28d | 0 to 28 days | CKAS: subjects in the FAS who provided at least 1 valid cellular kinetic concentration for tisagenlecleucel. Certain PK parameters were not estimable for all subjects due to insufficient samples. | copies/μg*days | GEOMETRIC_MEAN |
| SECONDARY | Cellular Kinetics Parameter: Clast | Post-infusion day 4, day 7, day 11, day 14, day 21, day 28, month 3, month 6, month 9, month 12, month 18, month 24, and then annually until Month 48 | CKAS: subjects in the FAS who provided at least 1 valid cellular kinetic concentration for tisagenlecleucel. Certain PK parameters were not estimable for all subjects due to insufficient samples. | copies/μg | GEOMETRIC_MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Response Rate (ORR) as Determined by Local Investigator | Tisagenlecleucel | 32.1 | 95% Confidence Interval 15.9 to 52.4 | Percentage of participants | |
| Duration of Response (DOR) | Tisagenlecleucel | NA | 95% Confidence Interval 1.0 to NA | months | |
| Event Free Survival (EFS) | Tisagenlecleucel | 2.1 | 95% Confidence Interval 1.1 to 2.8 | months | |
| Relapse Free Survival (RFS) | Tisagenlecleucel | NA | 95% Confidence Interval 1.0 to NA | months | |
| Progression Free Survival (PFS) | Tisagenlecleucel | 2.5 | 95% Confidence Interval 1.1 to 2.9 | months | |
| Overall Survival (OS) | Tisagenlecleucel | 10.4 | 95% Confidence Interval 3.4 to NA | months | |
| Cellular Kinetics Parameter: Cmax | Tisagenlecleucel | 5140 | Geometric Coefficient of Variation 238.9 | copies/μg | |
| Cellular Kinetics Parameter: Tmax | Tisagenlecleucel | 12.7 | Full Range 1.90 to 21.9 | days | |
| Cellular Kinetics Parameter: AUC0-28d | Tisagenlecleucel | 53500 | Geometric Coefficient of Variation 154.9 | copies/μg*days | |
| Cellular Kinetics Parameter: Clast | Tisagenlecleucel | 344 | Geometric Coefficient of Variation 350.4 | copies/μg |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow7
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Tisagenlecleucel | 34 | |
| Overall Study | Met Eligibilty Criteria and Were Infused | Tisagenlecleucel | 33 | |
| Overall Study | Met Eligibilty Criteria But Was Not Infused* | Tisagenlecleucel | 1 | |
| Overall Study | Completed Treatment & Primary Follow-up Phase | Tisagenlecleucel | 14 | |
| Overall Study | Discont. Treatment & Primary Follow-up Phase | Tisagenlecleucel | 19 | |
| Overall Study | COMPLETED | Tisagenlecleucel | 14 | |
| Overall Study | NOT COMPLETED | Tisagenlecleucel | 20 |
Source ClinicalTrials.gov
Adverse events226
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Tisagenlecleucel - Post-infusion | 11 | 33 | |
| other | Anaemia | Blood and lymphatic system disorders | Tisagenlecleucel - Pre-infusion Deaths | 0 | ||
| other | Bone marrow failure | Blood and lymphatic system disorders | Tisagenlecleucel - Post-infusion | 2 | 33 | |
| other | Bone marrow failure | Blood and lymphatic system disorders | Tisagenlecleucel - Pre-infusion Deaths | 0 | ||
| other | Coagulopathy | Blood and lymphatic system disorders | Tisagenlecleucel - Post-infusion | 2 | 33 | |
| other | Coagulopathy | Blood and lymphatic system disorders | Tisagenlecleucel - Pre-infusion Deaths | 0 | ||
| other | Febrile neutropenia | Blood and lymphatic system disorders | Tisagenlecleucel - Post-infusion | 2 | 33 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Tisagenlecleucel - Pre-infusion Deaths | 0 | ||
| other | Leukopenia | Blood and lymphatic system disorders | Tisagenlecleucel - Post-infusion | 5 | 33 | |
| other | Leukopenia | Blood and lymphatic system disorders | Tisagenlecleucel - Pre-infusion Deaths | 0 |
1 to 10 of 226Source ClinicalTrials.gov
See all 226 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Tisagenlecleucel - Post-infusion | 17 | 33 |
| Total, all-cause mortality | Tisagenlecleucel - Pre-infusion Deaths | 1 | 34 |
| Total, other adverse events | Tisagenlecleucel - Post-infusion | 33 | 33 |
| Total, other adverse events | Tisagenlecleucel - Pre-infusion Deaths | 0 | 0 |
| Total, serious adverse events | Tisagenlecleucel - Post-infusion | 24 | 33 |
| Total, serious adverse events | Tisagenlecleucel - Pre-infusion Deaths | 0 | 0 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34515338 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Tisagenlecleucel - Post-infusion vs Tisagenlecleucel - Pre-infusion Deaths: deaths from any cause |
Source Trial comparisons
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03610724
GET https://conductscience.org/api/trials/v1/NCT03610724/events?page=2