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Terminated due to slow enrollment
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This was a multi-center Phase II study investigating the efficacy and safety of reinfusion of tisagenlecleucel in pediatric and young adult patients with acute lymphoblastic leukemia (ALL) who were treated with tisagenlecleucel and experience B cell recovery.
This trial was a phase II, open label, multi-center trial to determine the efficacy and safety of tisagenlecleucel re-infusion in pediatric and adolescent young adult (AYA) patients with acute lymphoblastic leukemia (ALL) experiencing loss of B cell aplasia. Loss of B-cell aplasia is defined as: peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes. B-cell aplasia is defined as PB absolute B lymphocyte count <50/µL.
The study had the following phases for all patients: Screening, Treatment and Follow-up. The total duration of the study was about 12 months. After tisagenlecleucel re-infusion, efficacy was assessed at months 1, 3, 6, and End of Study at which time blood samples were obtained.
The study stopped early due to slow enrollment into the trial. The rate of enrollment made the trial no longer feasible to continue.
The patients were able to voluntarily withdraw from the study for any reason, at any time. Patients who received commercial tisagenlecleucel had to be followed for up to 15 years post-infusion. Patients could have been followed under the Center for International Blood and Marrow Transplant Research (CIBMTR) cellular therapy registry if consented for participation. For patients who do not provide consent for participation in the Center for International Blood and Marrow Transplant Research (CIBMTR) registry, adverse events were to be reported for 15 years or until the patient enrolls in the registry.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Tisagenlecleucel | Experimental | Tisagenlecleucel Cell Dispersion for Infusion given once during the study. The approved dose range for tisagenlecleucel is: 0.2 to 5.0×106 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×108 CAR-positive viable T cells for patients > 50 kg body weight. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Tisagenlecleucel | Biological | Tisagenlecleucel Cell Dispersion for Infusion given once during the study. The approved dose range for tisagenlecleucel is: 0.2 to 5.0×106 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×108 CAR-positive viable T cells for patients > 50 kg body weight. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Patients Who Establish B Cell Aplasia Within 9 Months of Reinfusion | Percentage of patients who establish B-cell aplasia at any visit following re-infusion with tisagenlecleucel. B-cell aplasia is defined as peripheral blood (PB) absolute B lymphocyte count <50/μL. Planned timeframe was 12 months but actual timeframe was approximately 9 months due to early termination of the trial. Day 1 is post lymphodepleting chemotherapy and pre-reinfusion of tisagenlecleucel. | Post-reinfusion up to 9 months (Day 1 is excluded) |
| Measure | Description | Time Frame |
|---|---|---|
| Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day | Overall remission rate (ORR) was defined as the percentage of participants with a best overall disease response of complete remission (CR) or CR with incomplete blood count recovery (CRi). However, the rate was not calculated due to low enrollment. Participants' best responses have been listed by day and participants may be counted more than once. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Childrens Hospital Los Angeles Divisionof Hematology/Oncology | Los Angeles | California | 90027 | United States | ||
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| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on novctrd.com | View source |
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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.
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There were 7 participants screened.
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| ID | Title | Description |
|---|---|---|
| FG000 | Tisagenlecleucel | Tisagenlecleucel Cell Dispersion for Infusion given once during the study. The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10^8 CAR-positive viable T cells for patients > 50 kg body weight. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 23, 2020 | Apr 14, 2022 |
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| Post-reinfusion up to 9 months |
| Participants With an Event | An event was defined as one of the following: death from any cause after remission, relapse, treatment failure (defined as no response in the study or discontinuation from the study for death, adverse event, lack of efficacy or progressive disease or new cancer therapy). | Reinfusion up to 9 months |
| Overall Survival (OS) | Deaths due to any reason. | Reinfusion up to 9 months |
| Ann and Robert H Lurie Childrens Hospital of Chicago |
| Chicago |
| Illinois |
| 60611 |
| United States |
| Children s Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| UT Southwestern Medical Center | Dallas | Texas | 75235 | United States |
| Full Analysis, Safety and Enrolled Sets |
|
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Tisagenlecleucel | Tisagenlecleucel Cell Dispersion for Infusion given once during the study. The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10^8 CAR-positive viable T cells for patients > 50 kg body weight. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
| ||||||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Patients Who Establish B Cell Aplasia Within 9 Months of Reinfusion | Percentage of patients who establish B-cell aplasia at any visit following re-infusion with tisagenlecleucel. B-cell aplasia is defined as peripheral blood (PB) absolute B lymphocyte count <50/μL. Planned timeframe was 12 months but actual timeframe was approximately 9 months due to early termination of the trial. Day 1 is post lymphodepleting chemotherapy and pre-reinfusion of tisagenlecleucel. | Full analysis set | Posted | Count of Participants | Participants | Post-reinfusion up to 9 months (Day 1 is excluded) |
|
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| ||||||||||||||||||||||||||
| Secondary | Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day | Overall remission rate (ORR) was defined as the percentage of participants with a best overall disease response of complete remission (CR) or CR with incomplete blood count recovery (CRi). However, the rate was not calculated due to low enrollment. Participants' best responses have been listed by day and participants may be counted more than once. | Full analysis set - Data was not adequate to perform overall remission rate analysis considering evaluable population size | Posted | Number | responses | Post-reinfusion up to 9 months |
|
| |||||||||||||||||||||||||||
| Secondary | Participants With an Event | An event was defined as one of the following: death from any cause after remission, relapse, treatment failure (defined as no response in the study or discontinuation from the study for death, adverse event, lack of efficacy or progressive disease or new cancer therapy). | Full analysis set - Data was not adequate to perform time to event analysis considering evaluable population size. | Posted | Number | participant | Reinfusion up to 9 months |
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| Secondary | Overall Survival (OS) | Deaths due to any reason. | Full analysis set - Data was not adequate to perform overall survival analysis considering evaluable population size | Posted | Number | deaths | Reinfusion up to 9 months |
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Adverse events were reported from first dose of study treatment until end of study for a duration of 274 days. .
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Tisagenlecleucel | Tisagenlecleucel Cell Dispersion for Infusion given once during the study. The approved dose range for tisagenlecleucel is: 0.2 to 5.0×10^6 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×10^8 CAR-positive viable T cells for patients > 50 kg body weight. | 0 | 5 | 3 | 5 | 4 | 5 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Cytokine release syndrome | Immune system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
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| Paraesthesia | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (24.1) | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA (24.1) | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Dental caries | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Stomatitis | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Tooth development disorder | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Catheter site erythema | General disorders | MedDRA (24.1) | Systematic Assessment |
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| Chills | General disorders | MedDRA (24.1) | Systematic Assessment |
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| Fatigue | General disorders | MedDRA (24.1) | Systematic Assessment |
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| Pain | General disorders | MedDRA (24.1) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (24.1) | Systematic Assessment |
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| Conjunctivitis | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
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| HCoV-OC43 infection | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
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| Paronychia | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
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| Polyomavirus viraemia | Infections and infestations | MedDRA (24.1) | Systematic Assessment |
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| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA (24.1) | Systematic Assessment |
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| Enterovirus test positive | Investigations | MedDRA (24.1) | Systematic Assessment |
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| Human rhinovirus test positive | Investigations | MedDRA (24.1) | Systematic Assessment |
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| Neutrophil count decreased | Investigations | MedDRA (24.1) | Systematic Assessment |
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| Platelet count decreased | Investigations | MedDRA (24.1) | Systematic Assessment |
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| Respirovirus test positive | Investigations | MedDRA (24.1) | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA (24.1) | Systematic Assessment |
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| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA (24.1) | Systematic Assessment |
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| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (24.1) | Systematic Assessment |
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| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA (24.1) | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (24.1) | Systematic Assessment |
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| Cancer fatigue | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (24.1) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (24.1) | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA (24.1) | Systematic Assessment |
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| Dermatitis acneiform | Skin and subcutaneous tissue disorders | MedDRA (24.1) | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (24.1) | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA (24.1) | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA (24.1) | Systematic Assessment |
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The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | + 1 862 778 8300 | Novartis.email@Novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 29, 2021 | Apr 14, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D002051 | Burkitt Lymphoma |
| D007938 | Leukemia |
| ID | Term |
|---|---|
| D007945 | Leukemia, Lymphoid |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D020031 | Epstein-Barr Virus Infections |
| D006566 | Herpesviridae Infections |
| D004266 | DNA Virus Infections |
| D014777 | Virus Diseases |
| D007239 | Infections |
| D014412 | Tumor Virus Infections |
| D016393 | Lymphoma, B-Cell |
| D008228 | Lymphoma, Non-Hodgkin |
| D008223 | Lymphoma |
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| ID | Term |
|---|---|
| C000626284 | tisagenlecleucel |
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