E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Paediatric patients with B-cell acute lymphoblastic leukaemia |
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E.1.1.1 | Medical condition in easily understood language |
B-cell acute lymphoblastic leukaemia |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000845 |
E.1.2 | Term | Acute lymphoblastic leukemia |
E.1.2 | System Organ Class | 100000004864 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Evaluate the incidence of B cell aplasia after reinfusion of tisagenlecleucel |
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E.2.2 | Secondary objectives of the trial |
- Evaluate the efficacy of reinfusion of tisagenlecleucel for loss of B cell aplasia as measured by overall remission rate (ORR) 12 months after tisagenlecleucel reinfusion, which includes CR and CR with incomplete blood count recovery (CRi) as determined by investigator assessment for pALL patients - Evaluate event free survival (EFS), Overall survival (OS) and the safety of reinfusion of tisagenlecleucel for B cell recovery or MRD. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Signed informed consent must be obtained prior to participation in the study - Must have an additional dose of tisagenlecleucel available and prescribed by a physician in the course of medical practice - Age up to and including 25 years - Patients must have CD-19+ Leukemia - Patients who were previously treated with tisagenlecleucel and present with evidence of B-cell recovery as defined by: Peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes Other protocol-defined Inclusion/Exclusion may apply. |
|
E.4 | Principal exclusion criteria |
-Prior gene therapy other than tisagenlecleucel - Prior adoptive T cell therapy other than tisagenlecleucel - Active CNS involvement by malignancy - Active or latent hepatitis B or active hepatitis C, or any uncontrolled infection at screening - HIV positive test within 8 weeks of screening Other protocol-defined Inclusion/Exclusion may apply. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Percentage of patients who establish B cell aplasia within 12 months of reinfusion, as measured by circulating B lymphocytes (< 50/µL) and presence of CTL019 cells by qPCR in the peripheral blood |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
reinfusion up to 12 months |
|
E.5.2 | Secondary end point(s) |
- Percentage of patients with overall remission rate (ORR= CR + CRi) per Investigator assessment -Event free survival (EFS) - Overall Survival (OS) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
reinfusion up to 12 months |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 2 |