E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Advanced lymphoid malignancies |
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E.1.1.1 | Medical condition in easily understood language |
Advanced lymphoid malignancies |
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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 | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10060390 |
E.1.2 | Term | Leukaemia lymphoblastic acute |
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 |
To evaluate the long-term safety of patients with advanced lymphoid malignancies who have been previously administered with UCART19. |
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E.2.2 | Secondary objectives of the trial |
- To assess the long-term anti leukemic activity (disease outcome, disease-free survival and overall survival), - To assess the proportion of patients who underwent allogeneic HSCT (Hematopoietic Stem Cell Transplantation), - To assess the time to transplant, - To monitor the long-term persistence of UCART19 in blood, - To monitor the long-term persistence in bone marrow (only in bone marrow samples performed as part of routine care and at the Investigators discretion) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Written informed consent obtained prior any study-specific procedure (patient or parent(s) or legal representative); - Patient dosed with UCART19 who completed or discontinued early from a sponsored or from any investigator-initiated study that tested UCART19, or patients who were administered UCART19 under a special access scheme (compassionate use); - Female patients of childbearing potential and male patients with partners of childbearing potential must continue to use an effective method of birth control as well as their partners. |
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E.4 | Principal exclusion criteria |
No exclusion criteria for this study |
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E.5 End points |
E.5.1 | Primary end point(s) |
The safety of UCART19 will be assessed throughout the study by evaluation of the following parameters: 1- Number, duration, outcome of all adverse events (AE), regardless the relationship with UCART19 2- Number, duration, outcome of AE of special interest regardless the relationship with UCART19 and any AE assessed as related to UCART19 3- For paediatric patients: the potential impact on growth curve and puberty will be assessed by height, weight, Tanner staging and menstrual cycle status |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1- within 12 months after allo-HSCT (if performed), or within 12 months after D84 post UCART19 infusion 2- throughout the study 3- Month 3 post last UCART19 infusion, Month 6 and Month 12, then every 6 months up to Year 3, and yearly until Year 15 |
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E.5.2 | Secondary end point(s) |
- Proportion of patients who relapse or progress among patients who had not relapsed (progression-free survival, disease specific survival) - Proportion of patients who underwent allogeneic HSCT - Time to transplant - Proportion of patients with detectable UCART19 levels in peripheral blood by qPCR - Proportion of patients with detectable UCART19 levels in bone marrow |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
M3 post last UCART19 infusion, M6 and M12, then every 6 months up to year 3, and yearly until year 15 |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
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) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
Long term follow-up study |
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E.7.2 | Therapeutic exploratory (Phase II) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
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 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
France |
United Kingdom |
United States |
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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.8.9.1 | In the Member State concerned years | 22 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 23 |
E.8.9.2 | In all countries concerned by the trial months | 7 |