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 | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10060390 |
E.1.2 | Term | Leukaemia lymphoblastic acute |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10008958 |
E.1.2 | Term | Chronic lymphocytic leukaemia |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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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 (progression-free survival, disease specific 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), occurring within 12 months post last UCART19 infusion.
2. Number, duration, outcome of AEs of special interest regardless of the relationship with UCART19 and any AE assessed as related to UCART19 throughout the study.
3. Number of AEs leading to death up to the end of the study.
4. For paediatric patients: the potential impact on growth curve and puberty will be assessed by height, weight, Tanner staging and menstrual cycle status.
5. If an allo-HSCT is performed, engraftment will be assessed through the reporting of haematological data and chimerism status (if data available from the patient's medical file), up to 3 months post allo-HSCT. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. Within 12 months post last UCART19 infusion.
2. Throughout the study.
3. Up to the end of the study.
4. Month 3 post last UCART19 infusion, month 6 and month 12, then every 6 months up to year 3 and yearly until year 15.
5. Up to 3 months post allo-HSCT. |
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E.5.2 | Secondary end point(s) |
- Proportion of patients who relapse or progress (progression free survival, disease specific survival) - Duration of response from the date of the first CR or CRi until the date of progression or death due to any cause, whichever occurs first. - Overall survival (OS) - Proportion of patients who underwent allogeneic HSCT if not transplanted within the parent study and time to transplant
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Month 3 after UCART19 infusion and then at month 6 and month 12 during year 1, then every 6 months up to year 3, then yearly up to 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 | Yes |
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) | 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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 7 |
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 | 23 |
E.8.9.1 | In the Member State concerned months | 7 |
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 |