E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute lymphoblastic leukemia |
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E.1.1.1 | Medical condition in easily understood language |
Acute lymphoblastic leukemia
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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 | 16.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10000846 |
E.1.2 | Term | Acute 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 |
Participants achieving an Objective Response Rate |
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E.2.2 | Secondary objectives of the trial |
Response duration
Progression Free Survival
Minimal residual disease
Safety
Pharmacokinetics
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1/ Previously treated Acute Lymphoblastic Leukemia of B cell origin (including Burkitt's lymphoma) in relapse or primary refractory. Patients in first relapse will be eligible regardless of the first remission duration.
2/ No more than 3 prior salvage therapies.
3/ Philapdelphia positive patients failing treatment with imatinib mesylate are accepted.
4/ CD19 positive patients. |
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E.4 | Principal exclusion criteria |
E 01. Age <16 years
E 02. ECOG performance status >2 |
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E.5 End points |
E.5.1 | Primary end point(s) |
Number of participants achieving an Objective Response Rate |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Number of participants with Adverse Event
Assessment of PK parameter - maximum concentration (Cmax)
Assessment of PK parameter - area under curve (AUC)
Assessment of PK parameter - half-life (T1/2)
Assessment of PK parameter - clearance
Assessment of PK parameter - volume in steady state (Vss)
Minimal residual disease |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Number of participants with Adverse Event: Up to 1 year
Assessment of PK parameter - maximum concentration (Cmax): Up to 8 months
Assessment of PK parameter - area under curve (AUC): Up to 8 months
Assessment of PK parameter - half-life (T1/2): Up to 8 months
Assessment of PK parameter - clearance: Up to 8 months
Assessment of PK parameter - volume in steady state (Vss): Up to 8 months
Minimal residual disease: 4 to 8 weeks |
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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 | 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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 4 |
E.8.5 | The trial involves multiple Member States | No |
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 |
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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 | 1 |
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 | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |