E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Continued treatment with lyophilized pegaspargase in paediatrics patients with Acute Lymphoblastic Leukemia |
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E.1.1.1 | Medical condition in easily understood language |
Acute Lymphoblastic Leukemia (ALL) |
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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 |
To provide treatment with lyophilized S95014 in pediatric patients with ALL who completed the CL2-95014-002 study during the induction phase. |
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E.2.2 | Secondary objectives of the trial |
To assess the safety profile of lyophilized S95014 during the consolidation phase |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patient who completed the CL2-95014-002 study
- Patient currently receiving clinical benefit from previous treatment with S95014 as per investigator's judgment
- Signed informed consent and assent, when appropriate
- Highly effective contraception method |
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E.4 | Principal exclusion criteria |
- Unlikely to cooperate in the study
- Pregnant and lactating women
- Participant already enrolled in the study (informed consent signed)
- Prior surgery or bone marrow transplant related to the studied disease
- History of sensitivity to polyethylene glycol (PEG) or PEG-based drugs
- Psychiatric illness/social situation that would limit compliance with study requirements
- Group "E" and "T-HR" patients according to ALL-MB 2015 protocol classification
- Major safety issue due to previous S95014 administration (e.g. non recovery of safety parameters, serious hypersensitivity, serious pancreatitis, serious haemorrhage, serious thromboembolic event)
- Significant laboratory abnormality or uncontrolled intercurrent illness (e.g. life-threatening acute tumor lysis syndrome, symptomatic congestive heart failure, cardiac arrhythmia, severe or uncontrolled active acute infection) likely to jeopardize the patients' safety or to interfere with the conduct of the study, in the investigator's opinion |
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E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of Adverse Events (AEs) (safety and tolerability)All Adverse events (AEs) including Treatment Emergent Adverse Events (TEAEs) causality and severity based on NCI CTCAE 5.0 |
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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) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
To provide continued treatment with lyophilized pegaspargase |
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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 | No |
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 | Yes |
E.8.1.7.1 | Other trial design description |
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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 |
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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End of Trial is defined as the date of the last visit of the last participant
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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 months | 7 |