E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute lymphoblastic leukemia |
acute lymfatische leukemie |
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E.1.1.1 | Medical condition in easily understood language |
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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 | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10001690 |
E.1.2 | Term | ALL |
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 reduce dexamethasone induced cerebral side-effects on mood, behaviour, and cognition by intervention treatment with physiological doses of cortisol compared to placebo. |
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E.2.2 | Secondary objectives of the trial |
I. To study if dexamethasone toxicity; e.g. side-effects like diabetes mellitus, insulin resistance, visceral fat gain, hypertension and hypercholesterolemia are less severe during intervention treatment compared to placebo.
II. To study the positive predictive value of novel in vivo diagnostic tests (consisting of salivary very low dose dexamethasone suppression test), on dexamethasone side-effects
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- written informed consent
- age 3-16
- histologically or cytologically confirmed acute lymphoblastic leukemia
- inclusion in DCOG ALL10 or ALL11 protocol or ALL R3 protocol
- able to comply with scheduled follow-up.
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E.4 | Principal exclusion criteria |
- patient or parent refusal
- anticipated compliance problems
- Underlying conditions which affect the absorption of oral medication
- pregnant or lactating patients
- current uncontrolled infection or any other complication which may interfere with dexamethasone treatment
- language barrier
- preexisting mental retardation syndrome
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary outcome parameter is the parent-reported strength and difficulty questionnaire in Dutch (SDQ-Dut) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
after 5 days of dexamethasone treatment with or without cortisol. |
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E.5.2 | Secondary end point(s) |
Secondary outcome parameters are mood and behaviour questionnaires and (neuro-)cognitive tests and metabolic parameters |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
after 5 days of dexamethasone treatment with or without cortisol |
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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 | No |
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 | 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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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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The end of the study is defined as the last patient’s last visit.
In case the study is ended prematurely, the sponsor will notify the accredited METC and the competent authority within 15 days, including the reasons for the premature termination.
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |