E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Adrenal recovery following long and short term steroid treatment. |
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E.1.1.1 | Medical condition in easily understood language |
Timing of the recovery of the body and its ability to cope with stress following short and long term steroid treatment. |
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E.1.1.2 | Therapeutic area | Body processes [G] - Physiological processes [G07] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066550 |
E.1.2 | Term | Chronic arthritis |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000842 |
E.1.2 | Term | Acute lymphatic leukaemia |
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 | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10008953 |
E.1.2 | Term | Chronic liver disease |
E.1.2 | System Organ Class | 10019805 - Hepatobiliary disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 |
The primary research question is to compare how quickly the adrenal glands take to recover following short term (4 weeks) or long term (more than 2 months) steroid treatment. |
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E.2.2 | Secondary objectives of the trial |
The secondary research question in the long term steroid users is whether there is a difference in adrenal recovery time in children on two different types of steroid medications. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
The inclusion criteria for the two categories include.
Short-term Glucocorticosteroid tratment group: Patients with newly diagnosed Acute Lymoblastic Leukemia (above 1 year of age) presenting to Birmingham Children’s Hospital, receiving high dose dexamethasone for <8 weeks. Long-term Glucocorticosteroid tratment group: Patients between 1-17 years of age with chronic lung, liver or rheumatological disorders requiring long-term (>8 weeks) Glucocorticosteroid (GC) therapy
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E.4 | Principal exclusion criteria |
Exclusion criteria for short and long term GC groups include, GC use for any other underlying conditions including use of topical or inhaled steroids.
Unwilling, or unable to give informed consent
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is adrenal recovery, as defined by the time taken for the adrenals to recover (peak 30min cortisol >550nmol/L) following short or long term corticosteroid therapy, i.e from the last GC dose to the first normal ACTH test.
For comparison of categorical data between the short- and long-term GC group, and between the two GC weaning groups, Chi-square and Fisher’s exact tests are used, giving binomial confidence intervals for % recovering at each time point. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Time points after last GC dose: day 7±1, day 14±1, day 21±1 and day 35±1. First normal synacthen test ends the study participation, i.e. if synacthen test is normal already on day 7±1, no further testing at other time points. Adrenal recovery is defined as the time taken for the adrenals to recover (peak 30min cortisol >550nmol/L) following short or long term corticosteroid therapy, i.e from the last GC dose to the first normal ACTH test. |
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E.5.2 | Secondary end point(s) |
A self-administered questionnaire to be completed by the patient or parent on each visit, evaluating the symptoms and signs of steroid withdrawal syndrome |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Frequency of signs/symptoms of the steroid withdrawal syndrome and the peak cortisol in nmol/l at which this occurs at day 7±1, day 14±1, day 21±1 and day 35±1 after last GC dose.
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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 | 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 | Yes |
E.6.13.1 | Other scope of the trial description |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
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) | Yes |
E.8.2.2 | Placebo | No |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |