E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Congenital adrenal hyperplasia (CAH) is a disorder of adrenal steroid
synthesis leading to cortisol deficiency and an increase in androgen
production.Treatment in children consists of hydrocortisone substitution
thereby also restoring the negative feedback on the pituitary gland and
consequently normalize androgen production. There is still no evidence
about the best timing of hydrocortisone use: The highest dosage of
hydrocortisone in the morning or the highest dosage at night |
Het adrenogenitaal syndroom (AGS) is een aangeboren ziekte van de
bijnier, die veroorzaakt wordt door een tekort van het enzym 21-
hydroxylase, dat nodig is voor de productie van hormonen in de bijnier.
Door een tekort aan dit enzym wordt onvoldoende cortisol
(stresshormoon) en tegelijkertijd een overmaat aan mannelijke
hormonen (androgenen) in de bijnier gemaakt. |
|
E.1.1.1 | Medical condition in easily understood language |
CAH is an inherited disease of the adrenal gland with ad deficiency of cortisol and aldosterone. Current treatment consists of lifelong replacement of
synthetic glucocorticoids. |
het adrenogenitaal syndroom is een ziekte van de bijnier met een tekort
aan hormonen cortisol en zouthormoon. behandling bstaat uit het
toedienen van deze hormonen |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To study the effects of 2 standard treatment timing strategies for glucocorticoid dosage on androgen concentration in CAH children: a. highest dosage in the morning, b. highest dosage in the evening. |
Wij willen graag evalueren welke van de 2 behandelopties leidt tot de
beste onderdrukking van bijnier androgenen. |
|
E.2.2 | Secondary objectives of the trial |
12 hours blood pressure and activity
and sleeping patterns during the 2 treatment strategies. |
12 uurs bloeddruk meting en slaappatroon |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Children with CAH due to 21-hydroxylase deficiency > 4 years old and
< 18 years old
• The diagnosis is confirmed by mutation analysis
• Patient are treated with hydrocortisone according to standard
guidelines in a thrice daily schedule
• Ability to collect saliva |
Kinderen met AGS tussen 4 - 18 jaar ouder
Diagnose bevestigd middele genetische analyse
Behandeling met hydrocortion
in staat zijn om speeksel te sparen |
|
E.4 | Principal exclusion criteria |
•Other forms of CAH
•Not able to collect saliva
•Chronical medication use other than hydrocortisone and
fludrocortisone |
andere vormen van AGS
niet in staat om speeksel te verzamelen
co medicatie anders dan hydrocortison en fludrocortison |
|
E.5 End points |
E.5.1 | Primary end point(s) |
17-hydroxyprogesterone and androstenedione levels measured in saliva
four times a day after 3 weeks of treatment with the 2 different timing
strategies. We will compare differences in levels of 17 OHP and A.
between the 2 dose schedules. |
17-hydroxyprogesterone and androstenedione concentraties gemeten in
speeksel na 3 weken behandeling in de 2 verschiillende dosis
verdelingen |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
at the end of each 3 week period |
aan het einde van elke 3 weken |
|
E.5.2 | Secondary end point(s) |
12 hour blood pressure and sleeping patterns during the 2 treatment
strategies. |
12 uurs bloeddruk meting en slaappatroon |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
daily sleeping score
RR once after every 3 week period |
daily sleepping score
Bloeddruk een keer per 3 weken |
|
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 | 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) | 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.8.3 |
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.8.5 | 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
|
|
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 | 6 |
E.8.9.1 | In the Member State concerned days | |