ovel mass spectrometric monitoring (steroid metabolomics) to detect treatment success and chronic over- or under-treatment in children with CAH (CAH metabolomics).
Completed
- Conditions
- adrenogenital syndromem congenital adrenal hyperplasia (CAH)1001469910001353
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 50
Inclusion Criteria
The subjects must have a genetically confirmed diagnosis of classical CAH (salt-losing or simple virilising) and
- group 1: being pre-pubertal and Tanner stage 1
or
- group 2: being late-pubertal and Tanner stage 3 plus or post-menarche
The subjects must personally provide, or have a legal guardian provide, written informed consent to participate in the trial, according to local regulations.
Exclusion Criteria
- Intercurrent illnesses at or within one week before visit.
- Poor understanding of language by subjects and/or legal guardians, leading to insufficient ability to understand the written study information form.
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint is therapy success, defined as no signs of over- or<br /><br>undertreatment plus absence of adrenal crisis which needs elevated hormonal<br /><br>replacement therapy (e.g. infection)</p><br>
- Secondary Outcome Measures
Name Time Method <p>The secondary endpoints are signs of over- or undertreatment, as defined in the<br /><br>study protocol table 1.</p><br>