Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatment
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Conventional Glucocorticoids (immediate release hydrocortisone, cortisone acetate, prednisone, prednisolone, dexamethasone), Dual release hydrocortisone (plenadren).
- Who it may be relevant to
- Registry conditions: Congenital Adrenal Hyperplasia. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Congenital Adrenal Hyperplasia: Innovative Once Daily Dual Release Hydrocortisone Treatment
Overview
This is a controlled, open study designed to compare the effects of dual-release hydrocortisone preparations versus conventional glucocorticoid therapy on clinical, anthropometric parameters, metabolic syndrome, hormonal profile, bone status, quality of life, reproductive, sexual and psychological functions and treatment compliance in patients affected by congenital adrenal hyperplasia due to 21 OH deficiency.
Detailed description
Congenital adrenal hyperplasia due to 21-hydroxylase deficiency is an autosomal recessive disorder characterized by cortisol and in some cases aldosterone deficiency, associated with androgen excess. Treatment goals are to replace cortisol deficiency, to control androgen levels, while avoiding the adverse effects of exogenous glucocorticoids. A variety of glucocorticoid treatments have been used in an attempt to control the overnight increase in adrenal androgens. However, there is no consensus on the optimum management of congenital adrenal hyperplasia adults. Current evidence in patients with adrenal insufficiency suggests that the inability of current regimens to replace physiological circadian cortisol levels, leads to adverse clinical outcomes, including metabolic syndrome, insulin resistance, increased risk factors for cardiovascular diseases, bone and immune alterations, sleep disturbances and quality of life impairment. Moreover, the risk for poor treatment compliance, in case of multiple daily doses treatment regimens, should not be excluded. In this trial a dual-release hydrocortisone preparation, that been able to mimic the circadian pattern of circulating cortisol, was studied in patients with adrenal insufficiency due to congenital adrenal hyperplasia.
All patients with a diagnosis of congenital adrenal hyperplasia due to 21-hydroxylase deficiency, irrespective of glucocorticoid treatment, are eligible for the inclusion in the study and may be asked to participate in the study. Patients are followed during the course of routine clinical practice for the duration of time that the study is active.
ARM1: Conventional glucocorticoid therapy is continued as before entering the study
ARM2: Dual release hydrocortisone oral tablets is administered once-daily in the fasting state. The dose is kept the same as patients had before entering the trial.
Interventions
- Drug Conventional Glucocorticoids (immediate release hydrocortisone, cortisone acetate, prednisone, prednisolone, dexamethasone)
Treatment of congenital adrenal hyperplasia - Drug Dual release hydrocortisone (plenadren)
Treatment of congenital adrenal hyperplasia
Primary outcome measures
- Change from baseline in measurement of total and LDL cholesterol (mg/dl) [Time frame: 0, + 6 months, + 12 months, +24 months]
Secondary outcome measures (12)
- Change from baseline in measurement of glycaemia (mg/dl) [Time frame: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of BMI (Kg/m2) [Time frame: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of blood pressure (mmHg) [Time frame: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of insulinemia (μU/mL) [Time frame: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of triglycerides (mg/dl) [Time frame: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of HDL-cholesterol (mg/dl) [Time frame: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of Glycated Haemoglobin (%) [Time frame: 0, + 6 months, + 12 months, +24 months]
- Changes in bone mineral density [Time frame: 0, + 12 months, +24 months]
- Changes in quality of life [Time frame: 0, + 6 months, + 12 months, +24 months]
- Changes in sex function in males [Time frame: 0, + 6 months, + 12 months, +24 months]
- Changes in sex function in females [Time frame: 0, + 6 months, + 12 months, +24 months]
- Changes in depression status [Time frame: 0, + 6 months, + 12 months, +24 months]
Eligibility criteria
Inclusion criteria
- males and females aged >18 years;
- established diagnosis of adrenal insufficiency in congenital adrenal hyperplasia due to 21-hydroxylase deficiency;
- stably treated with conventional glucocorticoids, available to change their regimen according to random allocation
- written informed consent/assent to participate in the study in compliance with local regulations.
Exclusion criteria
- clinical or laboratory signs of severe cerebral, respiratory, hepatobiliary or pancreatic diseases, renal dysfunction, gastrointestinal emptying, or motility disturbances (i.e. chronic diarrhea), significant psychiatric illnesses;
- history of/or current alcohol and/or drug abuse;
- night shift workers;
- underlying diseases that could necessitate treatment with glucocorticoids;
- therapies with hepatic enzyme induction drugs interfering with glucocorticoid kinetics, or immunosuppressive steroid therapy;
- patients with a documented intolerance/known hypersensitivity to dual release hydrocortisone;
- vulnerable populations, such as elderly, cancer patients, pregnant and lactating women;
- history of non-compliance to medical regimens, or potentially unreliable patients
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Italy · 1 center
- Federico II University — Naples
Identifiers
NCT: NCT03760835 · 140/16