Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatment
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- В протоколе указаны: Conventional Glucocorticoids (immediate release hydrocortisone, cortisone acetate, prednisone, prednisolone, dexamethasone), Dual release hydrocortisone (plenadren).
- Кому может быть актуально
- Состояния в реестре: Congenital Adrenal Hyperplasia. Базовые параметры: от 18 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Италия
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
Congenital Adrenal Hyperplasia: Innovative Once Daily Dual Release Hydrocortisone Treatment
Обзор
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.
Подробное описание
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.
Вмешательства
- Препарат Conventional Glucocorticoids (immediate release hydrocortisone, cortisone acetate, prednisone, prednisolone, dexamethasone)
Treatment of congenital adrenal hyperplasia - Препарат Dual release hydrocortisone (plenadren)
Treatment of congenital adrenal hyperplasia
Первичные конечные точки
- Change from baseline in measurement of total and LDL cholesterol (mg/dl) [Срок оценки: 0, + 6 months, + 12 months, +24 months]
Вторичные конечные точки (12)
- Change from baseline in measurement of glycaemia (mg/dl) [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of BMI (Kg/m2) [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of blood pressure (mmHg) [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of insulinemia (μU/mL) [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of triglycerides (mg/dl) [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of HDL-cholesterol (mg/dl) [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Change from baseline in measurement of Glycated Haemoglobin (%) [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Changes in bone mineral density [Срок оценки: 0, + 12 months, +24 months]
- Changes in quality of life [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Changes in sex function in males [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Changes in sex function in females [Срок оценки: 0, + 6 months, + 12 months, +24 months]
- Changes in depression status [Срок оценки: 0, + 6 months, + 12 months, +24 months]
Критерии участия
Критерии включения
- 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.
Критерии исключения
- 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
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Распределение
- Рандомизированное
- Модель
- Параллельные группы
- Маскирование
- Открытое
- Основная цель
- Лечение
Центры проведения
Италия · 1 центр
- Federico II University — Naples
Идентификаторы
NCT: NCT03760835 · 140/16