COrticosteroid in Congenital Adrenal Hyperplasia

Congenital Adrenal Hyperplasia Clinical Trial

— COCA  

Official title:

Comparative Study of the Use of Glucocorticoids in the Treatment of Congenital Adrenal Hyperplasia in Its Classical Form

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02552251
• Other study ID #: 11-059
• Status: Recruiting
• Phase: Phase 2/Phase 3
• First received: July 27, 2015
• Last updated: September 15, 2015
• Start date: August 2012

Study information

• Verified date: September 2015
• Source: University Hospital, Caen
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Agence Nationale de Sécurité du Médicament et des produits de santé
• Study type: Interventional

Clinical Trial Summary

Congenital adrenal hyperplasia (CAH) results from a deficiency of a key enzyme in the biosynthesis of cortisol, mainly 21-hydroxylase, resulting in its classic form a neonatal salt loss syndrome and / or a virilization syndrome in girls. The treatment of the disorder in adulthood involves administering steroidal compounds with the aim to substitute the gluco- and mineralocorticoid deficit on the one hand, and effectively curb the adrenal hyperplasia and adrenal androgen pathway in girls . The terms of glucocorticoid treatment are not clearly codified and are based on several steroidal compounds and various protocols. The advantages in terms of adrenal suppression and disadvantages - including bone and metabolic - different treatments have not been clearly established in the literature. The main objective of this study is to compare among adults with HCS in its classical form the impact on hormonal parameters adrenal suppression glucocorticoid of 3 types of treatment administered to equivalent dose and according to the usual procedures. The secondary objective is to compare in the same patients the impact of different drugs and treatments on several metabolic bone parameters. The study will include 40 adult patients bearing a HCS in its classical form and will include 3 treatment sequences of eight weeks each, during which they will be administered sequentially in random order at random and according to the known equivalences hydrocortisone, prednisone (CORTANCYL) and dexamethasone (DECTANCYL).

Randomization will be stratified based on previous DMARDs in the investigation that may be different for different patients, knowing that France hydrocortisone and dexamethasone are used mainly for the treatment of congenital adrenal hyperplasia. The judging criteria will be: i) the criteria of adrenal hormone suppression: plasma levels of testosterone, androstenedione, 17 OHP, ACTH and diurnal variations of the 17 OH progesterone salivary ii) the criteria of the metabolic impact of glucocorticoids: plasma glucose levels , blood lipids, and insulin sensitivity index HOMA-R calculated from glucose and insulin, iii) the criteria of bone impact of glucocorticoids: plasma for CTX bone resorption and bone alkaline phosphatase P1NP for bone formation iv) the living quality criteria evaluated by the PGWB Questionnaire (Psychological General Well-Being). The duration of the study period will be 24 months.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years to 55 Years

Eligibility

Inclusion Criteria:

• Pubescent women over 18 in genital activity (premenopausal)

• Suffering from congenital adrenal hyperplasia in its classical form with salt loss or pure virilizing

• Patients who have presented signs of congenital adrenal hyperplasia in its classical form (salt wasting syndrome and / or neonatal masculinization) with elevation of 17 OH progesterone with diagnosis of enzyme block 21 hydroxylase.

• Patients currently treated by: 1 or 2 Oral compound glucocorticoid as replacement and suppressive therapy + 1 mineralocorticoid if necessary with effective control of substitution + possibly by estrogen-progestin pill.

Exclusion Criteria:

• Liver disease, kidney, bone, diabetes, severe dyslipidemia, pregnancy

• Postmenopausal women, age over 55 years

• Concomitant therapy: glucocorticoids supra-physiological doses for other indications, bisphosphonates, vitamin D, oral antidiabetic agents or insulin, lipid lowering agents (eg inflammatory disease, asthma, systemic disease ... ..).

• participation of the subject to another biomedical research protocol for this study

• Inability to submit to medical monitoring study for geographical, social or psychological.

• Severe calorie diet planned or carried out during the study.

Study Design

Allocation: Randomized, Endpoint Classification: Pharmacokinetics Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Basic Science

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Biological:
• Hormonal balance measurements

• metabolic balance measurements

• bone balance measurements

Behavioral:
• quality of life assessment

Locations

Country	Name	City	State
France	Service Endocrinologie et Maladies Métaboliques	Caen

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Caen

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	hormonal parameters	6 points salivary 17 OHP cycle, and 8 am plasma ACTH,testosterone and androstenedione	change over baseline, week 8, week 16, week 24	No
Secondary	parameters of bone turnover:	CTX and bone alkaline phosphatase P1NP	change over baseline, week 8, week 16, week 24	No
Secondary	metabolic parameters:	blood glucose and insulin, cholesterol, triglycerides, HDL-cholesterol, LDL-cholesterol	change over baseline, week 8, week 16, week 24	No
Secondary	Quality of Life	Psychological General Well-Being questionnaire	change over baseline, week 8, week 16, week 24	No