Congenital Adrenal HyperplasiaPhase 2/3COCANCT02552251
COrticosteroid in Congenital Adrenal Hyperplasia
A Phase 2/3 study testing Hormonal balance measurements, metabolic balance measurements and bone balance measurements in people with Congenital Adrenal Hyperplasia, run by University Hospital, Caen at 1 site in France.
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. Read more about this study
- Who is it for?
- Ages 18 to 55 years, women only
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- What is being tested?
- Hormonal balance measurements, metabolic balance measurements and bone balance measurements
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteFrance
- Study dates
- Aug 2012to Dec 2015
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Service Endocrinologie et Maladies Métaboliques
Caen, 14000
Yves REZNIK, MD, PhD, 02.31.06.45.86, reznik-y@chu-caen.fr
About this study
Official title: Comparative Study of the Use of Glucocorticoids in the Treatment of Congenital Adrenal Hyperplasia in Its Classical Form
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.
- Conditions
- Congenital Adrenal Hyperplasia
- Being tested
- Hormonal balance measurements, metabolic balance measurements, bone balance measurements, quality of life assessment
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hormonal balance measurementsBiological
metabolic balance measurementsBiological
bone balance measurementsBiological
quality of life assessmentBehavioural
| Study group | Role | What they receive |
|---|---|---|
| A: hydrocortisone | Experimental | hydrocortisone equivalent to physiological doses for each patient Strategy: administration of glucocorticoids during sequences of eight weeks Receives: Biological: Hormonal balance measurements; Biological: metabolic balance measurements; Biological: bone balance measurements; Behavioral: quality of life assessment |
| B :dexamethasone (DECTANCYL) | Experimental | dexamethasone equivalent to physiological doses for each patient Strategy: administration of glucocorticoids during sequences of eight weeks Receives: Biological: Hormonal balance measurements; Biological: metabolic balance measurements; Biological: bone balance measurements; Behavioral: quality of life assessment |
| C : prednisone (CORTANCYL) | Experimental | prednisone equivalent to physiological doses for each patient Strategy: administration of glucocorticoids during sequences of eight weeks Receives: Biological: Hormonal balance measurements; Biological: metabolic balance measurements; Biological: bone balance measurements; Behavioral: quality of life assessment |
What is being measured
The primary outcome is the main question the study is designed to answer.
hormonal parameters
6 points salivary 17 OHP cycle, and 8 am plasma ACTH,testosterone and androstenedione
Measured: change over baseline, week 8, week 16, week 24
Secondary outcomes (3)
parameters of bone turnover:
CTX and bone alkaline phosphatase P1NP
Measured: change over baseline, week 8, week 16, week 24
metabolic parameters:
blood glucose and insulin, cholesterol, triglycerides, HDL-cholesterol, LDL-cholesterol
Measured: change over baseline, week 8, week 16, week 24
Quality of Life
Psychological General Well-Being questionnaire
Measured: change over baseline, week 8, week 16, week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2015 (estimated)
- Study startAug 2012
- Primary completionDec 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- More trials run by University Hospital, CaenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Caen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02552251
- Sponsor's protocol ID
- 11-059
- Acronym
- COCA
- Status last verified
- Sep 2015
- First submitted
- 27 Jul 2015
- Last updated
- 17 Sep 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparative Study of the Use of Glucocorticoids in the Treatment of Congenital Adrenal Hyperplasia in Its Classical Form
- ClinicalTrials.gov ID
- NCT02552251
- Acronym / sponsor ID
- COCA / 11-059
- Conditions
- Congenital Adrenal Hyperplasia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- University Hospital, CaenUniversity, hospital or other
- Study start
- Aug 2012
- Primary completion
- Dec 2015 (estimated)
- First posted
- 17 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| A: hydrocortisone | Experimental | Biological: Hormonal balance measurements, Biological: metabolic balance measurements, Biological: bone balance measurements, Behavioral: quality of life assessmenthydrocortisone equivalent to physiological doses for each patient Strategy: administration of glucocorticoids during sequences of eight weeks |
| B :dexamethasone (DECTANCYL) | Experimental | Biological: Hormonal balance measurements, Biological: metabolic balance measurements, Biological: bone balance measurements, Behavioral: quality of life assessmentdexamethasone equivalent to physiological doses for each patient Strategy: administration of glucocorticoids during sequences of eight weeks |
| C : prednisone (CORTANCYL) | Experimental | Biological: Hormonal balance measurements, Biological: metabolic balance measurements, Biological: bone balance measurements, Behavioral: quality of life assessmentprednisone equivalent to physiological doses for each patient Strategy: administration of glucocorticoids during sequences of eight weeks |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Hormonal balance measurements | Biological | A: hydrocortisone, B :dexamethasone (DECTANCYL), C : prednisone (CORTANCYL) |
| metabolic balance measurements | Biological | A: hydrocortisone, B :dexamethasone (DECTANCYL), C : prednisone (CORTANCYL) |
| bone balance measurements | Biological | A: hydrocortisone, B :dexamethasone (DECTANCYL), C : prednisone (CORTANCYL) |
| quality of life assessment | Behavioural | A: hydrocortisone, B :dexamethasone (DECTANCYL), C : prednisone (CORTANCYL) |
Primary outcome
hormonal parameters
Time frame: change over baseline, week 8, week 16, week 24
6 points salivary 17 OHP cycle, and 8 am plasma ACTH,testosterone and androstenedione
3 secondary outcomes
parameters of bone turnover:
Time frame: change over baseline, week 8, week 16, week 24
metabolic parameters:
Time frame: change over baseline, week 8, week 16, week 24
Quality of Life
Time frame: change over baseline, week 8, week 16, week 24
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2015.
Related trials for Congenital Adrenal Hyperplasia
RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Natural History Study of Patients With Excess Androgen
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
RecruitingPhase 3
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
Mild Autonomous Cortisol Secretion (MACS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Hormonal balance measurements, metabolic balance measurements and bone balance measurements in people with Congenital Adrenal Hyperplasia, run by University Hospital, Caen at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 17 Sep 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
hormonal parameters (time frame: change over baseline, week 8, week 16, week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2015.