The purpose of this research study is to compare the safety and effectiveness of Chronocort® with current glucocorticoid treatment regimens in the treatment of CAH over a 6 month period.
ID
Source
Brief title
Condition
- Adrenal gland disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
To demonstrate the superior efficacy of Chronocort® compared with standard
glucocorticoid replacement therapy in the treatment of CAH based on 17-OHP.
Secondary outcome
In adult subjects with CAH:
* To assess the safety and tolerability of Chronocort® treatment in adult
subjects with CAH over a
6-month period.
* To assess the efficacy of Chronocort® with regard to the effect on A4 over
the 6-month treatment period.
* To assess the impact of Chronocort® on body composition (using dual energy
X-ray absorbtimetry
[DEXA]) * fat mass, lean mass and total bone density * at selected sites.
Background summary
The glucocorticoid medications currently used to treat CAH are like cortisol,
but they cannot simulate natural cortisol in terms of how it is produced in the
body. This is because cortisol is normally released in different amounts
throughout the day with a pulse, or burst, during the early morning hours and
very low amounts in the body in the evening.
Chronocort® has been designed so that the amounts of cortisol in the body more
closely mimic the human body*s normal release pattern of cortisol.
It is expected that the better metabolic control results in less adverse
effects, such as obesitas, and consequently to a better quality of life.
Study objective
The purpose of this research study is to compare the safety and effectiveness
of Chronocort® with current glucocorticoid treatment regimens in the treatment
of CAH over a 6 month period.
Study design
randomized, open-label study with parallel groups
Intervention
Chronocort treatment versus standard regimen
Study burden and risks
Chronocort is a new formulation of a medicine, hydrocortisone, that has been
used as a treatment for CAH and similar conditions for several years There is a
lot of published information regarding the safety of this hydrocortisone. The
dose of hydrocortisone being administered as Chronocort® in this study is
within the expected dose level to replace cortisone in the body for patients
with CAH. Commons side effects from Hydrocortisone can include (occurring in
around 30% of patients):
*Increased appetite
*Irritability
*Difficulty sleeping (insomnia)
*Swelling in ankles and feet (fluid retention)
*Nausea (take with food)
*Heartburn
*Muscle weakness
*Impaired wound healing
*Increased blood sugar levels
Further to this, adverse effects may occur if too much or too little medication
is used. In these cases patients may experience symptoms of too little cortisol
replacement (e.g., fatigue) or too much cortisol replacement (e.g., weight
gain). Patient will undergo four 24 hour endocrine profiles to assess if they
are being treated with the correct amount of glucocorticoid to treat their CAH
adequately
The study involves a DEXA scan which will expose patient to radiation. The
study involves the inconvenience of needing to attend four overnight stays at
the hospital in order to have a 24 hour endocrine profile performed. Patients
will be advised in advance of the schedule for these visits* which will also
form part of the informed consent process
De Stadspoort 32
Eindhoven 5611 GZ
NL
De Stadspoort 32
Eindhoven 5611 GZ
NL
Listed location countries
Age
Inclusion criteria
1. Known CAH due to 21-hydroxylase deficiency (classic CAH) diagnosed in childhood with documented (at any time) elevated 17-hydroxyprogesterone (17-OHP) and/or androstenedione (A4) and currently treated with hydrocortisone, prednisone, prednisolone or dexamethasone (or a combination of the aforementioned glucocorticoids) on a stable glucocorticoid therapy for a minimum of 6 months.
2. Male or female subjects aged 18 and above.
3. Provision of signed written informed consent.
4. Non-pregnant, non-lactating females who are post menopausal, naturally or surgically sterile, or of childbearing potential with a negative urinary pregnancy test and using a medically acceptable method of contraception. (Note: female subjects with oligomenorrhoea or amenorrhoea aged 55 or under should be considered potnetially fertile and therefore it is expected that they, besides undergoing pregnancy testing, should use an acceptable method of contraception.
5. Plasma renin activity (PRA) less than 1.5 times the upper limit of normal (ULN) at screening or within 3 months prior to screening, except in subjects who have been diagnosed with hypertension where the renin is not being used to monitor fludrocortisone replacement.
Exclusion criteria
1. Co-morbid condition requiring daily administration of a medication (or consumption of any material) that interferes with the metabolism of glucocorticoids.
2. Clinical or biochemical evidence of hepatic or renal disease. Creatinine over twice the ULN or elevated liver function tests (ALT or AST >2 times the ULN).
3. Subjects on regular daily inhaled, topical, nasal or oral steroids for any indication other than CAH.
4. Subjects with any other significant medical or psychiatric conditions that in the opinion of the investigator would preclude participation in the trial.
5. History of malignancy (other than basal cell carcinoma successfully treated >6 months prior to entry into the study).
6. Participation in another clinical trial of an investigational or licensed drug or device within the 3 months prior to inclusion in this study.
7. Subjects with a history of bilateral adrenalectomy.
8. Subjects having previously been exposed to Chronocort®.
9. Subjects working routinely in night shifts and therefore do not sleep during the usual night time.
10. Subjects unable to comply with the requirements of the protocol.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2015-000711-40-NL |
| CCMO | NL54707.091.15 |