Primary outcome: Primary: Chronocort vs. Cortef Cortisol Concentrations (AUC Over 24 Hours - Time Points 0,.5,1,1.5,2,3,4,5,6,7,8,10,10.5,11, 11.5,12,13,15,17,17.5,18,18.5,19,20,22,24 Post Dose). — 5380; 3973 h*nmol/l
Study Design & Population
Study type
Interventional
Phase
Phase 2
Interventions
Chronocort (Drug); Cortef (Drug)
Age
Pediatric, Adult · 16+ yrs
Sex
All
Sponsor
Neurocrine UK Limited
Primary completion
May 2009
Outcome Measures
Outcome
Result
p-value
PRIMARY Chronocort vs. Cortef Cortisol Concentrations (AUC Over 24 Hours - Time Points 0,.5,1,1.5,2,3,4,5,6,7,8,10,10.5,11, 11.5,12,13,15,17,17.5,18,18.5,19,20,22,24 Post Dose).
5380; 3973
—
SECONDARY 17 Hydroxyprogesterone at 08.00 Hours
16.4; 86.9
—
Summary
This study will test a new, extended release form of hydrocortisone called Chronocort in patients with congenital adrenal hyperplasia (CAH). People with CAH do not make enough of the adrenal hormones cortisol and aldosterone, and their adrenal glands make too much of the sex hormone androgen. Medicines called glucocorticoids (hydrocortisone, dexamethasone and prednisone) are currently used to treat CAH, but finding the best dose of these drugs that effectively lowers androgens without causing undesirable side effects, such as weight gain and slow growth rate in children, is often difficult to achieve.
Adolescents and adults with CAH due to 21-hydroxylase deficiency may be eligible for this study. Children 16 years of age and older are eligible with confirmation by bone age that they are no longer growing.
Participants undergo the following tests and procedures during two inpatient visits one month apart at the NIH Clinical Center:
* Medical history and physical examination.
* Medications: Following 7 days of Cortef (standard drug treatment for CAH), patients begin taking Chronocort on day 3 of hospitalization and continue the tablets once a day for 1 month.
* Blood tests: A catheter (plastic tube) is inserted in a vein and left in place for frequent blood draws in order to avoid repeated needlesticks. Blood is drawn for chemistries, blood count, pregnancy test in women, and for serial tests (up to 26 samples in a 24-hour period) to measure hormone levels.
* 24-hour urine test.
* Height and weight measurements.
Between the two hospitalizations, patients are contacted by NIH weekly to check for possible side effects from Chronocort. Two weeks after the first visit, patients also will have blood drawn by their regular doctor or a local clinic. A few days before the second hospitalization, patients undergo a 20-minute telephone questionnaire about energy level and well being.
About 30 days after discharge from the second hospitalization, patients are followed up with a telephone call to see how they are doing.
Eligibility Criteria
INCLUSION CRITERIA:
Satisfactory pre-trial screening
Provision of signed written informed consent and written assent from patients less than 18 years old, as applicable.
Good general health.
Females of childbearing potential must have a negative pregnancy test initially and at all visits. Females who are engaging in sexual intercourse must be using medically acceptable method of contraception.
EXCLUSION CRITERIA
Co-morbid condition requiring daily administration of a medication that induces hepatic enzymes or interferes with the metabolism of glucocorticoids.
Clinical or biochemical evidence of hepatic or renal disease. Creatinine above the normal range or elevated liver function tests (Transaminases greater than 1.5 the upper limits of normal).
Females who are pregnant or lactating.
Patients with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the trial.
Participation in another clinical trial of an investigational or licensed drug or device within 3 months prior to inclusion in this study.
Data sourced from ClinicalTrials.gov (NCT00519818). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.