The purpose of this study is to gather safety and effectiveness information about a new formulation of Hydrocortisone (Chronocort®) used to treat patients with a disease called congenital adrenal hyperplasia (CAH). Hydrocortisone is the man-made version of the hormone cortisol, which is released in the body following a regular daily pattern. The objective of the study is to measure the levels of hydrocortisone that are absorbed into the bloodstream once Chronocort® is taken and what affects it has on other hormones in the body. Since Chronocort® is anticipated to mimic the same release pattern of cortisol in the body, it is hoped that patients with CAH will be treated more effectively to manage their disease.
This study is a Phase 2 pilot study to characterize and examine the pharmacokinetics and efficacy profile of Chronocort® in adults with congenital adrenal hyperplasia (CAH). It is designed as a two-part, single cohort, open label, multiple dose Phase 2 pilot study to: (Part A) characterize and examine the pharmacokinetics (PK) and disease bio-marker behavior following short-term dosing with Chronocort®; and to (Part B) examine the disease control after six months dose titration with Chronocort® in adults with CAH.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
Patients with congenital adrenal hyperplasia standardised on conventional therapy is enrolled onto the study and treatment is switched to Chronocort, initially for pharmacokinetic assessment followed by longer-term biochemical and efficacy assessment
National Institutes of Health Clinical Center
Bethesda, Maryland, United States
Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
The maximum plasma concentration (Cmax) of chronocort
Time frame: 24 hours
Pharmacokinetic Profile (AUC0-24) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
Area under the curve (AUC) from 0 to 24 hours (sampling occurs at the following timepoints: 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs)
Time frame: 24 hours (at 2300, 0100, 0300, 0500, 0600, 0700, 0800, 0900, 1000, 1100, 1200, 1300, 1400, 1500, 1600, 1700, 1900, 2100, 2300hrs)
Pharmacokinetic Profile (Tmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia
Time to maximum plasma concentration (tmax)
Time frame: 24 hours
The Percentage of Patients With 17-OHP and Androstenedione Levels at 0700h Within Proposed Optimal Ranges Whilst on Chronocort and Whilst on Standard Therapy (at Baseline)
Proposed optimal ranges of 17-OHP: 300-1200ng/dl Proposed optimal ranges of androstenedione: 40-150ng.dl for males and 30-200ng/dl for females
Time frame: Specific time point (0700hrs)
17-OHP Levels at 0700h, 1700h and 2300h
17-OHP levels at 0700h, 1700h and 2300h
Time frame: Specified time points (0700h, 1700h and 2300h)
Androstenedione Levels at 0700h, 1700h and 2300h
Androstenedione levels at 0700h, 1700h and 2300h
Time frame: Specified time points (0700h, 1700h and 2300h)
ACTH Levels at 0700h, 1700h and 2300h
ACTH levels at 0700h, 1700h and 2300h
Time frame: Specified time points (0700h, 1700h and 2300h)
AUC Values (Nmol*h/L) for Androstenedione
AUC values (nmol\*h/L) for Androstenedione for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h
Time frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
AUC Values (Nmol*h/L) for 17-OHP
AUC values (nmol\*h/L) for 17-OHP for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h
Time frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
AUC Values (Pmol*h/L) for ACTH
AUC values (pmol\*h/L) for ACTH for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h
Time frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)
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