Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01735617
• Date of registration: 20/11/2012
• Prospective Registration: Yes
• Primary sponsor: Diurnal Limited
• Public title: Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH
• Scientific title: A Phase 2 Pilot Study to Characterize and Examine the Pharmacokinetics and Disease Bio-marker Response of Chronocort® in Adults With Congenital Adrenal Hyperplasia
• Date of first enrolment: December 2012
• Target sample size: 16
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01735617
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Deborah P Merke, BS, MS, MD
• Affiliation: National Institutes of Health Clinical Center (CC)

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Known CAH due to 21-hydroxylase deficiency (classic CAH) based on hormonal and genetic
testing currently treated with hydrocortisone, prednisone, prednisolone or
dexamethasone on a stable dosage for a minimum of 3 months.

2. Male or female patients aged 18 and above.

3. Provision of signed written informed consent.

4. Good general health.

5. 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 a medically
acceptable method of contraception (as defined in the protocol, section 10.5).

6. Plasma renin activity must be within the clinically acceptable range at screening
(less than 1.5 times upper normal range).

Exclusion Criteria:

1. Co-morbid condition requiring daily administration of a medication that induces
hepatic enzymes or interferes with the metabolism of glucocorticoids.

2. Clinical or biochemical evidence of hepatic or renal disease. Creatinine above the
normal range or elevated liver function tests (ALT or AST) > 2 times the upper limits
of normal.

3. Females who are pregnant or lactating.

4. Women taking an estrogen-containing oral contraceptive pill and who have taken it
within 6 weeks of recruitment.

5. Patients taking spironolactone.

6. Patients on inhaled or oral steroids apart from treatment for CAH.

7. Patients with any other significant medical or psychiatric conditions that in the
opinion of the Investigator would preclude participation in the trial.

8. Participation in another clinical trial of an investigational or licensed drug or
device within the 3 months prior to inclusion in this study.

9. Patients with history of bilateral adrenalectomy.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Congenital Adrenal Hyperplasia

Endocrine Disease

Adrenal Insufficiency

Intervention(s)

Drug: Hydrocortisone Modified Release Capsules

Primary Outcome(s)

Pharmacokinetic Profile (AUC0-24) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia [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 (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia [Time Frame: 24 hours]

Pharmacokinetic Profile (Tmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia [Time Frame: 24 hours]

Secondary Outcome(s)

17-OHP Levels at 0700h, 1700h and 2300h [Time Frame: Specified time points (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 [Time Frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)]

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) [Time Frame: Specific time point (0700hrs)]

Androstenedione Levels at 0700h, 1700h and 2300h [Time Frame: Specified time points (0700h, 1700h and 2300h)]

AUC Values (Pmol*h/L) for ACTH [Time Frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)]

AUC Values (Nmol*h/L) for 17-OHP [Time Frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)]

Secondary ID(s)

DIUR-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Institutes of Health (NIH)

Results

• Results available: Yes
• Date Posted: 17/05/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01735617