Pilot Study to Characterize and Examine the Pharmacokinetics and Efficacy of Chronocort® in Adults With CAH
Condition: Endocrine Disease; Adrenal Insufficiency; Congenital Adrenal Hyperplasia
MeSH terms: Adrenal Hyperplasia, Congenital; Adrenogenital Syndrome; Adrenal Insufficiency; Adrenocortical Hyperfunction; Endocrine System Diseases; Hyperplasia
Intervention: Hydrocortisone Modified Release Capsules
Intervention description: 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
Intervention MeSH terms: Hydrocortisone
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 16.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: congenital adrenal hyperplasia; glucocorticoids; cortisol; adrenal

Summary:

      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.
    

Eligibility 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.