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Completed

NCT Number: NCT01735617

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

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.

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Key information

Conditions

Endocrine Disease Adrenal Gland Diseases Adrenal Hyperplasia, Congenital Adrenal Insufficiency Adrenogenital Syndrome Congenital Abnormalities Congenital Adrenal Hyperplasia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disorders of Sex Development Endocrine System Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Gonadal Disorders Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Steroid Metabolism, Inborn Errors Urogenital Abnormalities Urogenital Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892-1932, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.
  • Male or female patients aged 18 and above.
  • Provision of signed written informed consent.
  • 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 a medically acceptable method of contraception (as defined in the protocol, section 10.5).
  • Plasma renin activity must be within the clinically acceptable range at screening (less than 1.5 times upper normal range).

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 (ALT or AST) > 2 times the upper limits of normal.
  • Females who are pregnant or lactating.
  • Women taking an estrogen-containing oral contraceptive pill and who have taken it within 6 weeks of recruitment.
  • Patients taking spironolactone.
  • Patients on inhaled or oral steroids apart from treatment for CAH.
  • 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 the 3 months prior to inclusion in this study.
  • Patients with history of bilateral adrenalectomy.

Treatment and study plan

Hydrocortisone Modified Release Capsules

Drug

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

Other names: Chronocort

Primary outcomes

  1. Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia

    Time frame: 24 hours

    The maximum plasma concentration (Cmax) of chronocort

  2. 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)

    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)

  3. Pharmacokinetic Profile (Tmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia

    Time frame: 24 hours

    Time to maximum plasma concentration (tmax)

Secondary outcomes

  1. 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)

    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

  2. 17-OHP Levels at 0700h, 1700h and 2300h

    Time frame: Specified time points (0700h, 1700h and 2300h)

    17-OHP levels at 0700h, 1700h and 2300h

  3. Androstenedione Levels at 0700h, 1700h and 2300h

    Time frame: Specified time points (0700h, 1700h and 2300h)

    Androstenedione levels at 0700h, 1700h and 2300h

  4. ACTH Levels at 0700h, 1700h and 2300h

    Time frame: Specified time points (0700h, 1700h and 2300h)

    ACTH levels at 0700h, 1700h and 2300h

  5. AUC Values (Nmol*h/L) for Androstenedione

    Time frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)

    AUC values (nmol*h/L) for Androstenedione for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h

  6. AUC Values (Nmol*h/L) for 17-OHP

    Time frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)

    AUC values (nmol*h/L) for 17-OHP for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h

  7. AUC Values (Pmol*h/L) for ACTH

    Time frame: Specific time points (2300-2300h, 2300-0700h, 0700-1500h and 1500-2300h)

    AUC values (pmol*h/L) for ACTH for the following reporting periods: 24 hours (2300-2300h), 2300-0700h, 0700-1500h and 1500-2300h

Sponsors and collaborators

Lead sponsor

Neurocrine UK Limited

Industry

Collaborators

  • National Institutes of Health (NIH)

Registry information

Official study 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

Important dates

Study start
2012
Primary completion
2013
Study completion
2013
First posted
Nov 28, 2012
Registry last updated
May 17, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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