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Endocrine DiseasePhase 2NCT01735617

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

A Phase 2 study testing Hydrocortisone Modified Release Capsules in people with Endocrine Disease and Adrenal Insufficiency, run by Neurocrine UK Limited at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Hydrocortisone Modified Release Capsules
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteUnited States
Study dates
Dec 2012to Dec 2013
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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).

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • National Institutes of Health Clinical Center

    Bethesda, Maryland, 20892-1932

Other trials in United States

About this study

Official 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

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.

Read the detailed description

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.

Being tested
Hydrocortisone Modified Release Capsules
Keywords
congenital adrenal hyperplasia, glucocorticoids, cortisol, adrenal
Medical subject headings
Endocrine System Diseases, Adrenal Insufficiency, Adrenal Hyperplasia, Congenital

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Hydrocortisone Modified Release CapsulesDrug

    Also called Chronocort

    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

Study groups in NCT01735617
Study groupRoleWhat they receive
Hydrocortisone Modified Release CapsulesExperimentalChronocort Modified Release Capsules, 5mg, 10mg and 20mg Dosing frequency twice-daily (mane and nocte) Dose setting by titration to achieve optimal biochemical and therapeutic response
Receives: Drug: Hydrocortisone Modified Release Capsules

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    The maximum plasma concentration (Cmax) of chronocort

    Measured: 24 hours

  2. 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)

    Measured: 24 hours (at 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 to maximum plasma concentration (tmax)

    Measured: 24 hours

Secondary outcomes (7)

  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)

    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

    Measured: Specific time point (0700hrs)

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

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

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

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

    Androstenedione levels at 0700h, 1700h and 2300h

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

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

    ACTH levels at 0700h, 1700h and 2300h

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

  5. 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

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

  6. 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

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

  7. 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

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Nov 2012 (estimated)
  2. Study startDec 2012
  3. Primary completionDec 2013
  4. Study completionDec 2013

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01735617
Sponsor's protocol ID
DIUR-003
Status last verified
Sep 2026
First submitted
20 Nov 2012
Last updated
2 Oct 2026
Data monitoring committee
No
Individual data sharing
No

Publications

  • Mallappa A, Sinaii N, Kumar P, Whitaker MJ, Daley LA, Digweed D, Eckland DJ, Van Ryzin C, Nieman LK, Arlt W, Ross RJ, Merke DP. A phase 2 study of Chronocort, a modified-release formulation of hydrocortisone, in the treatment of adults with classic congenital adrenal hyperplasia. J Clin Endocrinol Metab. 2015 Mar;100(3):1137-45. doi: 10.1210/jc.2014-3809. Epub 2014 Dec 11.

    PubMed 25494662

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Hydrocortisone Modified Release Capsules in people with Endocrine Disease and Adrenal Insufficiency, run by Neurocrine UK Limited at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 May 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Pharmacokinetic Profile (Cmax) Following Short-term Treatment With Chronocort® in Adult Patients With Congenital Adrenal Hyperplasia (time frame: 24 hours). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01735617, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2026.

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