Chronocort for Congenital Adrenal Hyperplasia

Recruiting at 20 trial locations

Overseen By Diurnal Information Line

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Neurocrine UK Limited

Must be taking: Chronocort

Must not be taking: Steroids, others

Disqualifiers: Hepatic disease, Renal disease, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

76 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing Chronocort, a medication for people aged 16 and over with Congenital Adrenal Hyperplasia (CAH). It aims to see if Chronocort can safely and effectively manage their hormone levels. Chronocort works by releasing hormones in a way that mimics the body's natural rhythm. Chronocort is a modified-release formulation of hydrocortisone designed to better mimic the body's natural cortisol circadian rhythm.

Will I have to stop taking my current medications?

The trial requires that participants do not take medications that interfere with glucocorticoid metabolism or regular daily steroids for conditions other than CAH. If you are on such medications, you may need to stop them to participate.

What data supports the effectiveness of the drug Chronocort for congenital adrenal hyperplasia?

Chronocort, a modified-release hydrocortisone, was compared to conventional hydrocortisone in patients with congenital adrenal hyperplasia, suggesting it may offer improved hormonal control. This indicates potential effectiveness in managing the condition by providing a more physiological replacement of glucocorticoids. ^{1 2 3 4 5}

Is Chronocort safe for humans?

Chronocort, a modified-release hydrocortisone, has been studied for congenital adrenal hyperplasia and compared to conventional hydrocortisone. While the study focused on hormonal profiles, it implies that Chronocort is generally safe as it is being evaluated as an alternative to existing treatments. ^{1 2 3 6 7}

How does the drug Chronocort differ from other treatments for congenital adrenal hyperplasia?

Chronocort is a modified-release form of hydrocortisone that aims to provide a more natural hormone profile compared to conventional hydrocortisone treatments, potentially improving the management of congenital adrenal hyperplasia by mimicking the body's natural hormone rhythms. ^{1 2 3 4 8}

Research Team

D Merke

Principal Investigator

National Instiututes of Health Clinical Centre, Bethesda, Maryland, United States, 20892-1932

Eligibility Criteria

This trial is for individuals over 16 with Congenital Adrenal Hyperplasia (CAH) who've completed previous Chronocort studies. They must be able to consent and not have conditions that interfere with the study or glucocorticoid metabolism, no recent participation in other drug trials, not pregnant/lactating, and without severe liver/renal disease.

Inclusion Criteria

Participants with Congenital Adrenal Hyperplasia (CAH) who have successfully completed Chronocort study DIUR-006 (sites in France and US only) or study DIUR-014

I can sign and agree to follow the study's rules.

Exclusion Criteria

Participants who routinely work night shifts and so do not sleep during the usual night-time hours

My body weight is 50 kg or less.

Participants with any other significant medical or psychiatric conditions that in the opinion of the Investigator would preclude participation in the study

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants continue or switch to Chronocort treatment for long-term safety and tolerability assessment

Up to 32 months

Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Chronocort

Trial Overview The trial is testing the long-term safety of a medication called Chronocort for treating CAH. It's an open-label extension study across multiple countries where participants will continue using Chronocort to assess its ongoing safety profile.

Participant Groups

Group I: Chronocort (hydrocortisone modified-release capsule) Experimental Treatment 1 Intervention

Chronocort (hydrocortisone modified-release capsules) supplied as 5 mg and 10 mg per capsule for oral administration.

Who Is Running the Clinical Trial?

Neurocrine UK Limited

Lead Sponsor

Trials

19

Recruited

710+

Diurnal Limited

Lead Sponsor

Trials

19

Recruited

710+

Findings from Research

Modified-release hydrocortisone (MR-HC) provides a more physiological cortisol profile compared to conventional hydrocortisone (HC), with lower cortisol levels in the afternoon and night, which may improve treatment for congenital adrenal hyperplasia (CAH).

In a study of 14 patients, MR-HC resulted in better control of overnight adrenal androgens, but showed increased afternoon levels of certain hormones, indicating that a morning dose may be necessary for optimal management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pharmacokinetic and pharmacodynamic study of delayed- and extended-release hydrocortisone (Chronocort) vs. conventional hydrocortisone (Cortef) in the treatment of congenital adrenal hyperplasia. Verma, S., Vanryzin, C., Sinaii, N., et al.[2022]

References

1. England pubmed.ncbi.nlm.nih.gov

Monitoring treatment in congenital adrenal hyperplasia. [2019]

2. England pubmed.ncbi.nlm.nih.gov

A pharmacokinetic and pharmacodynamic study of delayed- and extended-release hydrocortisone (Chronocort) vs. conventional hydrocortisone (Cortef) in the treatment of congenital adrenal hyperplasia. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Hormonal circadian rhythms in patients with congenital adrenal hyperplasia: identifying optimal monitoring times and novel disease biomarkers. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Congenital adrenal hyperplasia complicated by central precocious puberty: linear growth during infancy and treatment with gonadotropin-releasing hormone analog. [2019]

5. Italy pubmed.ncbi.nlm.nih.gov

[Growth and puberal development in males with adreno-genital syndrome]. [2006]

6. Switzerland pubmed.ncbi.nlm.nih.gov

Timing and type of glucocorticoid replacement in adult congenital adrenal hyperplasia. [2018]

7. England pubmed.ncbi.nlm.nih.gov

Subjective health status in men and women with congenital adrenal hyperplasia: a population-based survey in Norway. [2022]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Gonadotropin releasing hormone analog treatment in children with congenital adrenal hyperplasia complicated by central precocious puberty. [2019]