What side effects are associated with this type of intervention?

Common treatments for Congenital Adrenal Hyperplasia (CAH), such as hydrocortisone and other glucocorticoids, can have significant side effects. These include growth retardation, Cushingoid appearance, elevated blood pressure, hyperglycemia, cataracts, and osteoporosis. These side effects are particularly concerning in children and require careful monitoring and management to minimize adverse outcomes.

What prior FDA approvals exist?

The FDA has approved various glucocorticoid treatments for Congenital Adrenal Hyperplasia (CAH), primarily focusing on hydrocortisone, prednisone, and dexamethasone. These treatments aim to replace deficient cortisol levels and manage androgen excess. Chronocort, a modified-release hydrocortisone, is designed to mimic the natural circadian rhythm of cortisol secretion, offering a more physiological approach to treatment. This formulation is currently being studied for its long-term safety and efficacy in CAH patients.

What other types of research exist?

Promising novel alternatives to Chronocort for CAH patients include Efmody (modified-release hydrocortisone) and gene therapy approaches such as CRISPR-Cas9 mediated gene editing. Efmody, like Chronocort, aims to mimic the natural circadian rhythm of cortisol. Gene therapy offers a potential long-term solution by correcting the underlying genetic defect causing CAH.

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Corticosteroid

Chronocort for Congenital Adrenal Hyperplasia

Phase 3

Waitlist Available

Led By D Merke

Research Sponsored by Diurnal Limited

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Must not have

Participants with a body weight of 50 kg or less

Participants with a history of bilateral adrenalectomy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 32 months

Awards & highlights

All Individual Drugs Already Approved

Approved for 60 Other Conditions

No Placebo-Only Group

Pivotal Trial

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, typical concerns may include potential impacts on organ function like liver or kidneys due to long-term medication use. Participants' health will be closely monitored for any adverse reactions.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My body weight is 50 kg or less.

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I have had both of my adrenal glands removed.

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My kidney or liver tests show they are not working well.

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I use steroids daily for reasons other than adrenal gland disorders.

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I am not pregnant or breastfeeding.

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I take daily medication that affects how my body uses steroids.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 32 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 32 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 32 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Signs and symptoms of over-treatment [Safety and Tolerability] throughout the study.

Signs and symptoms of under-treatment [Safety and Tolerability] throughout the study.

To measure signs or symptoms of under treatment [Safety and Tolerability] in terms of incidence of adrenal crises throughout the study.

+5 more

Secondary study objectives

To assess the impact of treatment on 17-Hydroxyprogesterone (17-OHP) levels - Change from pre-Chronocort baseline

To assess the impact of treatment on Androstenedione (A4) levels - Change from pre-Chronocort baseline

To assess the impact of treatment on body weight - Change from pre-Chronocort baseline

+5 more

Other study objectives

To assess the impact of treatment on 17-Hydroxyprogesterone (17-OHP) levels - Change from initial study baseline

To assess the impact of treatment on Androstenedione (A4) levels - Change from initial study baseline

To assess the impact of treatment on body weight - Change from initial study baseline

+7 more

Side effects data

From 2009 Phase 1 & 2 trial • 20 Patients • NCT00519818

21%

Headache

100%

80%

60%

40%

20%

Study treatment Arm

Cortef

Chronocort

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 60 Other Conditions

This treatment demonstrated efficacy for 60 other conditions.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Chronocort (hydrocortisone modified-release capsule)Experimental Treatment1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Hydrocortisone

FDA approved

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Congenital Adrenal Hyperplasia (CAH) involve glucocorticoid therapy, which aims to replace deficient cortisol and suppress excess adrenal androgen production. Modified-release hydrocortisone, such as Chronocort, mimics the natural circadian rhythm of cortisol secretion, providing more physiological hormone levels throughout the day. This approach helps to better control symptoms, reduce the risk of adrenal crises, and minimize side effects associated with traditional glucocorticoid regimens. For CAH patients, maintaining a stable and natural cortisol rhythm is crucial for overall health, growth, and development.