What is the mechanism of action of this treatment?

The most common treatments for Congenital Adrenal Hyperplasia (CAH) include glucocorticoids and CRF1 receptor antagonists like Crinecerfont. Glucocorticoids replace deficient cortisol and suppress excess adrenocorticotropic hormone (ACTH) production, reducing androgen overproduction. CRF1 receptor antagonists block the CRF1 receptor, decreasing ACTH release from the pituitary gland. This is crucial for CAH patients as it helps manage hormonal imbalances and prevent androgen overproduction, thereby mitigating symptoms and improving quality of life.

What side effects are associated with this type of intervention?

Common treatments for Congenital Adrenal Hyperplasia (CAH) include glucocorticoids and mineralocorticoids. Glucocorticoids, such as hydrocortisone, prednisone, or dexamethasone, can cause side effects like weight gain, hypertension, osteoporosis, and growth suppression in children. Mineralocorticoids, like fludrocortisone, may lead to hypertension, edema, and electrolyte imbalances. For CRF1 receptor antagonists like Crinecerfont, specific side effects are still being studied, but potential concerns could include gastrointestinal distress, headache, and possible impacts on cortisol levels.

What prior FDA approvals exist?

The FDA has approved several treatments for Congenital Adrenal Hyperplasia (CAH), primarily focusing on glucocorticoid and mineralocorticoid replacement therapies to manage hormone imbalances. Commonly used medications include hydrocortisone, prednisone, and dexamethasone for glucocorticoid replacement, and fludrocortisone for mineralocorticoid replacement. These treatments aim to reduce excess androgen production and maintain electrolyte balance. While Crinecerfont, a CRF1 receptor antagonist, is currently under investigation, it represents a novel approach targeting the hypothalamic-pituitary-adrenal axis to potentially offer more targeted and effective management of CAH.

What other types of research exist?

Promising novel alternatives to Crinecerfont for CAH patients include gene therapy approaches, new formulations of glucocorticoids with improved pharmacokinetics, and other CRF1 receptor antagonists currently under investigation. Additionally, advancements in enzyme replacement therapies and the development of selective steroidogenesis inhibitors are also being explored as potential treatments.

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Corticosteroid

Crinecerfont for Congenital Adrenal Hyperplasia

Phase 3

Waitlist Available

Research Sponsored by Neurocrine Biosciences

Eligibility Criteria Checklist

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

Must have

Have a medically confirmed diagnosis of 21-hydroxylase deficiency CAH.

Be on a stable regimen of steroidal treatment for CAH.

Must not have

Have a diagnosis of any of the other forms of classic CAH.

Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing a medication called crinecerfont to help children with a hormone disorder called CAH. The study will compare crinecerfont to another treatment over several months. The goal is to see if crinecerfont can safely and effectively balance their hormone levels.

Who is the study for?

This trial is for pediatric patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. Participants must be on stable steroidal treatment, have high androgen levels, and agree to birth control if applicable. Exclusions include unstable medical conditions, cancer history unless cured, recent investigational drug use or significant blood loss, other CAH forms, known drug hypersensitivity, certain surgeries or chronic conditions.

What is being tested?

The study tests the effectiveness of Crinecerfont compared to a placebo in children with CAH over a period of 28 weeks. After this phase, all participants receive Crinecerfont for another 24 weeks. Those interested can join an open-label extension for about three years to further assess long-term effects.

What are the potential side effects?

While specific side effects are not listed here, generally such trials monitor for adverse reactions related to the medication under investigation which could range from mild symptoms like headaches or nausea to more serious issues depending on individual patient response.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with 21-hydroxylase deficiency CAH.

Select...

I am on a consistent dose of steroids for congenital adrenal hyperplasia.

Select...

My androgen levels are high.

Exclusion Criteria

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

Select...

I have been diagnosed with a form of classic congenital adrenal hyperplasia.

Select...

I need long-term steroid treatment due to a condition like hypopituitarism or after having both adrenal glands removed.

Select...

I have a serious health condition that is not CAH.

Select...

I have a history of significant heart rhythm problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2021 Phase 2 trial • 8 Patients • NCT04045145

25%

Headache

13%

Myalgia

13%

Dermatitis contact

13%

Gastritis

13%

Frequent bowel movements

13%

Pyrexia

13%

Arthropod sting

13%

Nasopharyngitis

13%

Dizziness

13%

Blepharospasm

13%

Vomiting

100%

80%

60%

40%

20%

Study treatment Arm

Crinecerfont 50 mg

Awards & Highlights

Pivotal Trial

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

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: CrinecerfontExperimental Treatment1 Intervention

Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.

Group II: PlaceboPlacebo Group2 Interventions

Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Crinecerfont

2019

Completed Phase 2

~10

0 / 7Eligibility 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) include glucocorticoids and CRF1 receptor antagonists like Crinecerfont. Glucocorticoids replace deficient cortisol and suppress excess adrenocorticotropic hormone (ACTH) production, reducing androgen overproduction. CRF1 receptor antagonists block the CRF1 receptor, decreasing ACTH release from the pituitary gland. This is crucial for CAH patients as it helps manage hormonal imbalances and prevent androgen overproduction, thereby mitigating symptoms and improving quality of life.