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, thereby reducing the overproduction of androgens. CRF1 receptor antagonists block the corticotropin-releasing factor 1 receptor, reducing ACTH release from the pituitary gland. This helps manage the hormonal imbalances in CAH patients, which is essential for controlling symptoms and improving long-term health outcomes.

What side effects are associated with this type of intervention?

Common treatments for Congenital Adrenal Hyperplasia (CAH) include glucocorticoids and mineralocorticoids. Known side effects of glucocorticoids can include weight gain, hypertension, osteoporosis, and increased risk of infections. Mineralocorticoids may cause hypertension, edema, and electrolyte imbalances. For treatments similar to Crinecerfont, which is a CRF1 receptor antagonist, specific side effects are not well-documented, but potential side effects could include gastrointestinal distress, headache, and fatigue, based on the general profile of receptor antagonists.

What prior FDA approvals exist?

Historically, the treatment of Congenital Adrenal Hyperplasia (CAH) has primarily involved glucocorticoids like hydrocortisone, prednisone, and dexamethasone to replace deficient cortisol and suppress excess androgen production. Mineralocorticoids such as fludrocortisone are also used to manage salt-wasting forms of CAH. While Crinecerfont, a CRF1 receptor antagonist, is currently under investigation, it represents a novel approach targeting the underlying pathophysiology of CAH by modulating the hypothalamic-pituitary-adrenal axis. This could potentially offer an alternative to traditional steroid therapy, aiming to reduce the long-term side effects associated with chronic steroid use.

What other types of research exist?

Currently, there are no specific novel alternatives to Crinecerfont mentioned in the provided context for the treatment of Congenital Adrenal Hyperplasia (CAH). Patients should consult with their healthcare provider to discuss the latest research and emerging treatments in the field.

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Corticosteroid

Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst Trial)

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

Be on a stable regimen of steroidal treatment for CAH.

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

Must not have

Have a history of cancer unless considered cured.

Have a known history of clinically significant arrhythmia or abnormalities on ECG.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new medication called crinecerfont in adults with a genetic condition that affects hormone production. The study aims to see if crinecerfont can help balance their hormone levels and improve their symptoms. Participants will take the medication for several months, with an option to continue longer.

Who is the study for?

Adults with classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency can join. They must be on stable CAH medication, agree to use contraception if they can have children, and follow study rules. Excluded are those with unstable conditions, hypersensitivity to similar drugs, certain medical histories like cancer or chronic glucocorticoid therapy needs.

What is being tested?

The trial is testing Crinecerfont against a placebo for CAH over 24 weeks in a double-blind setup where neither the participants nor the researchers know who gets what. After this phase, there's an open-label period where everyone receives Crinecerfont for up to three years.

What are the potential side effects?

While specific side effects of Crinecerfont aren't listed here, common ones may include reactions at the injection site, hormonal imbalances leading to mood changes or skin issues, and potential risks associated with long-term hormone therapy.

Eligibility Criteria

Inclusion Criteria

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

Select...

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

Select...

I have been diagnosed with classic 21-hydroxylase deficiency CAH.

Exclusion Criteria

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

Select...

I have had cancer in the past but am considered cured.

Select...

I have a history of heart rhythm problems.

Select...

I have a serious health condition that is not CAH.

Select...

I have lost or donated more than 550 mL of blood in the last 8 weeks.

Select...

I need long-term steroid treatment due to a specific medical condition.

Select...

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

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%

Gastritis

13%

Myalgia

13%

Dermatitis contact

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 capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.

Group II: PlaceboPlacebo Group2 Interventions

Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Crinecerfont

2019

Completed Phase 2

~10

0 / 8Eligibility 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, thereby reducing the overproduction of androgens. CRF1 receptor antagonists block the corticotropin-releasing factor 1 receptor, reducing ACTH release from the pituitary gland. This helps manage the hormonal imbalances in CAH patients, which is essential for controlling symptoms and improving long-term health outcomes.