What is the mechanism of action of this treatment?

The most common treatments for Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency involve glucocorticoids and mineralocorticoids. Glucocorticoids provide cortisol replacement, which suppresses the overproduction of adrenocorticotropic hormone (ACTH) and reduces excessive adrenal androgen production. Mineralocorticoids help manage aldosterone deficiency, maintaining salt balance and blood pressure. These treatments are crucial for CAH patients to prevent adrenal crises, manage symptoms, and improve overall quality of life. The trial drug CRN04894 likely works by modulating adrenal steroidogenesis, similar to glucocorticoids, to address the enzyme deficiency.

What side effects are associated with this type of intervention?

Common treatments for Congenital Adrenal Hyperplasia (CAH) that modulate adrenal steroidogenesis, such as glucocorticoids and mineralocorticoid replacement, can have several side effects. Glucocorticoids may cause growth impairment in children, increased infection risk, hypertension, osteoporosis, and adrenal suppression. Mineralocorticoid replacement can lead to hypertension, hypokalemia, and fluid retention. Careful monitoring and dose adjustments are essential to manage these side effects effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Congenital Adrenal Hyperplasia (CAH) caused by 21-hydroxylase deficiency, primarily focusing on glucocorticoids like hydrocortisone, prednisone, and dexamethasone. These medications work by replacing cortisol and suppressing excess androgen production. Additionally, mineralocorticoids such as fludrocortisone are used to manage aldosterone deficiency. These treatments aim to modulate adrenal steroidogenesis, similar to the mechanism being studied in the trial CRN04894.

What other types of research exist?

Promising alternatives to CRN04894 for treating Congenital Adrenal Hyperplasia (CAH) include gene therapy approaches, such as those targeting the underlying genetic mutations causing 21-hydroxylase deficiency. Additionally, other novel steroidogenesis modulators or enzyme replacement therapies that are currently in clinical trials may also be viable options. Specific examples include advancements in gene editing technologies like CRISPR-Cas9 and other small molecule inhibitors that aim to correct or compensate for the enzymatic deficiencies in CAH.

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Corticosteroid

CRN04894 for Congenital Adrenal Hyperplasia

Phase 2

Recruiting

Research Sponsored by Crinetics Pharmaceuticals Inc.

Eligibility Criteria Checklist

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

Must have

Classic 21-hydroxylase deficiency

Minimum total daily dose of ≥15 mg hydrocortisone (or equivalent)

Must not have

History of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy

Participants with hypothyroidism who are not receiving adequate hormone replacement therapy based on thyroid hormone levels measured at the time of Screening

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug called CRN04894 in people with a genetic condition called classic congenital adrenal hyperplasia (CAH). The condition affects hormone production, and current treatments may not work well. The study will see if CRN04894 can safely and effectively help manage hormone levels by targeting the underlying cause of CAH.

Who is the study for?

Adults aged 18-75 with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency can join. They must be on stable glucocorticoid and, if needed, mineralocorticoid replacement therapy. Exclusions include major recent surgery, unstable heart conditions, certain drug use including testosterone or insulin changes for diabetes within the last six weeks.

What is being tested?

The trial is testing CRN04894's safety and effectiveness in managing CAH symptoms and how the body processes it. It's an open-label study where all participants know they're receiving CRN04894 at various doses to find the right balance between benefits and side effects.

What are the potential side effects?

Potential side effects of CRN04894 are not detailed here but generally could include reactions related to hormone levels adjustment such as fatigue, mood swings, weight changes or skin issues since it targets adrenal gland function.

Eligibility Criteria

Inclusion Criteria

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

Select...

I have been diagnosed with classic 21-hydroxylase deficiency.

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I take at least 15 mg of hydrocortisone daily.

Exclusion Criteria

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

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I have a condition that requires me to take steroids regularly.

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My hypothyroidism is not well-controlled with my current medication.

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I do not have any major health issues other than CAH.

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I haven't had a heart attack or severe heart-related issues in the last 3 months.

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I am not using testosterone, androgen supplements, aromatase inhibitors, or growth hormone.

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I have diabetes and started insulin or changed my dose significantly in the last 6 weeks.

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My diabetes is not well-managed, with an HbA1c level of 8.5% or higher.

Select...

My condition is not classic 21-hydroxylase deficiency CAH.

Select...

I have not taken dexamethasone in the last 30 days.

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

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Sequential DoseExperimental Treatment1 Intervention

Sequential, open-label, 12-week fixed-dose cohorts.

0 / 11Eligibility 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) due to 21-hydroxylase deficiency involve glucocorticoids and mineralocorticoids. Glucocorticoids provide cortisol replacement, which suppresses the overproduction of adrenocorticotropic hormone (ACTH) and reduces excessive adrenal androgen production. Mineralocorticoids help manage aldosterone deficiency, maintaining salt balance and blood pressure. These treatments are crucial for CAH patients to prevent adrenal crises, manage symptoms, and improve overall quality of life. The trial drug CRN04894 likely works by modulating adrenal steroidogenesis, similar to glucocorticoids, to address the enzyme deficiency.