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, hypokalemia, and fluid retention. Tildacerfont, a CRF1 receptor antagonist, is being studied for its safety and efficacy, but specific side effects are not detailed in the provided context. Generally, CRF1 receptor antagonists may have side effects such as gastrointestinal disturbances, headache, and fatigue.

What prior FDA approvals exist?

Congenital Adrenal Hyperplasia (CAH) is typically treated with glucocorticoids such as hydrocortisone, prednisone, or dexamethasone to replace deficient cortisol and suppress excess androgen production. Mineralocorticoids like fludrocortisone are also used to manage salt-wasting forms of CAH. While these treatments are standard, Tildacerfont represents a novel approach as a CRF1 receptor antagonist, aiming to reduce adrenocorticotropic hormone (ACTH) levels and subsequently lower androgen production. As of now, there are no FDA-approved CRF1 receptor antagonists specifically for CAH, making Tildacerfont a potentially pioneering treatment in this category.

What other types of research exist?

Promising novel alternatives to Tildacerfont for CAH patients include gene therapy approaches, such as CRISPR-Cas9 mediated gene editing, and new enzyme inhibitors targeting 21-hydroxylase deficiency. Additionally, advancements in modified-release formulations of glucocorticoids and mineralocorticoids are being explored to improve hormonal balance and reduce side effects.

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Corticosteroid

Tildacerfont for Congenital Adrenal Hyperplasia

Phase 2

Recruiting

Research Sponsored by Spruce Biosciences

Eligibility Criteria Checklist

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

Must have

Male and female subjects aged 2 to 17 years

Diagnosis of CAH due to 21-hydroxylase deficiency (OHD) and/or elevated 17- hydroxyprogesterone (OHP) requiring ongoing GC replacement since diagnosis

Must not have

CAH not due to 21-OHD

History of bilateral adrenalectomy or hypopituitarism

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4 weeks or 12 weeks

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a medication called tildacerfont to see if it is safe and effective for people with Congenital Adrenal Hyperplasia (CAH). The treatment involves taking the medication for a period of time, followed by a safety check. Tildacerfont aims to help balance hormones that are not properly regulated in people with CAH. Tildacerfont has been studied for its effectiveness in adults with Congenital Adrenal Hyperplasia.

Who is the study for?

This trial is for children aged 2 to 17 with Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. They must have been on a stable dose of glucocorticoid replacement for at least one month. It's not suitable for those without this specific CAH type, who've had both adrenal glands removed, have unstable medical conditions, are pregnant or nursing females, or have bleeding disorders.

What is being tested?

The study tests the safety and effectiveness of Tildacerfont in treating pediatric patients with CAH. This medication could potentially help manage hormone levels in these young patients.

What are the potential side effects?

While the trial aims to establish the side effects of Tildacerfont in children with CAH, potential risks may include hormonal imbalances, growth issues, and typical drug-related reactions like nausea or rashes.

Eligibility Criteria

Inclusion Criteria

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

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I am between 2 and 17 years old.

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I have CAH and need ongoing hormone therapy since my diagnosis.

Exclusion Criteria

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

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My adrenal hyperplasia is not caused by 21-hydroxylase deficiency.

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I have had surgery to remove both adrenal glands or have a condition affecting my pituitary gland.

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I am not pregnant or nursing.

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I have a history of bleeding disorders.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4 weeks or 12 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~4 weeks or 12 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 weeks or 12 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of participants with Treatment-emergent adverse event (TEAE) as assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

Secondary study objectives

Proportion of participants who achieve a reduction in androstenedione (A4) or reduction in glucocorticoid (GC) dosing

Proportion of participants with elevated baseline A4 who achieve a reduction in A4

Proportion of participants with elevated baseline A4 who achieve a reduction in A4 who achieve A4 normalization

+1 more

Awards & Highlights

No Placebo-Only Group

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