What side effects are associated with this type of intervention?

Common treatments for Congenital Adrenal Hyperplasia (CAH) include glucocorticoids and mineralocorticoids. Glucocorticoids can cause side effects such as weight gain, osteoporosis, hypertension, diabetes, and increased risk of infections. Mineralocorticoids may lead to hypertension, edema, and electrolyte imbalances. Tildacerfont, a CRF1 Receptor Antagonist, is being studied for its potential to reduce the need for high doses of glucocorticoids. While specific side effects of Tildacerfont are not detailed, CRF1 Receptor Antagonists in general may cause gastrointestinal disturbances, dizziness, and fatigue.

What prior FDA approvals exist?

Congenital Adrenal Hyperplasia (CAH) is commonly treated with glucocorticoids to replace deficient cortisol and suppress excess androgen production. The FDA has approved several glucocorticoids, such as hydrocortisone, prednisone, and dexamethasone, for this purpose. Mineralocorticoids like fludrocortisone are also used to manage salt-wasting forms of CAH. While Tildacerfont, a CRF1 receptor antagonist, is being studied for its potential to reduce glucocorticoid doses, there are no current FDA-approved CRF1 receptor antagonists specifically for CAH treatment.

What other types of research exist?

Promising novel alternatives to Tildacerfont for CAH patients in 2024 may include other CRF1 receptor antagonists, novel steroidogenesis inhibitors, and emerging gene therapies. Specific names of these alternatives are not provided in the current context, but ongoing research in these areas is likely to yield new treatment options.

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Tildacerfont for Congenital Adrenal Hyperplasia

Phase 2

Waitlist Available

Led By Ron Newfield, M.D

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 over 18 years old, inclusive

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Summary

This trial is testing Tildacerfont to see if it can help adults with classic CAH reduce their steroid doses. The study will initially compare Tildacerfont to another treatment for several months, then allow all participants to use Tildacerfont for an extended period, with an optional longer-term extension. The goal is to find a safer way to manage CAH by lowering the need for high steroid doses. Tildacerfont has been studied in earlier trials for adults with classic congenital adrenal hyperplasia (CAH).

Who is the study for?

This trial is for adults over 18 with classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency. It's not open to those with other forms of CAH, a history of bilateral adrenalectomy, or hypopituitarism.

What is being tested?

The study tests if Tildacerfont can reduce the need for high doses of glucocorticoids in CAH patients. Participants will receive either Tildacerfont or a placebo and may continue treatment up to 240 weeks.

What are the potential side effects?

While specific side effects are not listed here, participants should be aware that they might experience unexpected reactions from taking Tildacerfont or the placebo during the trial.

Eligibility Criteria

Inclusion Criteria

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

Select...

I am over 18 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 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

Proportion of subjects who can reduce GC dose at Week 24

Secondary study objectives

Change in the median cumulative HCe dose in subjects with CAH

Effectiveness in reducing cardiovascular risk in subjects with CAH

Percentage change in GC use in subjects with CAH

Other study objectives

Change in acne in patients with CAH with acne at baseline

Change in hirsutism in female CAH subjects with hirsutism at baseline

Incidence of Adverse Events and Serious Adverse Events (safety analysis)

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Tildacerfont GroupExperimental Treatment1 Intervention

Tildacerfont administered daily via oral tablet for 24 weeks at dose level 1; followed by open label tildacerfont for 52 weeks

Group II: PlaceboPlacebo Group1 Intervention

Placebo administered daily via oral tablet for 24 weeks; followed by open label tildacerfont for 52 weeks

0 / 1Eligibility 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 glucocorticoids and mineralocorticoids. Glucocorticoids work by replacing cortisol, a hormone that CAH patients cannot produce adequately, thereby reducing the overproduction of androgens and preventing adrenal crises. Mineralocorticoids help maintain salt balance and blood pressure, which are often disrupted in CAH. Tildacerfont, a CRF1 receptor antagonist, is being studied for its potential to reduce the need for high doses of glucocorticoids by inhibiting the CRF1 receptor, which plays a role in the stress response and adrenal hormone production. This is significant for CAH patients as it may help manage the condition with fewer side effects associated with long-term high-dose glucocorticoid therapy.