Tildacerfont for Congenital Adrenal Hyperplasia

Recruiting at 17 trial locations

Overseen ByClinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Spruce Biosciences

Must be taking: Gc replacement

Disqualifiers: Adrenalectomy, Hypopituitarism, Bleeding disorders, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

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 on the Research Team?

Will Charlton, MD

Principal Investigator

Spruce Biosciences

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I have been on a stable dose of glucocorticoid replacement for at least 1 month.

I am between 2 and 17 years old.

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

Exclusion Criteria

I do not have any unstable or serious long-term health conditions.

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

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

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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral Tildacerfont daily for 4 or 12 consecutive weeks

4 or 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

Tildacerfont

Trial Overview 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.

How Is the Trial Designed?

9Treatment groups

Experimental Treatment

Group I: Cohort 9: Treatment with TildacerfontExperimental Treatment1 Intervention