Tildacerfont for Congenital Adrenal Hyperplasia

Recruiting at 79 trial locations

Overseen By Pamela Wedel

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Spruce Biosciences

Must be taking: Glucocorticoids, Mineralocorticoids

Disqualifiers: Other CAH, Adrenalectomy, Hypopituitarism, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

100 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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).

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it mentions that participants should have been on a stable dose of glucocorticoid and mineralocorticoid replacements for at least one month before screening. This suggests you may continue your current medications.

Is Tildacerfont safe for humans?

Tildacerfont has been studied in adults with congenital adrenal hyperplasia, and while the research focuses on its ability to reduce certain hormone levels, it does not provide detailed safety data. However, it is important to note that clinical trials are designed to monitor safety, and any significant safety concerns would typically be reported. ^{1 2 3 4 5}

How is the drug Tildacerfont unique in treating congenital adrenal hyperplasia?

Tildacerfont is unique because it targets the corticotropin-releasing factor type-1 receptor, which may help reduce excess androgen production and allow for lower doses of glucocorticoids, unlike traditional treatments that rely on high doses of glucocorticoids. ^{1 2 5 6 7}

What data supports the effectiveness of the drug Tildacerfont for treating Congenital Adrenal Hyperplasia?

Research shows that Tildacerfont may help reduce excess androgen (male hormone) production in people with Congenital Adrenal Hyperplasia, which could allow for lower doses of glucocorticoids (a type of steroid medication) that are usually needed for treatment. ^{1 3 5 6 8}

Who Is on the Research Team?

Ron Newfield, M.D

Principal Investigator

Rady Children's Hospital-San Diego and Professor of clinical pediatrics at UC San Diego School of Medicine.

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I am over 18 years old.

Exclusion Criteria

I have a form of CAH that is not due to 21-hydroxylase deficiency.

You have had surgery to remove both adrenal glands or you have a condition called hypopituitarism.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Double-blind, placebo-controlled treatment with Tildacerfont or placebo for 24 weeks

24 weeks

Regular visits as per trial protocol

Open-label Treatment

All subjects receive open-label Tildacerfont for 52 weeks to continue reducing steroid dose and observe long-term safety

52 weeks

Regular visits as per trial protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label Extension (optional)

Participants may opt into continuation of treatment long-term for up to 240 weeks

Up to 240 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Tildacerfont

Trial Overview 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.

How Is the Trial Designed?

Group I: Tildacerfont Group Experimental Treatment 1 Intervention

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

Group II: Placebo Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Spruce Biosciences

Lead Sponsor

Trials

6

Recruited

330+

Citations

1. England pubmed.ncbi.nlm.nih.gov

Subjective health status in men and women with congenital adrenal hyperplasia: a population-based survey in Norway. [2022]

2. Italy pubmed.ncbi.nlm.nih.gov

Psychological stress, emotions, and quality of life in men and women with congenital adrenal hyperplasia. [2022]

3. Germany pubmed.ncbi.nlm.nih.gov

Recent advances in diagnosis, treatment, and outcome of congenital adrenal hyperplasia due to 21-hydroxylase deficiency. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Tildacerfont in Adults With Classic Congenital Adrenal Hyperplasia: Results from Two Phase 2 Studies. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Ovarian suppression reduces clinical and endocrine expression of late-onset congenital adrenal hyperplasia due to 21-hydroxylase deficiency. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia. [2023]

7. England pubmed.ncbi.nlm.nih.gov

Clinical advances in the pharmacotherapy of congenital adrenal hyperplasia. [2022]

8. Switzerland pubmed.ncbi.nlm.nih.gov

Inadequate cortisol response to the tetracosactide (Synacthen®) test in non-classic congenital adrenal hyperplasia: an exception to the rule? [2016]