100 Participants Needed

Tildacerfont for Congenital Adrenal Hyperplasia

Recruiting at 79 trial locations
DM
NR
A
CT
KL
PW
Overseen ByPamela Wedel
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Spruce Biosciences
Must be taking: Glucocorticoids, Mineralocorticoids
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

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.

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.12345

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.13467

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.12468

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).

Research Team

RN

Ron Newfield, M.D

Principal Investigator

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

Eligibility Criteria

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

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

Treatment Details

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.
Participant Groups
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

Find a Clinic Near You

Who Is Running the Clinical Trial?

Spruce Biosciences

Lead Sponsor

Trials
6
Recruited
330+

References

Subjective health status in men and women with congenital adrenal hyperplasia: a population-based survey in Norway. [2022]
Psychological stress, emotions, and quality of life in men and women with congenital adrenal hyperplasia. [2022]
Recent advances in diagnosis, treatment, and outcome of congenital adrenal hyperplasia due to 21-hydroxylase deficiency. [2018]
Tildacerfont in Adults With Classic Congenital Adrenal Hyperplasia: Results from Two Phase 2 Studies. [2022]
Ovarian suppression reduces clinical and endocrine expression of late-onset congenital adrenal hyperplasia due to 21-hydroxylase deficiency. [2019]
Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia. [2023]
Clinical advances in the pharmacotherapy of congenital adrenal hyperplasia. [2022]
Inadequate cortisol response to the tetracosactide (Synacthen®) test in non-classic congenital adrenal hyperplasia: an exception to the rule? [2016]
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