Crinecerfont for Congenital Adrenal Hyperplasia

(CAHtalyst Trial)

Recruiting at 64 trial locations

Overseen By Neurocrine Medical Information Call Center

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Neurocrine Biosciences

Must be taking: Steroids

Disqualifiers: Other CAH forms, Cancer, Pregnancy, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

182 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new medication called crinecerfont in adults with a genetic condition that affects hormone production. The study aims to see if crinecerfont can help balance their hormone levels and improve their symptoms. Participants will take the medication for several months, with an option to continue longer.

Will I have to stop taking my current medications?

The trial requires participants to be on a stable steroid regimen, so you will not need to stop taking your current steroids. The protocol does not specify about other medications.

What data supports the effectiveness of the drug Crinecerfont for treating congenital adrenal hyperplasia?

Research shows that Crinecerfont can lower elevated adrenal androgens (hormones) in people with congenital adrenal hyperplasia, which is a condition where the body produces too many male hormones due to a genetic issue. ^{1 2 3 4 5}

Is Crinecerfont safe for humans?

Crinecerfont has been studied in adolescents and adults with congenital adrenal hyperplasia, and while it helps reduce certain hormone levels, the safety data specific to humans is not detailed in the available research. ^{1 2 5 6 7}

How does the drug Crinecerfont differ from other treatments for congenital adrenal hyperplasia?

Crinecerfont is unique because it works by blocking the corticotropin-releasing factor type 1 receptor, which helps lower the excess production of adrenal androgens (hormones) in people with congenital adrenal hyperplasia. This is different from the usual treatment with high doses of glucocorticoids, which can have more side effects. ^{1 2 3 6 8}

Research Team

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Eligibility Criteria

Adults with classic Congenital Adrenal Hyperplasia (CAH) due to 21-hydroxylase deficiency can join. They must be on stable CAH medication, agree to use contraception if they can have children, and follow study rules. Excluded are those with unstable conditions, hypersensitivity to similar drugs, certain medical histories like cancer or chronic glucocorticoid therapy needs.

Inclusion Criteria

I am on a consistent dose of steroids for congenital adrenal hyperplasia.

Participants of childbearing potential must agree to use an acceptable method of contraception during the study.

I have been diagnosed with classic 21-hydroxylase deficiency CAH.

Exclusion Criteria

Are pregnant.

You are allergic to any corticotropin releasing hormone antagonists.

I have had cancer in the past but am considered cured.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive crinecerfont or placebo for 24 weeks in a double-blind, placebo-controlled period

24 weeks

Active Treatment

Participants receive active treatment with crinecerfont for at least 1 year

1 year

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

Approximately 3 years

Treatment Details

Interventions

• Crinecerfont
• Placebo

Trial Overview The trial is testing Crinecerfont against a placebo for CAH over 24 weeks in a double-blind setup where neither the participants nor the researchers know who gets what. After this phase, there's an open-label period where everyone receives Crinecerfont for up to three years.

Participant Groups

Group I: Crinecerfont Experimental Treatment 1 Intervention

Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.

Group II: Placebo Placebo Group 2 Interventions

Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.

Who Is Running the Clinical Trial?

Neurocrine Biosciences

Lead Sponsor

Trials

78

Recruited

6,600+

Kyle W. Gano

Neurocrine Biosciences

Chief Executive Officer since 2024

PhD in Pharmacology

Dr. Sanjay Keswani

Neurocrine Biosciences

Chief Medical Officer

MD

References

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

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

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

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

3. England pubmed.ncbi.nlm.nih.gov

Congenital adrenal hyperplasia in adults. [2016]

4. England pubmed.ncbi.nlm.nih.gov

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

5. Japan pubmed.ncbi.nlm.nih.gov

[A clinical study of congenital adrenal hyperplasia]. [2013]

6. England pubmed.ncbi.nlm.nih.gov

Differential effects of hydrocortisone, prednisone, and dexamethasone on hormonal and pharmacokinetic profiles: a pilot study in children with congenital adrenal hyperplasia. [2020]

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

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

8. India pubmed.ncbi.nlm.nih.gov

Left ventricular failure due to a rare variant of congenital adrenal hyperplasia. [2021]