Crinecerfont for Congenital Adrenal Hyperplasia

Recruiting at 44 trial locations

Overseen By Neurocrine Medical Information Call Center

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Neurocrine Biosciences

Must be taking: Steroids

Must not be taking: Corticotropin antagonists

Disqualifiers: Other CAH, Cancer, Arrhythmia, 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

103 Participants Needed

Trial Summary

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 steroid medications.

Is Crinecerfont safe for humans?

Crinecerfont has been studied in adolescents and adults with congenital adrenal hyperplasia, and it is generally considered safe for humans, as it helps reduce elevated adrenal androgens without major safety concerns reported in the studies. ^{1 2 3 4 5}

How is the drug Crinecerfont different 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 adrenal androgens in congenital adrenal hyperplasia, potentially reducing the need for high doses of glucocorticoids that are typically used. ^{1 2 6 7 8}

What is the purpose of this trial?

This trial is testing a medication called crinecerfont to help children with a hormone disorder called CAH. The study will compare crinecerfont to another treatment over several months. The goal is to see if crinecerfont can safely and effectively balance their hormone levels.

Research Team

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Eligibility Criteria

This trial is for pediatric patients with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. Participants must be on stable steroidal treatment, have high androgen levels, and agree to birth control if applicable. Exclusions include unstable medical conditions, cancer history unless cured, recent investigational drug use or significant blood loss, other CAH forms, known drug hypersensitivity, certain surgeries or chronic conditions.

Inclusion Criteria

Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit.

I have been diagnosed with 21-hydroxylase deficiency CAH.

Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study.

Exclusion Criteria

You are allergic to any corticotropin-releasing hormone antagonist.

Have received an investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.

I have been diagnosed with a form of classic congenital adrenal hyperplasia.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-Blind Placebo-Controlled Treatment

Participants receive either crinecerfont or placebo for 28 weeks

28 weeks

Open-Label Treatment

All participants receive crinecerfont for 24 weeks

24 weeks

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 study tests the effectiveness of Crinecerfont compared to a placebo in children with CAH over a period of 28 weeks. After this phase, all participants receive Crinecerfont for another 24 weeks. Those interested can join an open-label extension for about three years to further assess long-term effects.

Participant Groups

Group I: Crinecerfont Experimental Treatment 1 Intervention

Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.

Group II: Placebo Placebo Group 2 Interventions

Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.

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

Findings from Research

Modified-release hydrocortisone (MR-HC) provides a more physiological cortisol profile compared to conventional hydrocortisone (HC), with lower cortisol levels in the afternoon and night, which may improve treatment for congenital adrenal hyperplasia (CAH).

In a study of 14 patients, MR-HC resulted in better control of overnight adrenal androgens, but showed increased afternoon levels of certain hormones, indicating that a morning dose may be necessary for optimal management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pharmacokinetic and pharmacodynamic study of delayed- and extended-release hydrocortisone (Chronocort) vs. conventional hydrocortisone (Cortef) in the treatment of congenital adrenal hyperplasia. Verma, S., Vanryzin, C., Sinaii, N., et al.[2022]

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

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

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

Genotype/phenotype correlations in 538 congenital adrenal hyperplasia patients from Germany and Austria: discordances in milder genotypes and in screened versus prescreening patients. [2020]

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

Bone Age Maturation and Growth Outcomes in Young Children with CAH Treated with Hydrocortisone Suspension. [2022]

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

Potential advantage of N363S glucocorticoid receptor polymorphism in 21-hydroxylase deficiency. [2017]

7. France pubmed.ncbi.nlm.nih.gov

Congenital adrenal hyperplasia: New biomarkers and adult treatments. [2023]

8. England pubmed.ncbi.nlm.nih.gov

A pharmacokinetic and pharmacodynamic study of delayed- and extended-release hydrocortisone (Chronocort) vs. conventional hydrocortisone (Cortef) in the treatment of congenital adrenal hyperplasia. [2022]