Crinecerfont for Congenital Adrenal Hyperplasia

Not currently recruiting at 49 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called crinecerfont for children with classic congenital adrenal hyperplasia (CAH), a condition caused by a specific enzyme deficiency. Researchers aim to determine if crinecerfont is safe and effective compared to a placebo, which resembles the treatment but lacks the active medicine. The trial is ideal for children diagnosed with CAH, who have high levels of certain hormones (androgens), and are currently on stable steroid medication. Participants will first receive either the treatment or placebo for about seven months, followed by the real treatment for six more months. As a Phase 3 trial, this study represents the final step before FDA approval, offering a chance to contribute to a potentially groundbreaking treatment for CAH.

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 there any evidence suggesting that crinecerfont is likely to be safe for humans?

Research has shown that crinecerfont has been generally well-tolerated in past studies. In trials with adults, crinecerfont managed hormone levels without major safety issues. Independent experts closely monitored safety, emphasizing a strong focus on participant well-being.

Although this trial is in a late stage, providing extensive safety data, individual experiences may vary. Researchers will closely monitor participants throughout the study to ensure their safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Crinecerfont is unique because it targets the root cause of Congenital Adrenal Hyperplasia (CAH) by blocking a specific receptor involved in the hormonal imbalance that characterizes the condition. Unlike traditional treatments that primarily involve hormone replacement therapies like glucocorticoids and mineralocorticoids, Crinecerfont offers a novel mechanism of action, potentially reducing the need for high doses of steroids and their associated side effects. This new approach could lead to more balanced hormone levels and improved quality of life for patients with CAH. Researchers are excited about Crinecerfont's potential to offer a more targeted and effective treatment option for managing this challenging condition.

What evidence suggests that crinecerfont might be an effective treatment for congenital adrenal hyperplasia?

Research has shown that crinecerfont, which participants in this trial may receive, effectively treats congenital adrenal hyperplasia (CAH) by lowering elevated hormone levels associated with the condition. In earlier studies, crinecerfont significantly reduced levels of 17OHP and androstenedione, hormones that can cause issues when elevated. Another study found that crinecerfont helps balance adrenal hormones in both children and adults with CAH. It also reduced the need for glucocorticoids, commonly used in CAH treatment. These findings suggest that crinecerfont could help manage symptoms and improve overall hormone balance in people with CAH. ^{1 6 7 8 9}

Who Is on the Research Team?

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12543942/

SUN-423 Efficacy of Crinecerfont in Reducing Androgen ... Conclusion: Crinecerfont effectively reduces 17OHP and androstenedione levels in both pediatric and adult CAH populations, offering a promising ...

2. nejm.org nejm.org/doi/full/10.1056/NEJMoa2404655

Phase 3 Trial of Crinecerfont in Pediatric Congenital ... The results of this trial showed that crinecerfont therapy reduced production of excess adrenal androgens, which allowed substantial and ...

3. prnewswire.com prnewswire.com/news-releases/neurocrine-biosciences-presents-one-year-data-showing-sustained-efficacy-of-crenessity-crinecerfont-in-adult-patients-at-endo-2025-302503650.html

Neurocrine Biosciences Presents One-Year Data Showing ... These one-year data show the lasting ability of CRENESSITY to effectively manage the ACTH and adrenal steroid imbalances in adults.

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38828955/

Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal ... Conclusions: Among patients with CAH, the use of crinecerfont resulted in a greater decrease from baseline in the mean daily glucocorticoid dose ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S2666396125000330

Review CRF1 receptor antagonists in congenital adrenal ... ... Crinecerfont demonstrated consistent results supporting its efficacy and safety. These studies showed significant reductions in ACTH, 17-OHP ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04806451?term=CRINECERFONT&viewType=Table&rank=3

Study Details | NCT04806451 | Global Safety and Efficacy ... This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 ...

7. nadf.us nadf.us/uploads/1/3/0/1/130191972/crinecerfont_in_pediatric_cah.pdf

Phase 3 Trial of Crinecerfont in Pediatric Congenital ... 2024; baseline and key efficacy and safety data were presented at the annual meeting of the Endocrine Society in Boston, June. 1–4, 2024.

8. neurocrine.gcs-web.com neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-presents-one-year-data-showing-sustained

Neurocrine Biosciences Presents One-Year Data Showing ... "These one-year data show the lasting ability of CRENESSITY to effectively manage the ACTH and adrenal steroid imbalances in adults while ...

9. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/34653252/

Crinecerfont Lowers Elevated Hormone Markers in Adults ... Crinecerfont treatment for 14 days lowered ACTH and afforded clinically meaningful reductions of elevated 17OHP, androstenedione, testosterone (women), or ...