Crinecerfont for Congenital Adrenal Hyperplasia

(CAHtalyst Trial)

Not currently recruiting at 72 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, crinecerfont, to determine its effectiveness in helping adults with classic Congenital Adrenal Hyperplasia (CAH), a condition caused by a missing enzyme that leads to hormone imbalances. Participants will receive either crinecerfont or a placebo for 24 weeks, followed by crinecerfont for at least a year to assess its long-term effects. This trial suits adults officially diagnosed with classic CAH who are on a stable steroid regimen. As a Phase 3 trial, it represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

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.

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

Research has shown that crinecerfont has been safe in earlier studies. In one study with children, researchers found no major safety issues during a 28-week treatment period, indicating that the treatment is generally well-tolerated. Another study with adults also reported no significant safety concerns over a 24-week period. These results are encouraging and suggest that crinecerfont is safe for humans, at least in the short term. However, as with any treatment, monitoring for side effects remains important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for CAH?

Crinecerfont is unique because it targets the root of Congenital Adrenal Hyperplasia (CAH) by blocking the CRF1 receptor, which is a different approach from standard treatments like glucocorticoids and mineralocorticoids. This new mechanism allows Crinecerfont to potentially manage CAH symptoms with fewer side effects and complications related to long-term steroid use, such as growth suppression and metabolic issues. Researchers are excited about Crinecerfont because it offers a novel way to balance hormone production more effectively, potentially improving quality of life for those living with CAH.

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

Research shows that crinecerfont, which participants in this trial may receive, can help manage hormone imbalances in adults with congenital adrenal hyperplasia (CAH). Studies have found that crinecerfont lowers the production of excess adrenal androgens, the hormones responsible for CAH symptoms. Patients taking crinecerfont required lower doses of glucocorticoids, the steroid hormones often used to treat CAH. This indicates that crinecerfont not only helps balance hormones but also reduces the need for other medications. Overall, crinecerfont has consistently improved hormone regulation in people with CAH. ^{1 2 4 6 7}

Who Is on the Research Team?

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1. 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.

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. 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 ...

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

Review CRF1 receptor antagonists in congenital adrenal ... Phase 2b and Phase 3 clinical trials of CRF1 antagonists involving Crinecerfont demonstrated consistent results supporting its efficacy and safety.

5. 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 ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11309900/

Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal ... In this phase 3 trial, adults with CAH were randomized (2:1) to crinecerfont or placebo for 24 weeks. Glucocorticoid treatment was maintained stable for 4 weeks ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04490915

Study Details | NCT04490915 | Global Safety and Efficacy ... This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 24 weeks in approximately 165 adult ...