Crinecerfont for infants under 2 with classic congenital adrenal hyperplasia
A Phase 2, Open-Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Pharmacodynamics of Crinecerfont in Pediatric Subjects 0 to <2 Years of Age With Congenital Adrenal Hyperplasia
This trial will test crinecerfont in infants under 2 with classic CAH to see how the drug is processed by the body and whether it is safe and tolerable.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 6 (estimated) |
| Ages | 0 Years to 23 Months |
| Sex | All |
| Sponsor | Neurocrine Biosciences Industry-sponsored |
| Locations | 3 sites (Berlin and 2 other locations) |
| Trial ID | NCT07187375 on ClinicalTrials.gov |
What this trial studies
This Phase 2 interventional trial gives crinecerfont to infants aged 0 to under 2 years who have classic CAH due to 21‑hydroxylase deficiency and are on stable hydrocortisone (and fludrocortisone if needed). Investigators will collect timed blood samples to measure pharmacokinetics and will monitor safety, tolerability, vital signs, and laboratory values. Participants will attend regular clinic visits at Neurocrine clinical sites in Germany for dosing and assessments. The study aims to define how infants handle the drug and to document any adverse effects to inform dosing in this age group.
Who should consider this trial
Good fit: Infants aged 0 to under 2 years with a confirmed diagnosis of classic CAH due to 21‑hydroxylase deficiency who are on a clinically stable regimen of hydrocortisone (and fludrocortisone if applicable) are ideal candidates.
Not a fit: Children with non‑21‑hydroxylase forms of CAH, those requiring other chronic oral steroid therapy, or those with other significant medical conditions are unlikely to benefit or be eligible.
Why it matters
Potential benefit: If successful, crinecerfont could improve hormone control and potentially reduce the need for higher steroid doses in infants with CAH.
How similar studies have performed: Earlier-phase trials in older children and adults with CAH have shown promising reductions in ACTH and androgen markers with crinecerfont, so this trial extends prior work into infants.
Eligibility criteria
Show full inclusion / exclusion criteria
Key Inclusion Criteria: * Be a female or male between 0 to \<2 years of age at screening. * Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD). * Be on a clinically stable regimen of hydrocortisone (and fludrocortisone, if applicable) treatment. Key Exclusion Criteria: * Have a known or suspected diagnosis of any of the other forms of classic CAH. * Have any condition besides CAH that requires chronic daily therapy with orally administered steroids. * Have any other clinically significant medical condition or chronic disease. Note: Other protocol-defined inclusion and exclusion criteria may apply.
Where this trial is running
Berlin and 2 other locations
- Neurocrine Clinical Site — Berlin, Germany (Recruiting)
- Neurocrine Clinical Site — Düsseldorf, Germany (Recruiting)
- Neurocrine Clinical Site — Heidelberg, Germany (Recruiting)
Study contacts
- Study coordinator: Neurocrine Medical Information Call Center
- Email: medinfo@neurocrine.com
- Phone: 1-877-641-3461
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.