Crinecerfont safety testing in babies with classic CAH under 4 years old

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2 interventional trial evaluates safety, tolerability, and how the drug behaves in the body and its effects on the body’s response in pediatric participants aged 3 months to under 4 years with classic CAH due to 21-hydroxylase deficiency. Participants receive crinecerfont, with eligibility requiring a stable hydrocortisone dose for at least 4 weeks before screening. The study is conducted at Neurocrine Clinical Sites in San Francisco, Aurora, and Hartford to monitor safety and pharmacokinetics/pharmacodynamics in a young population.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

* Have a medically confirmed diagnosis of classic CAH (salt wasting or simple virilizing) due to 21-hydroxylase deficiency (21-OHD)
* Have received at least 3 months of hydrocortisone treatment before Day 1, with a stable dose for at least 4 weeks before screening.
* Have a body weight of at least 4.5 kilograms (kg) at screening.
* Have a newborn screen that is otherwise normal except for elevated 17-OHP or any other abnormality on newborn screen that was cleared upon evaluation by a pediatric specialist.

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 it's running

• Neurocrine Clinical Site
  San Francisco, California, United States Recruiting
• Neurocrine Clinical Site
  Aurora, Colorado, United States Recruiting
• Neurocrine Clinical Site
  Hartford, Connecticut, United States Recruiting
• Neurocrine Clinical Site
  Atlanta, Georgia, United States Recruiting
• Neurocrine Clinical Site
  Chicago, Illinois, United States Recruiting
• Neurocrine Clinical Site
  Indianapolis, Indiana, United States Recruiting

Study contacts

• Study coordinator: Neurocrine Medical Information Call Center
• Email: medinfo@neurocrine.com
• Phone: 1-877-641-3461

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.