Active — Not RecruitingPhase 2

Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old

Germany7 participantsStarted 2025-09-30

Plain-language summary

The main objective for this study is to evaluate the pharmacokinetics (PK) of crinecerfont in pediatric participants 0 to \<2 years of age with congenital adrenal hyperplasia (CAH).

Who can participate

Age range

0 Years – 23 Months

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Plasma Concentration of Crinecerfont

Timeframe: Days 7 and 15

Trial details

NCT IDNCT07187375

SponsorNeurocrine Biosciences

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2029-09-06

View on ClinicalTrials.gov More Congenital Adrenal Hyperplasia trials

Who can participate

Age range

0 Years – 23 Months

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.