ClinicalTrials.gov record NCT07536269

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Crinecerfont in Pediatric Participants 3 Months to <4 Years of Age With Classic Congenital Adrenal Hyperplasia

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.

Age3 months old to 4 years old

SexAll

LocationUnited States

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 2, open-label study gives oral crinecerfont to children aged 3 months to <4 years with classic congenital adrenal hyperplasia (CAH). The main goal is to assess safety and tolerability and to measure pharmacokinetics and pharmacodynamics. Participants are followed for adverse events and drug measurements up to 28 weeks.

What it is studying. The study is testing the safety and tolerability of crinecerfont in pediatric participants with classic CAH and is measuring how the drug is absorbed, processed, and affects the body (pharmacokinetics and pharmacodynamics).

What the researchers hope to learn. To evaluate the safety and tolerability of crinecerfont in children 3 months to <4 years with classic CAH and to characterize the drug's pharmacokinetics and pharmacodynamics in this age group.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to <4 years of age with CAH.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: Crinecerfont

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive crinecerfont as an oral solution and attend study visits for safety, tolerability, and pharmacokinetic/pharmacodynamic assessments. They will be monitored for treatment-emergent adverse events from Day 1 up to about 28 weeks. The registry does not list the exact visit schedule or the specific tests and procedures that will be done during visits.

How the study is structured. Phase 2, interventional (treatment) study. Open-label, single-group design: all participants receive oral crinecerfont (oral solution) with no placebo or comparison group and no masking. Participants are monitored over time for side effects and drug measurements.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on congenital adrenal hyperplasia

Clinical trials43 trials listed for congenital adrenal hyperplasiaEvery trial the registry lists for congenital adrenal hyperplasia, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in congenital adrenal hyperplasia researchLatest monthly update: September 2026. Includes: Bedtime hydrocortisone. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Jul 1, 2026
Primary completion: March 2028 (estimated)
Study completion: March 2028 (estimated)
Target enrollment: 20 participants (estimated)
Last updated on registry: Sep 16, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Neurocrine Switzerland GmbH

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Neurocrine Medical Information Call Center 📞 1-877-641-3461 ✉ medinfo@neurocrine.com

Participating sites (11)

Neurocrine Clinical SiteRecruiting
San Francisco, California, 94143 · United States
Neurocrine Clinical SiteRecruiting
Aurora, Colorado, 80045 · United States
Neurocrine Clinical SiteRecruiting
Hartford, Connecticut, 06106 · United States
Neurocrine Clinical SiteRecruiting
Atlanta, Georgia, 30329 · United States
Neurocrine Clinical SiteRecruiting
Chicago, Illinois, 60618 · United States
Neurocrine Clinical SiteRecruiting
Indianapolis, Indiana, 46202 · United States
Neurocrine Clinical SiteRecruiting
Ann Arbor, Michigan, 48109 · United States
Neurocrine Clinical SiteRecruiting
Minneapolis, Minnesota, 55454 · United States
Neurocrine Clinical SiteRecruiting
Philadelphia, Pennsylvania, 19104 · United States
Neurocrine Clinical SiteRecruiting
Dallas, Texas, 75235 · United States

Terms used on this page · explanations by MedicalUpdates.ai

stable dose: A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
before screening: The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
exclusion criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Key Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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