Congenital Adrenal HyperplasiaPhase 2NCT07536269
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old
A Phase 2 study testing Crinecerfont in people with Congenital Adrenal Hyperplasia, run by Neurocrine Switzerland GmbH at 11 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 47 months
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- What is being tested?
- Crinecerfont
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 11 sitesUnited States
- Study dates
- 1 Jul 2026to Mar 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 47 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
11 sites in 1 country. Contact the study team or a site near you to ask about taking part.
11 sites in 1 country
United States11 sites, 11 recruiting
- Recruiting
Neurocrine Clinical Site
Ann Arbor, Michigan, 48109
- Recruiting
Neurocrine Clinical Site
Atlanta, Georgia, 30329
- Recruiting
Neurocrine Clinical Site
Aurora, Colorado, 80045
- Recruiting
Neurocrine Clinical Site
Chicago, Illinois, 60618
- Recruiting
Neurocrine Clinical Site
Dallas, Texas, 75235
- Recruiting
Neurocrine Clinical Site
Hartford, Connecticut, 06106
- Recruiting
Neurocrine Clinical Site
Indianapolis, Indiana, 46202
- Recruiting
Neurocrine Clinical Site
Minneapolis, Minnesota, 55454
- Recruiting
Neurocrine Clinical Site
Philadelphia, Pennsylvania, 19104
- Recruiting
Neurocrine Clinical Site
San Francisco, California, 94143
- Recruiting
Neurocrine Clinical Site
Seattle, Washington, 98101
About this study
Official title: 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
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.
- Conditions
- Congenital Adrenal Hyperplasia
- Being tested
- Crinecerfont
- Keywords
- Congenital Adrenal Hyperplasia, CAH, Crinecerfont, Pediatric
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CrinecerfontDrug
Oral solution
| Study group | Role | What they receive |
|---|---|---|
| Crinecerfont | Experimental | Participants with CAH will receive crinecerfont. Receives: Drug: Crinecerfont |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Measured: Day 1 up to 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2026
- Study start1 Jul 2026
- Primary completionMar 2028 (estimated)
- Study completionMar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Neurocrine Switzerland GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Development Lead, Study director, Neurocrine Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07536269
- Sponsor's protocol ID
- NBI-74788-CAH2032
- Status last verified
- Sep 2026
- First submitted
- 10 Apr 2026
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07536269
- Acronym / sponsor ID
- NBI-74788-CAH2032
- Conditions
- Congenital Adrenal Hyperplasia
- Keywords
- Congenital Adrenal Hyperplasia, CAH, Crinecerfont, Pediatric
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Neurocrine Switzerland GmbHIndustry (pharma, biotech, devices)
- Study start
- 1 Jul 2026 (actual)
- Primary completion
- Mar 2028 (estimated)
- Study completion
- Mar 2028 (estimated)
- First posted
- 17 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Crinecerfont | Experimental | Drug: CrinecerfontParticipants with CAH will receive crinecerfont. |
Who may qualify
- Ages
- 3 to 47 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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 (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CrinecerfontOral solution | Drug | Crinecerfont |
Primary outcome
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Time frame: Day 1 up to 28 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Clinical Development Lead study director
Neurocrine Biosciences
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Crinecerfont in people with Congenital Adrenal Hyperplasia, run by Neurocrine Switzerland GmbH at 11 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Neurocrine Medical Information Call Center (1-877-641-3461, medinfo@neurocrine.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-emergent Adverse Events (TEAE) (time frame: Day 1 up to 28 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026.