Congenital Adrenal HyperplasiaPhase 2NCT07187375
Pharmacokinetics, Safety and Tolerability of Crinecerfont in Participants With Congenital Adrenal Hyperplasia Who Are Less Than 2 Years Old
A Phase 2 study testing Crinecerfont in people with Congenital Adrenal Hyperplasia, run by Neurocrine Switzerland GmbH at 3 sites in Germany.
Trial 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). Read more about this study
- Who is it for?
- Ages 0 years to 23 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
- 7 participants
- Where
- 3 sitesGermany
- Study dates
- 30 Sep 2025to 4 Oct 2029
- 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 0 years to 23 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
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
3 sites in 1 country.
Germany3 sites
Neurocrine Clinical Site
Berlin, 13353
Neurocrine Clinical Site
Düsseldorf, 40225
Neurocrine Clinical Site
Heidelberg, 69120
About this study
Official title: 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
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).
- Conditions
- Congenital Adrenal Hyperplasia
- Being tested
- Crinecerfont
- Keywords
- Congenital Adrenal Hyperplasia, CAH, Crinecerfont
- 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 during an initial 14-day treatment period, followed by an optional 36-month open-label extension (OLE). Receives: Drug: Crinecerfont |
What is being measured
The primary outcome is the main question the study is designed to answer.
Plasma Concentration of Crinecerfont
Measured: Days 7 and 15
Secondary outcomes (1)
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Measured: Up to Month 37
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2025
- Study start30 Sep 2025
- Primary completion6 Sep 2029 (estimated)
- Study completion4 Oct 2029 (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
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Switzerland GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07187375
- Sponsor's protocol ID
- NBI-74788-CAH2011
- Other IDs
- 2024-514127-42-00
- Status last verified
- Aug 2026
- First submitted
- 18 Sep 2025
- Last updated
- 28 Aug 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 28 Aug 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 Pharmacokinetics, Safety, Tolerability, and Pharmacodynamics of Crinecerfont in Pediatric Subjects 0 to <2 Years of Age With Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT07187375
- Acronym / sponsor ID
- NBI-74788-CAH2011
- Conditions
- Congenital Adrenal Hyperplasia
- Keywords
- Congenital Adrenal Hyperplasia, CAH, Crinecerfont
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Neurocrine Switzerland GmbHIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2025 (actual)
- Primary completion
- 6 Sep 2029 (estimated)
- Study completion
- 4 Oct 2029 (estimated)
- First posted
- 23 Sep 2025 (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 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).
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 during an initial 14-day treatment period, followed by an optional 36-month open-label extension (OLE). |
Who may qualify
- Ages
- 0 years to 23 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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 (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
Plasma Concentration of Crinecerfont
Time frame: Days 7 and 15
1 secondary outcome
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Up to Month 37
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 28 Aug 2026.
- Individual participant dataSharing planned: No
Related trials for Congenital Adrenal Hyperplasia
RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Natural History Study of Patients With Excess Androgen
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
RecruitingPhase 3
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
Mild Autonomous Cortisol Secretion (MACS)
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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
3 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 3 sites in Germany.
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 3 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: under way, not looking for participants, as of the sponsor's last update on 28 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Germany. 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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Plasma Concentration of Crinecerfont (time frame: Days 7 and 15). 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 28 Aug 2026.