Congenital Adrenal HyperplasiaPhase 3NCT04806451
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
A Phase 3 study testing Crinecerfont in people with Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 46 sites in 10 countries.
Trial summary
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- 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?
- Yes, some participants may receive a placebo
- How many people
- 103 participants
- Where
- 46 sitesin 10 countries
- Study dates
- 25 Jun 2021to Aug 2027
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit.
- Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency.
- Be on a stable steroid regimen.
- Have elevated androgen levels.
- Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study.
Who cannot join
- Have a diagnosis of any of the other forms of classic CAH.
- Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
- Have a clinically significant unstable medical condition or chronic disease other than CAH.
- Have a history of cancer unless considered to be cured.
- Have a known history of clinically significant arrhythmia or abnormalities on electrocardiogram (ECG).
- Have a known hypersensitivity to any corticotropin-releasing hormone antagonist.
Show all 9Show fewer
- Have received an investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.
- Have current substance dependence or substance (drug) or alcohol abuse.
- Have had a significant blood loss or donated blood or blood products within 8 weeks prior to the study.
Trial locations and contacts
46 sites in 10 countries.
46 sites in 10 countries
United States22 sites
Neurocrine Clinical Site
Ann Arbor, Michigan, 48109
Neurocrine Clinical Site
Atlanta, Georgia, 30329
Neurocrine Clinical Site
Aurora, Colorado, 80045
Neurocrine Clinical Site
Birmingham, Alabama, 35233
Neurocrine Clinical Site
Boston, Massachusetts, 02115
Neurocrine Clinical Site
Dallas, Texas, 75235
Neurocrine Clinical Site
Hartford, Connecticut, 06106
Neurocrine Clinical Site
Indianapolis, Indiana, 46202
Neurocrine Clinical Site
Los Angeles, California, 90027
Neurocrine Clinical Site
Minneapolis, Minnesota, 55454
Neurocrine Clinical Site
New Hyde Park, New York, 11040
Neurocrine Clinical Site
New York, New York, 10065
Neurocrine Clinical Site
Oklahoma City, Oklahoma, 73104
Neurocrine Clinical Site
Orange, California, 92868
Neurocrine Clinical Site
Philadelphia, Pennsylvania, 19104
Neurocrine Clinical Site
Pittsburgh, Pennsylvania, 15224
Neurocrine Clinical Site
San Diego, California, 92123
Neurocrine Clinical Site
San Francisco, California, 94158
Neurocrine Clinical Site
Seattle, Washington, 98105
Neurocrine Clinical site
St Louis, Missouri, 63104
Neurocrine Clinical Site
Tulsa, Oklahoma, 74135
Neurocrine Clinical Site
Washington D.C., District of Columbia, 20010
France5 sites
Neurocrine Clinical Site
Angers, 49933
Neurocrine Clinical Site
Bordeau, 33076
Neurocrine Clinical Site
Le Kremlin-Bicêtre, 94270
Neurocrine Clinical Site
Paris, 75015
Neurocrine Clinical Site
Paris, 75019
Italy4 sites
Neurocrine Clinical Site
Bologna, 40138
Neurocrine Clinical Site
Milan, 20132
Neurocrine Clinical Site
Naples, 80131
Neurocrine Clinical Site
Roma, 00165
Canada3 sites
Neurocrine Clinical Site
Edmonton, Alberta, T6G 1C9
Neurocrine Clinical Site
Montreal, Quebec, H3T 1C5
Neurocrine Clinical Site
Vancouver, British Columbia, V6H 3V4
Germany3 sites
Neurocrine Clinical Site
Berlin, 13353
Neurocrine Clinical Site
Heidelberg, 69120
Neurocrine Clinical Site
Magdeburg, 39120
Belgium2 sites
Neurocrine Clinical Site
Brussels, 1200
Neurocrine Clinical Site
Ghent, 9000
Greece2 sites
Neurocrine Clinical Site
Athens, 115 27
Neurocrine Clinical Site
Athens, 11527
Poland2 sites
Neurocrine Clinical Site
Gdansk, 80-214
Neurocrine Clinical Site
Rzeszów, 35-301
Spain2 sites
Neurocrine Clinical Site
Barcelona, 08035
Neurocrine Clinical Site
Seville, 41013
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).
- Conditions
- Congenital Adrenal Hyperplasia
- Being tested
- Crinecerfont, Placebo
- Keywords
- classic congenital adrenal hyperplasia (CAH), 21-hydroxylase deficiency
- Medical subject headings
- Adrenal Hyperplasia, Congenital, Congenital adrenal hyperplasia due to 21 hydroxylase deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CrinecerfontDrug
Also called NBI-74788, Crenessity
CRF type 1 receptor antagonist
PlaceboDrug
Non-active dosage form
| Study group | Role | What they receive |
|---|---|---|
| Crinecerfont | Experimental | Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks. Receives: Drug: Crinecerfont |
| Placebo | Placebo comparator | Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks. Receives: Drug: Crinecerfont; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Serum Androstenedione at Week 4
Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
Measured: Baseline, Week 4
Secondary outcomes (6)
Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
Measured: Baseline, Week 4
Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28
Measured: Baseline, Week 28
Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28
Measured: Week 28
Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28
Measured: Baseline, Week 28
Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28
Measured: Baseline, Week 28
Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28
Measured: Baseline, Week 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2021
- Study start25 Jun 2021
- Primary completion10 Mar 2023
- Study completionAug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- More trials run by Neurocrine BiosciencesThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 103 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Development Lead, Study director, Neurocrine Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04806451
- Sponsor's protocol ID
- NBI-74788-CAH2006
- Other IDs
- 2020-004381-19, 2023-509170-33-00, 2023
- Status last verified
- Jan 2025
- First submitted
- 16 Mar 2021
- Last updated
- 5 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Sarafoglou K, Kim MS, Lodish M, Felner EI, Martinerie L, Nokoff NJ, Clemente M, Fechner PY, Vogiatzi MG, Speiser PW, Auchus RJ, Rosales GBG, Roberts E, Jeha GS, Farber RH, Chan JL; CAHtalyst Pediatric Trial Investigators. Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia. N Engl J Med. 2024 Aug 8;391(6):493-503. doi: 10.1056/NEJMoa2404655. Epub 2024 Jun 2.
PubMed 38828945
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment
- ClinicalTrials.gov ID
- NCT04806451
- Acronym / sponsor ID
- NBI-74788-CAH2006
- Conditions
- Congenital Adrenal Hyperplasia
- Keywords
- classic congenital adrenal hyperplasia (CAH), 21-hydroxylase deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 103 participants (actual)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 25 Jun 2021 (actual)
- Primary completion
- 10 Mar 2023 (actual)
- Study completion
- Aug 2027 (estimated)
- First posted
- 19 Mar 2021 (actual)
- Results
- Posted 5 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Crinecerfont | Experimental | Drug: CrinecerfontCrinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks. |
| Placebo | Placebo comparator | Drug: Crinecerfont, Drug: PlaceboPlacebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks. |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit.
- Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency.
- Be on a stable steroid regimen.
- Have elevated androgen levels.
- Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study.
Key exclusion criteria (9 listed)
- Have a diagnosis of any of the other forms of classic CAH.
- Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
- Have a clinically significant unstable medical condition or chronic disease other than CAH.
- Have a history of cancer unless considered to be cured.
- Have a known history of clinically significant arrhythmia or abnormalities on electrocardiogram (ECG).
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 |
|---|---|---|
| CrinecerfontAlso: NBI-74788, CrenessityCRF type 1 receptor antagonist | Drug | Crinecerfont, Placebo |
| PlaceboNon-active dosage form | Drug | Placebo |
Primary outcome
Change From Baseline in Serum Androstenedione at Week 4
Time frame: Baseline, Week 4
Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
6 secondary outcomes
Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
Time frame: Baseline, Week 4
Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28
Time frame: Baseline, Week 28
Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28
Time frame: Week 28
Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28
Time frame: Baseline, Week 28
Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28
Time frame: Baseline, Week 28
Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28
Time frame: Baseline, Week 28
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38828945Sarafoglou K, Kim MS, Lodish M, Felner EI, Martinerie L, Nokoff NJ, Clemente M, Fechner PY, Vogiatzi MG, Speiser PW, Auchus RJ, Rosales GBG, Roberts E, Jeha GS, Farber RH, Chan JL; CAHtalyst Pediatric Trial Investigators. Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia. N Engl J Med. 2024 Aug 8;391(6):493-503. doi: 10.1056/NEJMoa2404655. Epub 2024 Jun 2.
- 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. This study is not recruiting at the moment.
Clinical Development Lead study director
Neurocrine Biosciences
46 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Crinecerfont in people with Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 46 sites in 10 countries.
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 5 inclusion and 9 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 5 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in United States, France, Italy, Canada, Germany and 5 more countries. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Serum Androstenedione at Week 4 (time frame: Baseline, Week 4). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04806451, which also lists 1 linked publication.
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 5 Feb 2025.