Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)

April 20, 2026 updated by: Neurocrine Biosciences

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult 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 24 weeks in approximately 165 adult participants with classic CAH due to 21-hydroxylase deficiency. The study consists of a 24-week randomized, double-blind, placebo-controlled period, followed by 1 year of active 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 20 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 Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
182

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Graz, Austria, 8036
        • Neurocrine Clinical Site
      • Vienna, Austria, 1090
        • Neurocrine Clinical Site
  • Belgium
      • Brussels, Belgium, 1070
        • Neurocrine Clinical Site
      • Leuven, Belgium, 3000
        • Neurocrine Clinical Site
  • Bulgaria
      • Sofia, Bulgaria, 1431
        • Neurocrine Clinical Site
      • Sofia, Bulgaria, 1606
        • Neurocrine Clinical Site
  • Canada
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 2Y9
        • Neurocrine Clinical Site
  • Czechia
      • Hradec Králové, Czechia, 50005
        • Neurocrine Clinical Site
  • France
      • Angers, France, 49933
        • Neurocrine Clinical Site
      • Grenoble, France, 38700
        • Neurocrine Clinical Site
      • Le Kremlin-Bicêtre, France, 94270
        • Neurocrine Clinical Site
      • Nantes, France, 44093
        • Neurocrine Clinical Site
      • Paris, France, 75651
        • Neurocrine Clinical Site
      • Paris, France, 75679
        • Neurocrine Clinical Site
  • Germany
      • Dresden, Germany, 01307
        • Neurocrine Clinical Site
      • Essen, Germany, 45147
        • Neurocrine Clinical Site
      • Frankfurt, Germany, 60590
        • Neurocrine Clinical Site
      • Leipzig, Germany, 04103
        • Neurocrine Clinical Site
      • Munich, Germany, 80336
        • Neurocrine Clinical Site
  • Greece
      • Athens, Greece, 106 76
        • Neurocrine Clinical Site
      • Athens, Greece, 115 27
        • Neurocrine Clinical Site
      • Athens, Greece, 11527
        • Neurocrine Clinical Site
      • Thessaloniki, Greece, 54642
        • Neurocrine Clinical Site
  • Israel
      • Afula, Israel, 1834111
        • Neurocrine Clinical Site
      • Beersheba, Israel, 8410101
        • Neurocrine Clinical Site
      • Petah Tikva, Israel, 4941480
        • Neurocrine Clinical Site
      • Tel Aviv, Israel, 64239
        • Neurocrine Clinical Site
  • Italy
      • Ancona, Italy, 60126
        • Neurocrine Clinical Site
      • Bologna, Italy, 40138
        • Neurocrine Clinical Site
      • Florence, Italy, 50139
        • Neurocrine Clinical Site
      • Messina, Italy, 98125
        • Neurocrine Clinical Site
      • Milan, Italy, 20132
        • Neurocrine Clinical Site
      • Milan, Italy, 20149
        • Neurocrine Clinical Site
      • Naples, Italy, 80131
        • Neurocrine Clinical Site
      • Padova, Italy, 35128
        • Neurocrine Clinical Site
      • Roma, Italy, 00161
        • Neurocrine Clinical Site
  • Netherlands
      • Leiden, Netherlands, 2333
        • Neurocrine Clinical Site
  • Poland
      • Krakow, Poland, 31-011
        • Neurocrine Clinical Site
      • Poznan, Poland, 60-355
        • Neurocrine Clinical Site
      • Warsaw, Poland, 01-809
        • Neurocrine Clinical Site
  • Portugal
      • Porto, Portugal, 4200-319
        • Neurocrine Clinical Site
  • Serbia
      • Belgrade, Serbia, 11000
        • Neurocrine Clinical Site
  • Spain
      • Madrid, Spain, 28034
        • Neurocrine Clinical Site
      • Seville, Spain, 41013
        • Neurocrine Clinical Site
  • Sweden
      • Gothenburg, Sweden, 41345
        • Neurocrine Clinical Site
      • Stockholm, Sweden, 17176
        • Neurocrine Clinical Site
  • United Kingdom
      • Cardiff, United Kingdom, CF14 4HH
        • Neurocrine Clinical Site
      • London, United Kingdom, WC1B 5EH
        • Neurocrine Clinical Site
      • Manchester, United Kingdom, M20 4BX
        • Neurocrine Clinical Site
      • Salford, United Kingdom, M6 8HD
        • Neurocrine Clinical Site
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Neurocrine Clinical Site
      • San Diego, California, United States, 92123
        • Neurocrine Clinical Site
      • San Francisco, California, United States, 94143
        • Neurocrine Clinical Site
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Neurocrine Clinical Site
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Neurocrine Clinical Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Neurocrine Clinical Site
    • Maryland
      • Bethesda, Maryland, United States, 20982
        • Neurocrine Clinical Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Neurocrine Clinical Site
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Neurocrine Clinical Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Neurocrine Clinical Site
      • Rochester, Minnesota, United States, 55905
        • Neurocrine Clinical Site
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Neurocrine Clinical Site
    • New York
      • Great Neck, New York, United States, 11021
        • Neurocrine Clinical Site
      • New York, New York, United States, 10029
        • Neurocrine Clinical Site
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157
        • Neurocrine Clinical Site
    • Oklahoma
      • Tulsa, Oklahoma, United States, 74135
        • Neurocrine Clinical Site
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Neurocrine Clinical Site
      • Pittsburgh, Pennsylvania, United States, 15224
        • Neurocrine Clinical Site
    • Texas
      • Dallas, Texas, United States, 75390
        • Neurocrine Clinical Site
    • Washington
      • Seattle, Washington, United States, 98105
        • Neurocrine Clinical Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Be willing and able to adhere to the study procedures, including all requirements at the study center and return for the follow-up visit.
  2. Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency.
  3. Be on a stable steroid regimen.
  4. Participants of childbearing potential must agree to use an acceptable method of contraception during the study.

Exclusion Criteria:

  1. Have a diagnosis of any of the other known forms of classic CAH.
  2. Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.
  3. Have a clinically significant unstable medical condition or chronic disease other than CAH.
  4. Have a history of cancer unless considered cured.
  5. Are pregnant.
  6. Have a known history of clinically significant arrhythmia or abnormalities on ECG.
  7. Have a known hypersensitivity to any corticotropin releasing hormone receptor antagonists.
  8. Have received any other investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.
  9. Have current substance dependence, or current substance (drug) or alcohol abuse.
  10. Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Crinecerfont
Crinecerfont capsule, administered orally, twice daily for 24 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 1 year.
Drug: Crinecerfont
CRF type 1 receptor antagonist
Other Names:
  • NBI-74788
  • Crenessity
Placebo Comparator: Placebo
Placebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
Drug: Placebo
Non-active dosage form
Drug: Crinecerfont
CRF type 1 receptor antagonist
Other Names:
  • NBI-74788
  • Crenessity

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24
Time Frame: Baseline, Week 24
Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.
Baseline, Week 24

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4
Time Frame: Baseline, Week 4
Baseline, Week 4
Change From Baseline in Serum Androstenedione at Week 4
Time Frame: Baseline, Week 4
Baseline, Week 4
Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 24
Time Frame: Week 24
Week 24
Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24
Time Frame: Baseline, Week 24
Baseline, Week 24
Percent Change From Baseline in Body Weight at Week 24
Time Frame: Baseline, Week 24
Baseline, Week 24
Change From Baseline in Percent Total Fat Mass at Week 24
Time Frame: Baseline, Week 24
Baseline, Week 24
Change From Baseline in Blood Pressure at Week 24
Time Frame: Baseline, Week 24
Baseline, Week 24
Change From Baseline in Glucose Tolerance at Week 24
Time Frame: Baseline, Week 24
Baseline, Week 24
Change From Baseline in Waist Circumference at Week 24
Time Frame: Baseline, Week 24
Baseline, Week 24
Change From Baseline in Menstrual Regularity at Week 24
Time Frame: Baseline, Week 24
Baseline, Week 24
Change From Baseline in Testicular Adrenal Rest Tumor (TART) Volume at Week 24
Time Frame: Baseline, Week 24
Baseline, Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Neurocrine Biosciences

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Development Lead, Neurocrine Biosciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 16, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 19, 2023

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 24, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 24, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 29, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 11, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 20, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • NBI-74788-CAH3003
  • 2019-004873-17 (EudraCT Number)
  • 2023-509171-16-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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