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Congenital Adrenal HyperplasiaPhase 3CAHtalystNCT04490915

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

A Phase 3 study testing Crinecerfont in people with Congenital Adrenal Hyperplasia, run by Neurocrine Biosciences at 70 sites in 18 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
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
182 participants
Where
70 sitesin 18 countries
Study dates
16 Dec 2020to 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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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.
  • Participants of childbearing potential must agree to use an acceptable method of contraception during the study.

Who cannot join

  • Have a diagnosis of any of the other known 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 cured.
  • Are pregnant.
  • Have a known history of clinically significant arrhythmia or abnormalities on ECG.
Show all 10Show fewer
  • Have a known hypersensitivity to any corticotropin releasing hormone receptor antagonists.
  • 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.
  • Have current substance dependence, or current substance (drug) or alcohol abuse.
  • Have had a blood loss ≥550 mL or donated blood or blood products within 8 weeks prior to the study.

Trial locations and contacts

70 sites in 18 countries.

70 sites in 18 countries

United States20 sites

  • Neurocrine Clinical Site

    Ann Arbor, Michigan, 48109

  • Neurocrine Clinical Site

    Atlanta, Georgia, 30329

  • Neurocrine Clinical Site

    Aurora, Colorado, 80045

  • Neurocrine Clinical Site

    Bethesda, Maryland, 20982

  • Neurocrine Clinical Site

    Boston, Massachusetts, 02115

  • Neurocrine Clinical Site

    Dallas, Texas, 75390

  • Neurocrine Clinical Site

    Great Neck, New York, 11021

  • Neurocrine Clinical Site

    Indianapolis, Indiana, 46202

  • Neurocrine Clinical Site

    Los Angeles, California, 90027

  • Neurocrine Clinical Site

    Minneapolis, Minnesota, 55454

  • Neurocrine Clinical Site

    New York, New York, 10029

  • Neurocrine Clinical Site

    Philadelphia, Pennsylvania, 19104

  • Neurocrine Clinical Site

    Pittsburgh, Pennsylvania, 15224

  • Neurocrine Clinical Site

    Rochester, Minnesota, 55905

  • Neurocrine Clinical Site

    San Diego, California, 92123

  • Neurocrine Clinical Site

    San Francisco, California, 94143

  • Neurocrine Clinical Site

    Seattle, Washington, 98105

  • Neurocrine Clinical Site

    St Louis, Missouri, 63110

  • Neurocrine Clinical Site

    Tulsa, Oklahoma, 74135

  • Neurocrine Clinical Site

    Winston-Salem, North Carolina, 27157

Other trials in United States

Italy9 sites

  • Neurocrine Clinical Site

    Ancona, 60126

  • Neurocrine Clinical Site

    Bologna, 40138

  • Neurocrine Clinical Site

    Florence, 50139

  • Neurocrine Clinical Site

    Messina, 98125

  • Neurocrine Clinical Site

    Milan, 20132

  • Neurocrine Clinical Site

    Milan, 20149

  • Neurocrine Clinical Site

    Naples, 80131

  • Neurocrine Clinical Site

    Padova, 35128

  • Neurocrine Clinical Site

    Roma, 00161

Other trials in Italy

France6 sites

  • Neurocrine Clinical Site

    Angers, 49933

  • Neurocrine Clinical Site

    Grenoble, 38700

  • Neurocrine Clinical Site

    Le Kremlin-Bicêtre, 94270

  • Neurocrine Clinical Site

    Nantes, 44093

  • Neurocrine Clinical Site

    Paris, 75651

  • Neurocrine Clinical Site

    Paris, 75679

Other trials in France

Germany5 sites

  • Neurocrine Clinical Site

    Dresden, 01307

  • Neurocrine Clinical Site

    Essen, 45147

  • Neurocrine Clinical Site

    Frankfurt, 60590

  • Neurocrine Clinical Site

    Leipzig, 04103

  • Neurocrine Clinical Site

    Munich, 80336

Other trials in Germany

Greece4 sites

  • Neurocrine Clinical Site

    Athens, 106 76

  • Neurocrine Clinical Site

    Athens, 115 27

  • Neurocrine Clinical Site

    Athens, 11527

  • Neurocrine Clinical Site

    Thessaloniki, 54642

Other trials in Greece

Israel4 sites

  • Neurocrine Clinical Site

    Afula, 1834111

  • Neurocrine Clinical Site

    Beersheba, 8410101

  • Neurocrine Clinical Site

    Petah Tikva, 4941480

  • Neurocrine Clinical Site

    Tel Aviv, 64239

Other trials in Israel

United Kingdom4 sites

  • Neurocrine Clinical Site

    Cardiff, CF14 4HH

  • Neurocrine Clinical Site

    London, WC1B 5EH

  • Neurocrine Clinical Site

    Manchester, M20 4BX

  • Neurocrine Clinical Site

    Salford, M6 8HD

Other trials in United Kingdom

Poland3 sites

  • Neurocrine Clinical Site

    Krakow, 31-011

  • Neurocrine Clinical Site

    Poznan, 60-355

  • Neurocrine Clinical Site

    Warsaw, 01-809

Other trials in Poland

Austria2 sites

  • Neurocrine Clinical Site

    Graz, 8036

  • Neurocrine Clinical Site

    Vienna, 1090

Other trials in Austria

Belgium2 sites

  • Neurocrine Clinical Site

    Brussels, 1070

  • Neurocrine Clinical Site

    Leuven, 3000

Other trials in Belgium

Bulgaria2 sites

  • Neurocrine Clinical Site

    Sofia, 1431

  • Neurocrine Clinical Site

    Sofia, 1606

Other trials in Bulgaria

Spain2 sites

  • Neurocrine Clinical Site

    Madrid, 28034

  • Neurocrine Clinical Site

    Seville, 41013

Other trials in Spain

Sweden2 sites

  • Neurocrine Clinical Site

    Gothenburg, 41345

  • Neurocrine Clinical Site

    Stockholm, 17176

Other trials in Sweden

Canada1 site

  • Neurocrine Clinical Site

    Halifax, Nova Scotia, B3H 2Y9

Other trials in Canada

Czechia1 site

  • Neurocrine Clinical Site

    Hradec Králové, 50005

Other trials in Czechia

Netherlands1 site

  • Neurocrine Clinical Site

    Leiden, 2333

Other trials in Netherlands

Portugal1 site

  • Neurocrine Clinical Site

    Porto, 4200-319

Other trials in Portugal

Serbia1 site

  • Neurocrine Clinical Site

    Belgrade, 11000

Other trials in Serbia

About this study

Official title: 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).

Being tested
Crinecerfont, Placebo
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

    Also called NBI-74788, Crenessity

    CRF type 1 receptor antagonist

  • PlaceboDrug

    Non-active dosage form

Study groups in NCT04490915
Study groupRoleWhat they receive
PlaceboPlacebo comparatorPlacebo capsule, administered orally, twice daily for 24 weeks, followed by active treatment with crinecerfont for at least 1 year.
Receives: Drug: Crinecerfont; Drug: Placebo
CrinecerfontExperimentalCrinecerfont 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.
Receives: Drug: Crinecerfont

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24

    Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.

    Measured: Baseline, Week 24

Secondary outcomes (11)

  1. Change From Baseline in Serum Androstenedione at Week 4

    Measured: Baseline, Week 4

  2. Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 24

    Measured: Week 24

  3. Change From Baseline in Homeostatic Model Assessment of Insulin Resistance (HOMA-IR) at Week 24

    Measured: Baseline, Week 24

  4. Percent Change From Baseline in Body Weight at Week 24

    Measured: Baseline, Week 24

  5. Change From Baseline in Percent Total Fat Mass at Week 24

    Measured: Baseline, Week 24

  6. Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4

    Measured: Baseline, Week 4

  7. Change From Baseline in Blood Pressure at Week 24

    Measured: Baseline, Week 24

  8. Change From Baseline in Glucose Tolerance at Week 24

    Measured: Baseline, Week 24

  9. Change From Baseline in Waist Circumference at Week 24

    Measured: Baseline, Week 24

  10. Change From Baseline in Menstrual Regularity at Week 24

    Measured: Baseline, Week 24

  11. Change From Baseline in Testicular Adrenal Rest Tumor (TART) Volume at Week 24

    Measured: Baseline, Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Jul 2020
  2. Study start16 Dec 2020
  3. Primary completion19 Jul 2023
  4. Study completionAug 2027 (estimated)

Resources

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
182 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04490915
Sponsor's protocol ID
NBI-74788-CAH3003
Other IDs
2019-004873-17, 2023-509171-16-00, 2023, 2019
Acronym
CAHtalyst
Status last verified
Apr 2026
First submitted
24 Jul 2020
Last updated
11 May 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Auchus RJ, Hamidi O, Pivonello R, Bancos I, Russo G, Witchel SF, Isidori AM, Rodien P, Srirangalingam U, Kiefer FW, Falhammar H, Merke DP, Reisch N, Sarafoglou K, Cutler GB Jr, Sturgeon J, Roberts E, Lin VH, Chan JL, Farber RH; CAHtalyst Adult Trial Investigators. Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal Hyperplasia. N Engl J Med. 2024 Aug 8;391(6):504-514. doi: 10.1056/NEJMoa2404656. Epub 2024 Jun 1.

    PubMed 38828955

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 70 sites in 18 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 4 inclusion and 10 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 11 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 70 sites in United States, Italy, France, Germany, Greece and 13 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?

Percent Change From Baseline in Glucocorticoid Daily Dose at Week 24 (time frame: Baseline, Week 24). 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 NCT04490915, 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 11 May 2026.

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