Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Last updated: January 10, 2025
Sponsor: Neurocrine Biosciences
Overall Status: Active - Not Recruiting

Phase

3

Condition

Congenital Adrenal Hyperplasia

Male Hormonal Deficiencies/abnormalities

Treatment

Placebo

Crinecerfont

Clinical Study ID

NCT04806451
NBI-74788-CAH2006
2020-004381-19
2023-509170-33-00
  • Ages 2-17
  • All Genders

Study 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. 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).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Be willing and able to adhere to the study procedures, including all requirements atthe study center, and return for the follow-up visit.

  • Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylasedeficiency.

  • Be on a stable steroid regimen.

  • Have elevated androgen levels.

  • Participants of childbearing potential must be abstinent or agree to use appropriatebirth control during the study.

Exclusion

Exclusion Criteria:

  • Have a diagnosis of any of the other forms of classic CAH.

  • Have a history of bilateral adrenalectomy, hypopituitarism, or other conditionrequiring chronic glucocorticoid therapy.

  • Have a clinically significant unstable medical condition or chronic disease otherthan CAH.

  • Have a history of cancer unless considered to be cured.

  • Have a known history of clinically significant arrhythmia or abnormalities onelectrocardiogram (ECG).

  • Have a known hypersensitivity to any corticotropin-releasing hormone antagonist.

  • Have received an investigational drug within 30 days before initial screening orplan 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 weeksprior to the study.

Study Design

Total Participants: 103
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 3
Study Start date:
June 25, 2021
Estimated Completion Date:
August 31, 2027

Connect with a study center

  • Neurocrine Clinical Site

    Brussels, 1200
    Belgium

    Site Not Available

  • Neurocrine Clinical Site

    Ghent, 9000
    Belgium

    Site Not Available

  • Neurocrine Clinical Site

    Edmonton, Alberta T6G 1C9
    Canada

    Site Not Available

  • Neurocrine Clinical Site

    Vancouver, British Columbia V6H 3V4
    Canada

    Site Not Available

  • Neurocrine Clinical Site

    Montréal, Quebec H3T 1C5
    Canada

    Site Not Available

  • Neurocrine Clinical Site

    Angers, 49933
    France

    Site Not Available

  • Neurocrine Clinical Site

    Bordeau, 33076
    France

    Site Not Available

  • Neurocrine Clinical Site

    Le Kremlin-Bicêtre, 94270
    France

    Site Not Available

  • Neurocrine Clinical Site

    Paris, 75015
    France

    Site Not Available

  • Neurocrine Clinical Site

    Berlin, 13353
    Germany

    Site Not Available

  • Neurocrine Clinical Site

    Heidelberg, 69120
    Germany

    Site Not Available

  • Neurocrine Clinical Site

    Magdeburg, 39120
    Germany

    Site Not Available

  • Neurocrine Clinical Site

    Athens, 11527
    Greece

    Site Not Available

  • Neurocrine Clinical Site

    Bologna, 40138
    Italy

    Site Not Available

  • Neurocrine Clinical Site

    Milan, 20132
    Italy

    Site Not Available

  • Neurocrine Clinical Site

    Napoli, 80131
    Italy

    Site Not Available

  • Neurocrine Clinical Site

    Roma, 00165
    Italy

    Site Not Available

  • Neurocrine Clinical Site

    Gdańsk, 80-214
    Poland

    Site Not Available

  • Neurocrine Clinical Site

    Rzeszów, 35-301
    Poland

    Site Not Available

  • Neurocrine Clinical Site

    Barcelona, 08035
    Spain

    Site Not Available

  • Neurocrine Clinical Site

    Sevilla, 41013
    Spain

    Site Not Available

  • Neurocrine Clinical Site

    London, WC1N 3JH
    United Kingdom

    Site Not Available

  • Neurocrine Clinical Site

    Birmingham, Alabama 35233
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Los Angeles, California 90027
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Orange, California 92868
    United States

    Site Not Available

  • Neurocrine Clinical Site

    San Diego, California 92123
    United States

    Site Not Available

  • Neurocrine Clinical Site

    San Francisco, California 94158
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Hartford, Connecticut 06106
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Washington, District of Columbia 20010
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Atlanta, Georgia 30329
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Indianapolis, Indiana 46202
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Minneapolis, Minnesota 55454
    United States

    Site Not Available

  • Neurocrine Clinical site

    Saint Louis, Missouri 63104
    United States

    Site Not Available

  • Neurocrine Clinical Site

    New Hyde Park, New York 11040
    United States

    Site Not Available

  • Neurocrine Clinical Site

    New York, New York 10065
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Queens, New York 11040
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Oklahoma City, Oklahoma 73104
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Tulsa, Oklahoma 74135
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Pittsburgh, Pennsylvania 15224
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Dallas, Texas 75235
    United States

    Site Not Available

  • Neurocrine Clinical Site

    Seattle, Washington 98105
    United States

    Site Not Available

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