Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
NCT ID: NCT04806451
Last Updated: 2025-02-05
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE3
103 participants
INTERVENTIONAL
2021-06-25
2027-08-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Crinecerfont
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.
Crinecerfont
CRF type 1 receptor antagonist
Placebo
Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.
Crinecerfont
CRF type 1 receptor antagonist
Placebo
Non-active dosage form
Interventions
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Crinecerfont
CRF type 1 receptor antagonist
Placebo
Non-active dosage form
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
* 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.
2 Years
17 Years
ALL
No
Sponsors
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Neurocrine Biosciences
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Development Lead
Role: STUDY_DIRECTOR
Neurocrine Biosciences
Locations
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Neurocrine Clinical Site
Birmingham, Alabama, United States
Neurocrine Clinical Site
Los Angeles, California, United States
Neurocrine Clinical Site
Orange, California, United States
Neurocrine Clinical Site
San Diego, California, United States
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San Francisco, California, United States
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Aurora, Colorado, United States
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Hartford, Connecticut, United States
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Washington D.C., District of Columbia, United States
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Atlanta, Georgia, United States
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Indianapolis, Indiana, United States
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Boston, Massachusetts, United States
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Ann Arbor, Michigan, United States
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Minneapolis, Minnesota, United States
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St Louis, Missouri, United States
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New Hyde Park, New York, United States
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New York, New York, United States
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Oklahoma City, Oklahoma, United States
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Tulsa, Oklahoma, United States
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Philadelphia, Pennsylvania, United States
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Pittsburgh, Pennsylvania, United States
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Dallas, Texas, United States
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Seattle, Washington, United States
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Brussels, , Belgium
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Ghent, , Belgium
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Edmonton, Alberta, Canada
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Vancouver, British Columbia, Canada
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Montreal, Quebec, Canada
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Angers, , France
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Bordeau, , France
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Le Kremlin-Bicêtre, , France
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Paris, , France
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Paris, , France
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Berlin, , Germany
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Heidelberg, , Germany
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Magdeburg, , Germany
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Athens, , Greece
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Athens, , Greece
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Bologna, , Italy
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Milan, , Italy
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Napoli, , Italy
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Roma, , Italy
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Gdansk, , Poland
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Rzeszów, , Poland
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Barcelona, , Spain
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Seville, , Spain
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London, , United Kingdom
Countries
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References
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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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2020-004381-19
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-509170-33-00
Identifier Type: CTIS
Identifier Source: secondary_id
NBI-74788-CAH2006
Identifier Type: -
Identifier Source: org_study_id
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