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

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Congenital Adrenal Hyperplasia

In brief

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).

Key facts

Study ID: NCT04806451
Run by: Neurocrine Biosciences
People needed: 103
Starts: 2021-06-25
Expected to finish: 2027-08-01
Last updated by the study team: 2025-02-05

Who can join

Age: 2 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

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.
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.

Where it is running

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
Neurocrine Clinical Site — San Francisco, California, United States
Neurocrine Clinical Site — Aurora, Colorado, United States
Neurocrine Clinical Site — Hartford, Connecticut, United States
Neurocrine Clinical Site — Washington D.C., District of Columbia, United States
Neurocrine Clinical Site — Atlanta, Georgia, United States
Neurocrine Clinical Site — Indianapolis, Indiana, United States
Neurocrine Clinical Site — Boston, Massachusetts, United States
Neurocrine Clinical Site — Ann Arbor, Michigan, United States
Neurocrine Clinical Site — Minneapolis, Minnesota, United States
Neurocrine Clinical site — St Louis, Missouri, United States
Neurocrine Clinical Site — New Hyde Park, New York, United States
Neurocrine Clinical Site — New York, New York, United States
Neurocrine Clinical Site — Oklahoma City, Oklahoma, United States
Neurocrine Clinical Site — Tulsa, Oklahoma, United States
Neurocrine Clinical Site — Philadelphia, Pennsylvania, United States
Neurocrine Clinical Site — Pittsburgh, Pennsylvania, United States
Neurocrine Clinical Site — Dallas, Texas, United States
Neurocrine Clinical Site — Seattle, Washington, United States
Neurocrine Clinical Site — Brussels, Belgium
Neurocrine Clinical Site — Ghent, Belgium
Neurocrine Clinical Site — Edmonton, Alberta, Canada

Full record on ClinicalTrials.gov

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